2025 BOD Election - Candidate Profiles - Oligonucleotide Therapeutics Society

Annemieke Aartsma-Rus, PhD

Leiden University Medical Center

PERSONAL STATEMENT/MOTIVATION:

After a mandatory break from the OTS Board of Directors, I hope to be able to return to the Board. I have been passionate about the OTS since attending the first conference in New York and am eager to actively contribute. The OTS focuses on the science and the people doing the science, especially the next generation of oligonucleotide researchers. I share this vision.

I will bring my organizational skills and also some ‘corporate memory’ due to previous board memberships, and having had the privilege of being the President of the Society from 2019-2021.

Furthermore, I will be the link between the Board and the Society Journal, Nucleic Acids Therapeutics, for which I am co-editor in chief.

I am currently participating in the mentorship program of the society and have been on several scientific organizing committees for annual meetings including being meeting (co)chair for the meeting in Leiden and Phoenix. I have served on the award committee jury multiple times. I also had the privilege of being the keynote speaker at the 2024 meeting in Montreal.

I will continue to help the OTS whether I am a member of the Board or not, but I think I can contribute more if I am part of the Board.

BIOGRAPHY:

I am one of the pioneers of the antisense oligonucleotide mediated exon skipping approach for Duchenne muscular dystrophy. I started working on this topic during my PhD studies as a side project and have been and still am actively involved in preclinical efforts to optimize this approach. I am currently a professor of translational genetics, leading a group working on Duchenne muscular dystrophy and exon skipping, an co-leading (with Prof. Willeke van Roon-Mom) a group working on N=1 antisense oligonucleotide therapies for patients in the Netherlands with nanorare mutations.

I have published extensively on both topics. Furthermore, I am actively involved in patient-education, explaining to Duchenne families and rare disease families how different therapeutic approaches work and what the current state of the art is.

AFFILIATIONS:

I am co-chair of the scientific advisory board of the N=1 collaborative, chair of the TREAT-NMD advisory committee for therapeutics, serve on various advisory boards for companies working in the Duchenne muscular dystrophy or oligonucleotide fields, am co-editor in chief for Nucleic Acids Therapeutics and serve on advisory boards / review panels for patient organization in the Duchenne or muscle disease field.

Marc Abrams, PhD

SanegeneBio

PERSONAL STATEMENT/MOTIVATION:

I am extremely optimistic about the future of oligonucleotide therapeutics, particularly for common indications including cardiometabolic diseases, oncology and infectious diseases. My perspective and outlook have been shaped by 20+ years in the space. After completing my Biochemistry PhD where I gained my first experience using siRNAs as research tools, I was fortunate to have been a biopharma discovery biologist at Merck in the early days of the field. One of my roles was to forge collaborations with our internal therapeutic area scientists, who were mostly skeptical of the potential of RNAi as a drug modality in this pre-Nobel Prize, pre commercialization era. After helping to establish siRNA platforms, delivery technology and development programs as part of a pioneering team Merck/SirnaTx, I moved on to leadership roles at Dicerna Pharmaceuticals (now a division of Novo Nordisk), and now at SanegeneBio where I serve as CTO. It has been amazing to have participated in decades of progress with inspiring colleagues, mentors, employees and teams, and this is only the beginning. It has become the mission of my career to unlock new indications for RNAi by promoting technology innovation, iterating both the nucleic acid payload and the delivery chemistry. If elected to the OTS board, I would be most interested in promoting the development of RNAi not only for chronic indications, but also for the incredible opportunities in the prevention and longevity space. I want the message to be heard worldwide (including in China, where my current employer has significant operations) that oligonucleotide therapeutics will soon be as or more impactful to human health than any other class of genetic medicine or biologics. I also want to promote more seamless partnerships between industry and academia across US/EU and APAC, for the benefit of the scientific community. I feel strongly that I can support and help advance progress in the OligoTx community as an OTS board member. As biopharma leader in a company with a China presence and track record in oligonucleotide therapeutics development form both big pharma and small biotech, I feel that I could bring a strong and unique skill set to OTS as the field evolves.

BIOGRAPHY:

I am currently serving as Chief Technology Officer & Head of US Operations at SanegeneBio, an RNAi medicines company with R&D in the US and China focused on cardiometabolic and autoimmune diseases. This role underscores the opportunity to promote collaboration between scientists, clinicians, investors and other stakeholders across the US, EU and APAC for the advancement of the field. I have been involved in genetic medicines development in biopharma for more than 20 years, previously as CSO of Carbon Biosciences, SVP of Discovery Research at Dicerna Pharmaceuticals (a Novo Nordisk company), and roles of increasing responsibility at Merck and Co, Inc. I have also been fortunate to serve on scientific advisory boards of other oligo-focused companies and have consulted for startups and investors. My therapeutic area experience spans diverse indication spaces including oncology, antiviral and metabolic diseases; and am particularly proud of involvement on teams developing the siRNAs Rivfloza® (nedosiran) for primary hyperoxaluria, xalnesiran for chronic HBV infection, and lepodisiran for cardiovascular disease. I am also passionate about platform development, including as a key inventor of GalXC-Plus™ and other extrahepatic delivery technologies. My academic training includes a Ph.D. in Biochemistry from Thomas Jefferson University.

AFFILIATIONS:

None currently, other than my employer.

Julia Alterman, PhD

RTI/UMass Chan Medical School

PERSONAL STATEMENT/MOTIVATION:

I have been attending OTS meetings since I was a Research Associate in Anastasia Khvorova’s Lab in 2014. Through almost a decade of engagement in this community, I have come to revere the OTS as the most important and insightful contributor to the-oligonucleotide therapeutic field. I find the intersection of industry and academia particularly engaging as it supports the community’s goal of realizing the potential of oligonucleotide therapeutics to impact human health, while encouraging innovative new discoveries in chemistry and biology of disease. Over my career, I have gained experience from both industry and academia.I worked for 6 years in two small biotech companies from 2007-2013, after which I earned my PhD at the RNA Therapeutics Institute at UMass Chan Medical School focused on siRNA development for CNS conditions that led to the establishment of a new biotech start up. I learned to appreciate the dedication required to ensure the success of a small biotech company while also grasping the scientific rigor necessary for pre-clinical development of novel therapeutics.

As an Assistant Professor,I am most fascinated by oligonucleotide chemical biology and understanding the role chemistry plays in impacting pharmacokinetics/pharmacodynamics. In this role,I have been working on developing these molecules for use in multiple organ systems(skin, muscle, eye, and bone), often in collaboration with industry partners. I believe that having a broad understanding of both the early research and late-stage pre-clinical work required for the development of successful technologies will complement the society’s mission of fusing both fields for the benefit of science and patients.

In addition to my research experience, I also cherish the opportunities I have had to mentor young scientists and have been part of the OTS mentoring program over the past year.

If given the honor to serve as a board member of the OTS I look forward to supporting the society’s mission by sharing new ideas,helping to build the growing academia-industry relations fueled by OTS,and engaging in trainee development, much like the OTS has done for me.

BIOGRAPHY:

I began my scientific career in chemical biology and in vitro pharmacology working at two small biotech companies in the Boston area. During this time, I worked on novel small molecules and peptide-lipid conjugates for the treatment of inflammatory indications and metabolic diseases, respectively.

In 2014, I joined Anastasia Khvorova’s lab at the University of Massachusetts Chan Medical School to pursue my PhD developing chemically durable, safe, and effective therapeutic oligonucleotides. My work focused on a novel branched siRNA scaffold with widespread and potent silencing in the central nervous system, which has been utilized as a tool for multiple labs interested in CNS and was licensed exclusively to Atalanta Therapeutics focused on developing this structure for the treatment of neurodegenerative diseases.

I am currently an Assistant Professor at UMass Chan Medical School, with a research focus on “solving” the problem of oligonucleotide delivery to extra hepatic tissues. My group is working on targeting skin, muscle, bone, and eye, as well as designing and identifying hyper-functional sequences for specific genetic targets.We are passionate about using our siRNA chemical toolbox in collaboration with academia and industry to support quick and targeted therapeutic solutions to a wide array of genetic diseases.

AFFILIATIONS:

Assistant Professor, RNA Therapeutics Institute, UMass Chan Medical School
Editorial Board, MTNA
SAB (Vect-Horus, GenKardia, DUBTx, RNAmed)

Sritama Bose, PhD

Orfonyx Bio, Oxford, UK

PERSONAL STATEMENT/MOTIVATION:

I am applying for the Oligonucleotide Therapeutics Society (OTS) Board of Directors with a deep commitment to its mission. Committed to oligonucleotide chemistry across academic, government- funded, and industry settings, I align with the OTS mission to foster community, collaboration, innovation, and education. My unique blend of scientific depth and strategic leadership provides the diverse skill set necessary to effectively govern and strengthen the Society’s future.

My motivation to serve stems from a commitment to the global growth of our field. I am eager to contribute to OTS’s role in uniting experts and accelerating the translation of research. Specifically, I intend to leverage my experience in program development, stakeholder management, and international collaboration, to enhance the OTS’s reach and impact, particularly in expanding educational and research opportunities.

The core talents and skills I would bring to the Board directly address the responsibilities outlined for directors:

Strategic Leadership and Governance: As an Associate Director of Chemistry at Orfonyx Bio and a former member of the Senior Leadership Team at NATA, I have extensive experience in strategic decision-making, project portfolio review, and performing go/no-go assessments. I am proficient in budget management and financial oversight required to develop the annual budget and ensure fiscal responsibility (Role 3).
Scientific and Translational Expertise: My Ph.D. in organic chemistry and active involvement in oligonucleotide research, evidenced by patents and peer-reviewed publications on oligonucleotide conjugates provide a current and relevant perspective on scientific excellence and innovation. I currently serve as a Scientific Advisory Board member for a major conference and am an Elected Committee Member of the Royal Society of Chemistry Nucleic Acid Group, demonstrating existing commitment to community service.
Diversity and Outreach: I am a strong advocate for dissemination and outreach. While at NATA, I created the first international secondment position and led work experience programs for students from socio-economically backward schools. I am committed to enhancing the organization’s public standing (Role 7) and ensuring OTS remains a welcoming, diverse, and accessible platform for the next generation of scientists.

My qualifications are rounded out by formal training, including the ILM recognized Leadership and Management Development Programmes from UKRI, ensuring I bring tested leadership methodologies to the Board. I am prepared to fulfill the fiduciary duties, actively participate in subcommittees, and speak on behalf of the organization to the community, helping the OTS maintain its trajectory as the premier global society for oligonucleotide therapeutics.

BIOGRAPHY:

I am a passionate and results-oriented leader with a demonstrated history of working in academia and industry. Currently I serve as Associate Director of Chemistry at Orfonyx Bio, a biotech co-founded by Professor Matthew Wood from University of Oxford. I lead the oligonucleotide chemistry team, overseeing project execution across various therapeutic targets and line-managing scientists. As part of the senior leadership team, I am involved in strategic decision making and identifying new discovery opportunities. I am responsible for managing external CROs and developing collaborative partnerships with academic institutions. Previously, I served as the Head of Chemistry Research & Innovation at MRC’s Nucleic Acid Therapy Accelerator. I was responsible for setting up the research and innovation facility and building the research and innovation team. I successfully built an in-house portfolio for NATA by establishing a conjugation platform and developing new technologies like novel linker chemistries for oligonucleotide therapeutics. I led a team of five scientists and managed multiple collaborative and in-house projects. As part of the Senior Leadership Team, I was involved in strategic decision making, reviewing the project portfolio, and being actively involved in go-no go decision making for two research consortia which NATA funded.

I earned my PhD in synthetic organic chemistry from the Indian Association for the Cultivation of Science, specializing in total synthesis of bioactive natural products. My diverse international postdoctoral experiences include research at Peking University, Stellenbosch University, and Slovak Academy of Sciences. I specialized in nucleic acid chemistry as a Postdoctoral Researcher at Durham University, UK. With a strong drive for translational research, I transitioned to Sygnature Discovery, a leading drug discovery CRO. As a Senior Scientist at Sygnature, I applied my organic synthesis expertise to drug development, gaining valuable medicinal chemistry insights that I now bring to my leadership role at Orfonyx.

AFFILIATIONS:

Elected Committee Member of Royal Society of Chemistry Nucleic Acid Group (RSC NAG).
Scientific Advisory Board Member for International Oligonucleotide & Peptide Conference (IOPC)
Mentor at Royal Society of Chemistry (RSC) Mentorloop Program
Member of Royal Society of Chemistry (RSC)
Member of American Chemical Society (ACS)
Member of Royal Institution (Ri), UK

Ronald Buijsen, PhD

LUMC

PERSONAL STATEMENT/MOTIVATION:

I am eager to serve on the Oligonucleotide Therapeutics Society Board of Directors because I am committed to advancing oligonucleotide therapeutics and fostering an inclusive, collaborative, and innovative scientific community. The Board plays a critical role in shaping OTS’s strategic priorities, supporting emerging scientists, and accelerating the translation of RNA-targeting therapies from bench to clinic. Serving on the Board would allow me to contribute my scientific expertise, leadership experience, and dedication to mentorship and community-building.

Over the past decade, I have focused on RNA-targeting research in neurodegenerative and rare neurological disorders. Beyond research, I have developed leadership and organizational skills through coordinating international research networks, leading work packages in translational programs, and mentoring PhD students and early-career scientists. These experiences have strengthened my ability to manage complex projects, facilitate collaborations across institutions and countries, and communicate scientific concepts effectively to diverse audiences.

My prior service within OTS has provided me with deep insight into the society’s operations and priorities. I have served as a junior Board member, co-chaired the scientific organizing committee for the 2021 annual OTS conference, chaired the OTS Local Delivery Grant program, organize and host the OTS webinar series, participate as a mentor in the OTS Mentorship Program and currently serve on the OTS Scientific Advisory Council. These roles have strengthened my skills in event planning, scientific communication, and community engagement, while allowing me to contribute directly to initiatives supporting both early-career researchers and established investigators. They have also given me a strong familiarity with OTS’s structure and priorities, which I can leverage to contribute effectively as a Board member. Additionally, my role as Chair of the Stichting Dutch Antisense Therapeutics has further developed my leadership and strategic planning experience in promoting ASO research and national collaboration. I am particularly committed to ensuring that young scientists and those new to the field have opportunities to engage with oligonucleotide therapeutics and develop their careers.

Beyond OTS-specific roles, I have a strong track record of mentoring and service across the scientific community. I supervise PhD students, organize courses and workshops on RNA therapies, and actively engage with patient communities to ensure research is translational. I also participate in committees and working groups focused on model systems, ASO delivery, and human disease modeling technologies, demonstrating my capacity to work collaboratively and contribute thoughtfully to strategic discussions.

I bring a combination of scientific expertise, leadership experience, mentoring skills, and familiarity with the society, along with a strong commitment to supporting early-career researchers. I am confident that my experience and dedication will enable me to contribute meaningfully to the Board’s mission, promoting innovation, career development, and the clinical translation of RNA therapeutics worldwide.

BIOGRAPHY:

My research focuses on translating RNA-targeting strategies into clinical applications for neurodegenerative and rare neurological disorders. During my PhD at Erasmus MC, I identified a protein gain-of-function mechanism driving Fragile X-associated tremor/ataxia syndrome (FXTAS) and demonstrated that RNA-targeting compounds can mitigate disease pathology. This work, which led to international collaborations and several peer-reviewed publications, strengthened my expertise in molecular mechanisms, RNA biology, and translational neurogenetics.

At the NeuroD group at Leiden University Medical Center, I expanded my research to SCA1, where I investigated preclinical development of ASO therapies. I have secured multiple competitive grants, including from the Dutch Brain Foundation, ZonMw, and Ataxia UK, and recently initiated a project focused on allele-specific RNA-targeting strategies for SCA1. I also coordinate the “brain” work package in the EFFecT Marie Skłodowska-Curie Doctoral Network, aimed at advancing oligonucleotide therapeutics and delivery strategies across tissues.

My scientific leadership extends beyond research execution. I supervise PhD students, mentor early- =career scientists, and foster international collaborations that bridge basic and translational science. I developed patient-derived hiPSC models to better understand disease mechanisms and evaluate RNA-targeting therapies, collaborating with groups across Europe. As Chair of the Stichting Dutch Antisense Therapeutics, I help coordinate national efforts to promote ASO research and collaboration.

I received the OTS Dr. Alan M. Gewirtz Memorial Scholarship for postdocs for my contributions to the field. My overarching goal is to accelerate the development of patient-specific RNA-targeting therapies through innovative model systems, strong collaboration, and mentorship, translating molecular insights into meaningful therapies for patients with neurodegenerative diseases.

AFFILIATIONS:

Member OTS Scientific Advisory Council
Chair Stichting Dutch Antisense Therapeutics
Lead working group model systems & preclinical trials Ataxia Global Initiative
Member Bachelor Internship Committee Biomedical Sciences LUMC

Michael J Byrne, PhD

Wave Life Sciences

PERSONAL STATEMENT/MOTIVATION:

My PhD involved looking at micro RNA expression and its control of embryo development, completed in 2008. This led to a job at RXi Pharmaceuticals, a company started by Craig Mello and eventually led by Anastasia Khovorva as CSO. RXi was small, really small, at one point with 10 people we had discovery and clinical programs. That size provides a person a lot of opportunities to experience all aspects of drug development, at least to Phase II. I absorbed all I could and led and IND and clinical startup for siRNA in the eye and contributed to our skin IND.

I would bring a broad understanding of the oligo drug development process to the society. I have done ASOs, siRNA, exon skipping molecules and ADAR editing oligos. I would also bring a historical knowledge of troubleshooting these programs at almost all stages. I have enjoyed my hybrid career as someone who does oligo drug discovery leadership and the pharmacology leadership on how to test and evaluate top oligos.

I’ve been an athlete and coach my entire life, and I find that mentoring and working with scientists—helping to build a team and build people up—moves things forward and is incredibly exciting and rewarding. I believe matrix working environments internally and collaboration externally are key. Sharing of ideas and experiences advances everyone and helps patients. I’ve been part of the society for many years, attended many meetings, and know many of you. I look forward to the opportunity to work with the society to help the next generation of scientists understand how great this field of study is, and the people who study within it.

BIOGRAPHY:

I’ve spent my entire career working with oligonucleotides. My PhD involved looking at micro RNA expression and its control of embryo development, which led me into a job at RXi Pharmaceuticals, a company started by Craig Mello and eventually led by Anastasia Khovorva. I worked on cholesterol-conjugated siRNAs and took those into the clinic in skin and the eye.

I spent eight and a half years at RXi running CNS and ocular programs and then moved to WAVE Life Sciences, where I’ve already spent another eight-plus years. I was not convinced of the role of stereochemistry in oligos when I joined WAVE, but quickly came to understand that great platforms are integral in moving this field forward and that stereochemistry is one of the key contributors to an oligo’s effectiveness. At the annual meeting we currently get to see many great platforms delivering unique ways to solve big problems. I’ve led CNS, ocular, muscle and other extrahepatic programs while at Wave.

I have now led programs with siRNAs, ASOs, and exon skipping molecules. My pharmacology team has managed the in vivo work for all programs at RXi and WAVE, including our ADAR editing programs. I have had the pleasure of managing programs and teams from early discovery to IND-enabling toxicology. I’ve prepared INDs and moved oligos into the clinic through phase one and two.

I enjoy mentorship and have been a mentor to a high school student outside of work for several years and this year I joined the OTS mentorship program.

I am hoping to bring a broad spectrum of experience in the oligo field and experience in team building to the society and contributing as part of the group wherever I might be most useful.

AFFILIATIONS:

OTS
ARVO
ASGCT
Former associate editor for Molecular Therapy
Reviewer for multiple journals

Masad J. Damha, PhD, FCIC

McGill University

PERSONAL STATEMENT/MOTIVATION:

I have been associated with the OTS since its first annual meeting in 2005 (Rockefeller U, NY) where I met some of the most prominent (and new then) figures in our field. I knew immediately I have found a Society to which I could contribute in a meaningful way. I participated first as poster and oral presenter, and later as board member, eventually serving as President of the Society (2012-13). Those were exciting years; new committees started to be formed under the leadership of our Board and subsequent Presidents (Brett Monia, Art Krieg, Annemieke Aartsma-Rus) and our Society witnessed great growth. When my term in the Board ended in 2014, I continued to serve in the Scientific Advisory Council, and co-hosted of the 12th Annual Meeting (Montreal, 2016 and 2024). I also introduced (and chaired) our Chemistry Sessions at OTS meetings for several years. I would like to continue working on the OTS Board of Directors, continuing building the mentorship program for new investigators (and members at large), facilitating their growth at their workplace, serving as ‘service point’ that connect them to academic and industrial centers. One of my best experiences as an academic and graduate supervisor is sharing my own personal experiences with new scientist and equipping them with tools that will help them move and succeed at the workforce. I would like to continue do this as a mentor not only at McGill but also at my “OTS “University!

BIOGRAPHY:

I teach Chemistry at McGill, and I work with students pursuing studies in Nucleic Acid Chemistry & Biology. We specialize in developing chemically modified oligonucleotides and sustainable methods for oligonucleotide synthesis (RNA, DNA and their analogues). Our studies have been essential in deciphering structure-function relationships between our oligomers and the enzymatic machinery associated with the antisense, RNAi, and CRISPR-Cas9/12a pathways. We start with chemistry to design and construct molecules, then test them biochemically/biophysically in vitro and, through collaborative work, we apply them in live-cells and animal disease models (Duchenne Muscular Dystrophy, ALS, etc.). For more information about our work, students, and scientific publications, please visit our website: http://damha-group.mcgill.ca/

AFFILIATIONS:

Department of Chemistry, McGill University
Board of Directors, OTS
Editorial Board, Nucleic Acids Therapeutics
SAB (Codexis, Deep Genomics, Hongene, Korro Bio)

Sarah L DeVos, PhD

Curie.Bio

PERSONAL STATEMENT/MOTIVATION:

From the beginning of my scientific career, my motivation has been clear: to transform the lives of patients and families affected by devastating neurodegenerative diseases. Oligonucleotide therapeutics have given me the rare privilege of seeing this vision move from hypothesis to clinical reality. I am seeking election to the OTS Board of Directors to help strengthen the community that has enabled these breakthroughs, expand the reach of our science, and mentor the next generation of leaders in the field.

One of the most meaningful milestones of my career was contributing to the preclinical data package that supported the development of BIIB080 (MAPT ASO), the first tau-targeting oligonucleotide advanced into the clinic. This work laid the foundation for a therapy that, in early trials, demonstrated reversal of pre-existing tau pathology in patients—a profound validation of the therapeutic power of oligonucleotides that has now expanded to other neurodegenerative targets.

Complementing this, at Denali Therapeutics I established the Oligonucleotide Transport Vehicle (OTV) platform, designed to deliver ASOs across the blood–brain barrier. As Project Team Lead, I shepherded OTV from initial proof-of-concept in mice and monkeys to multiple therapeutic programs advancing through IND-enabling stages. This work demonstrated the feasibility of systemic administration for oligonucleotides in the CNS (work presented at OTS), a major leap forward for the field.

Today, as Senior Director at Curie.Bio, I work with founders to launch and shape their early-stage oligonucleotide companies. I help guide scientific strategy, design preclinical packages, and provide direct hands-on leadership and support to ensure programs advance with rigor and urgency. This role allows me to apply lessons learned from BIIB080 and OTV while building the infrastructure for the next wave of oligonucleotide innovation.

In parallel to my scientific career, I have consistently invested in community service and mentorship. I have co-organized international conferences (Global Tau 2025, Keystone Neurodegeneration, Charleston Conference on AD), delivered invited talks at OTS and other leading meetings, mentored dozens of young scientists, and organized community events to support research for Alzheimer’s Disease. These experiences reinforced my belief in convening diverse voices, cultivating inclusivity, and ensuring that our progress translates into meaningful impact.

On the OTS Board, I would bring:

Domain expertise in CNS oligonucleotides, delivery platforms, and translational strategy.
Strategic and operational experience across academia, biotech, and venture creation.
A collaborative and inclusive mindset, with a strong record of mentorship and community
building.

It would be a privilege to contribute my energy, experience, and vision to advancing the Society’s mission worldwide.

BIOGRAPHY:

Sarah L. DeVos, Ph.D., is a neuroscientist and drug developer with over 16 years of experience advancing oligonucleotide and other novel therapeutics for neurodegenerative disease. Her career spans academic discovery, industry platform innovation, and company building, unified by a commitment to translating science into meaningful patient impact.

Dr. DeVos earned her doctorate in Neuroscience at Washington University in St. Louis in Dr. Tim Miller’s lab, where she initiated and led research that laid the foundation for BIIB080 (MAPT ASO), the first tau-targeting ASO to enter human clinical trials. Her work demonstrated the feasibility of tau reduction as a therapeutic strategy and contributed to the clinical translation of BIIB080, which has shown promising effects on reversing tau pathology in early patient studies. She then completed her postdoc at Massachusetts General Hospital / Harvard Medical School, where she expanded her expertise in tau biology and oligonucleotides through a BMS collaboration on their MAPT ASO program, further reinforcing her ability to bridge academic discovery with industry translation.

In 2018, Dr. DeVos joined Denali Therapeutics, where she helped conceive and lead the Oligonucleotide Transport Vehicle (OTV) platform, enabling systemic delivery of ASOs across the BBB. Under her leadership, OTV advanced from proof-of-concept in mice and NHPs into multiple IND-enabling programs and scaled into a cross-functional effort spanning biology, chemistry, protein engineering, and translational sciences.

Currently, Dr. DeVos serves as Senior Director at Curie.Bio, where she partners with founders to launch and guide early-stage oligonucleotide companies. She guides scientific strategy, drives experimental execution, and serves as Head of Drug Discovery for select oligonucleotide portfolio companies, ensuring robust preclinical packages to support investment and clinical advancement.

She has authored over 35 peer-reviewed publications, is an inventor on 12 patents, and has been invited to speak at 20+ international meetings including OTS & TIDES, and Cold Spring Harbor. She also serves on scientific advisory boards, co-organizes major conferences, and mentors early-career scientists.

AFFILIATIONS:

I am a full-time employee of Curie.Bio where I currently serve as a fractional employee of three
oligonucleotide portfolio companies within Curie.Bio.
I currently serve on the Advisory Board for the Charleston Conference on Alzheimer’s Disease as well
as the SAB for Synapticure.

MariJean Eggen, PhD

Eli Lilly

PERSONAL STATEMENT/MOTIVATION:

I am honored to apply for a position on the Oligonucleotide Therapeutics Society Board of Directors.
With over 25 years of experience in pharmaceutical R&D, I have led scientific and strategic initiatives across therapeutic areas, modalities, and technologies. My current role as Associate Vice President, ExploR&D Head of Molecule Discovery, reflects my commitment to advancing multimodal innovation, accelerating novel targets, and integrating external partnerships to shape the future of drug discovery.

The convergence of RNA therapeutics, delivery science, and translational biology is unlocking new possibilities for treating complex diseases. The deeply personal and groundbreaking story and treatment for Mila, marked a turning point for individualized oligonucleotide therapy we now feel as optimism for more patients—proof of the field’s rapid evolution and its potential to reshape medicine. By integrating the promise of RNA therapeutics with other therapeutic modalities, including small molecules, peptides, and antibodies, and using them as conjugates—we can tailor interventions to the biology of each condition, improve delivery and specificity, and overcome limitations of single-modality approaches.

Throughout my career, I have consistently focused on identifying the right mechanism of action—and now the right modality—to address the biological complexity of disease. Although RNA therapeutics is a relatively newer focus for me, I played a key role in establishing Lilly’s RNA discovery team in 2019, helping to build its foundational chemistry strategy, workflows, and delivery platforms. This approach has guided my work across modalities and conjugates—bringing together chemistry, biology, biophysics, and translational science to solve problems others deemed intractable. I have led the development of novel delivery systems and co-invented multiple patents and applications in genetic medicine and small molecules.

I bring to the Board a deep understanding of drug discovery, RNA and small molecule therapeutics, delivery strategies, and modality selection. I have built and led high-performing teams, managed large portfolios, and driven innovation through strategic clarity and collaborative execution. My ability to maintain focus amidst ambiguity, challenge assumptions constructively, and empower diverse teams has been central to my success.

Beyond scientific leadership, I am deeply committed to mentorship and inclusion. I have championed diversity across therapeutic teams, participated in panels for Women in Science, and served on committees within the American Chemical Society. I recently co-chaired the ACS MED session on oligonucleotide-based therapeutic discovery and actively contribute to conferences, advisory boards, and educational outreach.

Serving on the OTS Board would allow me to contribute my experience and vision to a community that is redefining the boundaries of therapeutic innovation and amplify its impact across industry, academia, and global health.

Thank you for your consideration.

BIOGRAPHY:

MariJean Eggen is Associate Vice President, ExploR&D Head of Molecule Discovery at Eli Lilly. In this role, MariJean leads efforts to partner with biotech innovators, leveraging Lilly’s scientific expertise and global resources to help accelerate the development of transformative medicines.

MariJean joined Lilly Genetic Medicine in 2019 as a founding member of RNA Therapeutics, making significant technical contributions in siRNA and ASO design for multiple therapeutic areas and tissues.

Her career began at Pharmacia as a medicinal chemist in infectious disease research, followed by joining Lilly in 2003 as a Senior Organic Chemist in Discovery Chemistry Research & Technology. Her final small molecule role was Director of Medicinal Chemistry- Explore leading ligand ID strategies, hit identification and resourcing for the entire Lilly small molecule portfolio. MariJean has developed a reputation for molecule innovation, design, and technology, earning recognition such as the Lilly President’s Research Award in 2010.

Externally, MariJean has served as an NSF panel reviewer, member of the Scientific Advisory Committee for Biophysics in Drug Discovery, plenary lecturer at the 2019 National Organic Chemistry Symposium, and member of the American Chemical Society MEDI long-range planning committee (2024–2026). She is recognized for building effective, diverse teams, leading research across therapeutic areas, and solving challenging problems.

She has published two book chapters including ‘RNA-based Agents as Therapeutics’ now published online as part of the Burger Medicinal Chemistry, Drug Discovery and Development (ISBN: 9780471266945).

MariJean holds a B.S. in Chemistry (minor in Biology) from North Dakota State University with Professor Mukund Sibi, a Ph.D. in Organic Chemistry from the University of Nebraska–Lincoln with David Berkowitz and completed postdoctoral research with Gunda I. Georg formerly of the University of Kansas focused on total synthesis of Cryptophycins.

AFFILIATIONS:

American Chemical Society (ACS) Member since 1992

- ACS Long Range Planning Committee – MEDI Division (2024–2026)
- WCC–Rising Star Selection Committee (2022)
- WCC–Eli Lilly Travel Award Committee (2023, 2024, 2025)
- Speaker at ACS Exceptional Leaders Conference (2024)
- Plenary Lecturer, National Organic Chemistry Symposium (2019)
- Cochair

Scientific Committee, Biophysics Drug Discovery Conference (2018–present)

- Chair, Small Molecules Targeting RNA/Understanding Small-Molecule Selectivity Toward RNA, 9th Biophysics in Drug Discovery Conference (2023)
- Chair, Protein Protein Interaction Inhibitor Session, 5th Annual Biophysics in Drug Discovery Conference (2018)

Advisory Boards

University of Nebraska–Lincoln Scientific Advisory Board (2014–present)
National Science Foundation (NSF)
- Panel Reviewer (2014, 2015)

Hassan H. Fakih, PhD

RTI/ UMass Chan Medical School

PERSONAL STATEMENT/MOTIVATION:

The OTS Society has had a profound impact on my life and career. As a graduate student in materials chemistry focused on drug delivery, I attended the 2019 OTS meeting, where I interacted with leading scientists and trainees in the field. This experience inspired me to pivot my research to oligotherapeutics. The meeting offered top-tier science, productive networking opportunities, and a strong focus on fostering the career and growth of young scientists.

Since then, I have actively engaged with the OTS community, attending the meeting every year, and helping host the educational webinars offered by the society. I also had the privilege to serve as a trainee representative to the board for two years. I have benefited immensely from the rich scientific environment and the mentorship and support of OTS members. I am eager to serve on the OTS board to give back to the community that has supported me so much. Since my first election to the board (2023), I have co-led the establishment of the OTS Mentorship Program, which pairs scientists in the community with experienced leaders to help elevate their careers. The program has become a fixed offering, with increasing participation every year.

As a board member, I would focus on three key areas:

Supporting the next generation of scientists and trainees. The OTS community has a vibrant culture of mentorship and collaboration. I would work to strengthen these programs and create new opportunities for young scientists to network, learn, and grow.
Expand on the educational reach of the society by helping to grow the educational webinars and organize regional meetings across the world (Asia, middle east, Australia)
Continue to work and improve on the mentorship program, by organizing new activities and implementing feedback from the society to maintain high standards.

I am confident that my skills, experience, and passion for oligotherapeutics would be a valuable asset to the OTS board. I am committed to serving the community and helping to advance the field.

Biography:

I am a Postdoctoral Associate in the lab of Prof. Anastasia Khvorova at the RNA Therapeutics Institute at UMass Chan Medical School. My research focuses on improving the extra-hepatic delivery of siRNA therapeutics using self-assembled nucleic acid particles and specific protein-binding conjugates. I am particularly interested in applying this research to develop treatments for cancer, muscle diseases, and CNS disorders, with a focus on multi-targeting strategies.

I received my B.Sc. in premedical chemistry from the American University of Beirut in 2016 and my Ph.D. in chemistry from McGill University in 2021. My Ph.D. work focused on developing DNA nanocarriers for nucleic acid therapeutics. I developed DNA-based nanocarriers with characteristics that enable them to move faster into clinical trials, such as improved cost-effectiveness, biocompatibility, specificity, and stability. The work resulted in the development of sequence-controlled nucleic acid-polymer conjugates that assemble into spherical nucleic acids (SNAs) with stimuli-responsive activation, as well as chemically modified SNAs that show promising efficacy without transfection agents.

Some of this work has been recognized by the OTS community, where I have received several awards for my research, including the Dr. Alan Gewirtz Memorial Scholarship for Graduate Students in 2021 and for Postdoctoral Fellows in 2024. I have also been actively involved with the OTS Society since 2020. I have helped host many of the OTS monthly webinars and served as a trainee representative on the board for the 2022-2023 term. In 2022, I hosted the next-generation session in Phoenix and co-led the establishment of the first official cohort of the OTS Mentorship Program, which is now a fixed yearly offering with an annual budget to support mentees in attending the annual meeting and other mentorship events.

Affiliations:

Scientific advisor to Banook Group on nucleic acid therapies (Banook Group, based in Nancy France, Montreal Canada, and Boston USA) 2022-active
OTS board member 2024-2025
Trainee Representative board member to the OTS 2022-2023
Controlled Release Society member (CRS and Canadian Chapter –CRS, VP communications)
Hereditary Disease Foundation Fellow 2022-2024
Society for the Advancement of Science and Technology in the Arab World (SASTA) member

Alex Garanto, PhD

Radboud university medical center

PERSONAL STATEMENT/MOTIVATION:

My first OTS was in Leiden back in 2015. At that meeting, I had the chance to give my first international talk on my postdoc work on antisense oligonucleotides for CEP290. I was amazed by the meeting, how the community made me feel and the feedback I got after my talk. Since I became a junior independent principal investigator in 2019, I have joined all Oligo meetings. In fact, in 2021 I had the great honor of receiving the Mary Ann Liebert, Inc. publishers Young Investigator Award and in 2022, Prof. Aartsma-Rus and Prof. Corey believed in me to serve as the scientific co-chair of the 2023 Annual Oligo meeting which was held in Barcelona. After that, I was elected as part of the board of directors, where I had the chance to return to the OTS community with their trust in me. I would like to continue serving on the board of directors to help maintain the continuity and spirit of the society and bring new perspectives from a relatively young European academic, and actively promoting diversity, equity and inclusion at multiple levels. This commitment was already visible during the OTS 2023 in Barcelona where we achieved balanced representation across gender and academia/industry, supported geographic inclusion, and encouraged early-career researchers. As such, I have been actively supporting mentorship and creating opportunities for the next generation of scientists who will play a crucial role in further developing and shaping our community. For example, I have participated in the mentoring program, and this year I co-organized an educational session designed to help newcomers gain knowledge on various aspects of oligonucleotide therapeutics.

I greatly enjoy building new connections and collaborating with colleagues from around the world. I have served on the management committee of the European network DARTER (2018–2022), I coordinate the European doctoral network EFFecT (2025–2028), and I co-founded the Dutch Antisense Therapeutics Society, which organizes an annual symposium. This initiative aims to strengthen the antisense field in the Netherlands and neighboring countries by providing an accessible platform for young researchers to connect and interact with both national and international experts in the field.

BIOGRAPHY:

I started my PhD project in the field of Genetics after finishing my studies in Biology at the University of Barcelona. After my PhD, I studied the contribution of Ubiquitin and Sumo signaling to the determination of photoreceptor cell fate. In October 2012, I moved to the Netherlands as a postdoc in the Blindness Genetic Therapy group at the Department of Human Genetics of the Radboudumc. During this time, I contributed to the development of novel therapeutic interventions for inherited retinal disorders based on antisense technology for CEP290 and ABCA4 retinal conditions. In 2017, I entered the tenure-track program which led to an Assistant Professor tenured position in 2021.Since2023, I am an Associate Professor and my research focuses on the development of molecular therapies, mainly on antisense technology, for these diseases. I share group with Prof. Collin and our group is embedded within the Departments of Pediatrics and Human Genetics and it is closely connected to the Translational Metabolic Laboratory and the Department of Ophthalmology of the Radboudumc, facilitating close contact between clinicians, researchers and patients. During the last years, our group has extensively worked on personalized medicine by developing mutation-specific RNA-based strategies to treat disease. Besides designing new therapeutic strategies, another important aspect of our research is to find reliable and suitable models to identify molecular mechanisms of disease and test potential new drugs. For that reason, we are actively working on the development of patient-derived cellular models and potential read-outs to not only elucidate the pathophysiological mechanisms and identify therapeutic targets, but also test the efficacy, safety, (bio)distribution and longevity of therapeutic molecules. Our group is well connected to several European consortia, such as CHARLIE (CHAnging Rare disorders of LysInE metabolism), ERTC (European Retinal Therapeutics Consortium), the terminated DARTER (Delivery of Antisense RNA ThERapeutics) European Action as well as the national hDMT organ-on-chip network or Dutch Center for RNA Therapeutics. More recently, the spirit of DARTER was continued in a European doctoral network called EFFecT (A European Training Program to Foster the Full Therapeutic Potential of Antisense Technology across Tissues), in which we aim to train 12 doctoral candidates in 8 different countries.

AFFILIATIONS:

Research Group Leader at the Radboudumc
Co-founder and Board member of the Dutch Antisense Therapeutics Society that organizes the annual Dutch Antisense Therapeutics Symposium (www.dutchantisense.org)
Member of the Oligonucleotide Therapeutics Society
Member of the Netherlands Society for Gene and Cell Therapy (NVGCT)
Member of the European Society for Gene and Cell Therapy (ESGCT)
Member of the Association for Research in Vision and Ophthalmology (ARVO)
Vice-chair of the hDMT Eye-on-Chip working group

Felix Gnerlich, PhD

Ribocure Pharmaceuticals

PERSONAL STATEMENT/MOTIVATION:

The OTS has been a cornerstone of my professional journey since I first attended an OTS meeting. As a chemist, I appreciate that OTS allows me to engage with like-minded brains who are dedicated to overcoming the technical challenges the field faced in the early 2010s. Even today, I feel a strong sense of community, despite the many new members drawn by the remarkable drug approvals and scientific advancements over the past decade.

Oligonucleotides have evolved from a niche innovation to a central focus for nearly all major pharmaceutical companies, and my career mirrors this evolution. I have moved between biotech and big pharma across Europe, the USA, and China, gaining exposure to diverse organizational cultures, scientific strategies, and leadership responsibilities. I have also had the privilege of working on a variety of technical platforms, including siRNA, ASOs, and immunostimulatory oligos, providing me with broad technical expertise and a deep understanding of the field.

In my current role, as Global Head of Chemistry, I am gaining fresh insights into oligonucleotide drug discovery through our integrated EU–China operation, where I am helping to align and integrate diverse scientific approaches to foster innovation. This experience has strengthened my ability to bridge teams across regions, balance strategic and technical priorities, and drive collaborative initiatives.

I am applying to the OTS Board of Directors because I want to contribute more directly to the Society that has supported my career and shaped the field.

I am particularly motivated to help expand international collaborations, mentor the next generation of oligonucleotide scientists, and support initiatives that ensure OTS continues to be a vibrant, inclusive, and innovative community.

I have been involved with the OTS mentorship initiative since its launch, and I look forward to continuing to inspire and guide early-career scientists, sharing my passion for oligonucleotide therapeutics and their potential to address unmet medical needs.

I believe my combination of scientific expertise, leadership experience across multiple regions, and commitment to fostering collaboration positions me to make a meaningful contribution to the OTS Board.

Serving on the Board would allow me to help shape the Society’s trajectory around the globe, support its members, and ensure that OTS remains at the forefront of oligonucleotide drug discovery.

BIOGRAPHY:

I am dedicated throughout my academic and professional career to advancing therapeutic oligonucleotides across a range of disease areas.

During my PhD in organic and nucleic acid chemistry with Prof. Thomas Carell at LMU Munich, I developed novel nucleotide building blocks and solid-phase synthesis methods to study the functions of epigenetic nucleobase modifications. During my PhD, I also worked on siRNA–carbohydrate conjugates and stable isotope-labeled nucleotides to investigate the solution structures of non- canonical bases.

In 2013, I joined Sanofi’s Nucleic Acid Therapeutics Platform in Germany, where I led a team focused on the synthesis of therapeutic siRNA libraries and peptide- and carbohydrate-conjugated siRNAs to support early-stage drug discovery programs.

In 2017, I moved to Rigontec, a clinical-stage biotech company, where I built and led the Chemistry R&D team. There, I helped establish oligonucleotide chemistry capabilities to develop immunostimulatory RNAs as cancer therapeutics. The company was acquired by Merck & Co. (MSD) just three years after its founding, which marked a major success for the startup.

Following the acquisition, I joined AstraZeneca in Sweden as Director of New Modalities – Oligonucleotides within the Medicinal Chemistry department. I led targeted delivery and therapeutic oligonucleotide projects, contributed to the early-stage portfolio, and helped shape AstraZeneca’s oligonucleotide strategy in respiratory and immunology diseases.

Since 2024, I have been serving as Global Head of Chemistry at Ribocure Pharmaceuticals in Sweden and its parent company Suzhou Ribo in China. Ribocure is a clinical-stage biotech focused on siRNA therapeutics, operating under an integrated “bench-to-bedside” model with its own clinical trial unit. Our mission is to address unmet medical needs in rare cardiovascular, renal, and oncology diseases, and I’m proud to play a key role in driving innovation and advancing our scientific capabilities.

AFFILIATIONS:

No further affiliations

Bruno Miguel da Cruz Godinho, PhD

Atalanta Therapeutics

PERSONAL STATEMENT/MOTIVATION:

My journey with the Oligonucleotide Therapeutics Society (OTS) began in 2016 at the Annual Meeting in Montreal. The quality of the meeting and the energy of the scientists/members that make the fabric of this community was astounding. Adding a passion for RNA therapeutics, and it was hard not to get hooked! Since then, the OTS has been my go-to platform for learning about the latest advances in genetic medicines and finding inspiration from leaders in the field.

For the past four years, it’s been an honor to serve on the OTS Board. I’ve been fortunate to witness the dedication that goes into every decision, and I’ve actively contributed in various ways.

During my two terms, I chaired the location committee, which defines where the annual meeting is going next; and supported the activities of a variety of subcommittees. Contributed in several instances, as a poster reviewer, travel grant reviewer, and mentor on the mentorship program. This year I had the privilege of being nominated co-chair of the Annual Meeting in Budapest (Hungary); and have previously served on the organizing committees for the 2021 and 2023 Annual Meetings, where I co-chaired pre-clinical sessions. Finally, I have also supported other OTS-backed activities like local delivery meetings, such as the 1st Symposium on Oligonucleotide Technologies & Therapeutics in Portugal.

The OTS has provided me with invaluable opportunities, and I am committed to give back and offer my unique perspective. Thus, it would be a privilege to continue to share the responsibility of shaping the future of the society, as well as to help educate/train the next generation of oligonucleotide scientists and the broader community on the transformative potential of oligonucleotides in human medicine.

BIOGRAPHY

Bruno Godinho received his foundational pharmacy training at the Lisbon School of Health Technology (ESTESL, Portugal) where he obtained his Bachelor’s degree. During his time in college he undertook research internships at Utrecht University (Utrecht, TheNetherlands) and Universidade NOVA de Lisboa (Caparica, Portugal), which were key shaping his interest in pursuing a career in scientific research. His Master degree in Clinical Pharmacology obtained at the University of Glasgow (Scotland, UK) fostered, for the first time, his interest in gene therapy and gene silencing. He later received his Doctorate degree from University College Cork (Cork, Ireland) where he studied formulation-based approaches for the delivery of therapeutic oligonucleotides to the central nervous system (CNS). He then joined Prof. Khvorova’s Lab at the RNA Therapeutics institute (UMass Medical School) as a postdoctoral fellow to learn and gain experience in the design and delivery of fully-modified conjugated therapeutic oligonucleotides. During his training,he made significant contributions towards the identification of neuroactive siRNA scaffolds that enable potent and sustained gene silencing in the CNS, without the need for synthetic formulation. In 2016, he received the Milton-Safenowitz Post-Doctoral Fellowship Award from the Amyotrophic Lateral Sclerosis Association (ALSA) to support his research. Bruno is also the recipient of other career awards, including the Dr. Alan M. Gewirtz Memorial Scholarship Award (Oligonucleotide Therapeutics Society), the STAT 2019 Wunderkind Award (STAT News) and a Silver Medal from the Polytechnic Institute in Lisbon (Instituto Politécnico de Lisboa). While a postdoc, Bruno acted as an Adjunct Professor at the Lisbon School of Health Technology (ESTESL, Portugal) and Guest Lecturer at the Instituto de Engenharia de Lisboa (ISEL), teaching pharmacy-related subjects and advanced therapeutics.

At Atalanta Therapeutics, a CNS-focused RNAi therapeutics company, Bruno leads platform development and innovation efforts, and is the lead in vivopharmacologist for several internal and partnership programs.

AFFILIATIONS:

Associate Director, Atalanta Therapeutics
Director, OTS Board (since 2022)
External Scientific Advisor at the Health & Technology Research Centre (Escola Superior de Tecnologia da Saúde de Lisboa (ESTESL), Lisbon, Portugal)
Editor (Molecular Therapy Nucleic Acids; Nucleic Acid Therapeutics; Saúde&Tecnologia)

Aurelie Goyenvalle, PhD

UMR1179-University of Versailles, France

PERSONAL STATEMENT/MOTIVATION:

My journey with the Oligonucleotide Therapeutics Society began in 2011 when I attended my first OTS meeting in Copenhagen. Over the years, I have witnessed the OTS community’s growth and its commitment to fostering exceptional science, encouraging collaboration between academia and industry, and nurturing a vibrant exchange between scientists at different career stages.I am proud to have received two prestigious awards from the society: the Dr. Alan M. Gewirtz Memorial Scholarship Award in 2011 and the Mary Ann Liebert Publishers, Inc. Young Investigator Award in 2016. These serve as a reminder of my commitment to advancing the field of oligonucleotide therapeutics and the kind recognition and support I have received from the OTS community.

One of the most fulfilling aspects of my involvement in OTS has been my role in the annual meetings. I had the privilege to be a part of the organizing committee for the past two annual meetings in 2022 and 2023. These experiences have allowed me to contribute to the annual meetings’ success, and I have found great reward in this collaborative endeavor.

Beyond my own research projects, I have actively participated in building networks that promote the application of oligonucleotide technology in Europe. As a founding member and management committee member of the European networks “Networking towards clinical application of antisense-mediated exon skipping” (COST Action BM1207) and “Delivery of Antisense RNA therapeutics” (COST Action CA17103), I have played a pivotal role in connecting European experts in the field. In the latter network, I led the working group on Safety and Toxicology, further underscoring my dedication to sharing knowledge and promoting the safe and effective application of oligonucleotide therapeutics.It would be a great pleasure to serve on the OTS board of directors to contribute to the society’s mission, particularly its commitment to education and inclusivity, and to work with the dedicated individuals who share a passion for advancing oligonucleotide therapeutics.

BIOGRAPHY

Aurélie Goyenvalle obtained her PhD in Virology at the University of Paris VII in 2006 from her work at Genethon in France, where she developed an exon-skipping gene therapy strategy for Duchenne muscular dystrophy (DMD) using AAV vectors encoding U7snRNA constructs carrying antisense sequences. She then joined Pr. Kay Davies’ laboratory at the University of Oxford as a post-doctoral scientist supported by an EMBO fellowship. During her postdoc in Oxford and subsequent position at the Medical Research Council (MRC),she developed various splicing modulation approaches for neuromuscular and neurodegenerative diseases, including peptide-conjugated antisense oligonucleotides.

In 2011, she joined the Institute of Myology in Paristo investigate splice switching approaches using novel antisense oligonucleotides and in 2012, she was awarded a Chair of Excellence program to establish her own group at the University of Versailles to develop novel RNA based technology for the treatment of neuromuscular diseases. She notably demonstrated the therapeutic potential of a novel class of AONs made of tricyclo-DNA (tcDNA), which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration.

In 2015, Aurélie was appointed a permanent research scientist at INSERM and in 2020 Director of Research to pursue this line of research. Aurélie Goyenvalle is currently directing the laboratory Biotherapies for neuromuscular diseases at the University of Versailles (UMR 1179) focused on gene and antisense therapies for the treatment of neuromuscular disorders.

AFFILIATIONS:

Member of the Oligonucleotide Therapeutics Society
European editor of Nucleic Acid Therapeutics
Editorial board of Molecular Therapy Nucleic Acids
Reviews editor of Gene Therapy
Founding member of the Delivery of Antisense RNA therapeutics (DARTER) network
Member of the extended TREAT-NMD Advisory Committee for Therapeutics (TACT)

Shuling Guo, PhD

Vertex Pharmaceuticals

PERSONAL STATEMENT/MOTIVATION:

Since I first joined Isis/Ionis in 2004, the OTS conference has consistently held a special place on my calendar. It has been a forum where I stay connected to the latest scientific breakthroughs in the oligonucleotide therapeutic community and, just as importantly, where I reconnect with longtime colleagues and forge new relationships. Over the years, these gatherings have proven invaluable for sparking collaborations and driving innovation across our field.

Earlier this year, I began an exciting new chapter as Head of Biology and Associate Site Head at Vertex Pharmaceuticals in San Diego, where I lead research initiatives in nucleic acid therapeutics and targeted delivery. While my professional home has changed, my commitment to the OTS mission remains steadfast. I am deeply grateful to the seasoned leaders within OTS who have served as role models and mentors throughout my career. Their guidance has shaped me as both a scientist and a leader, and it now inspires me to give back to the community that has given me so much.

My candidacy for the OTS Board of Directors is backed by a set of qualifications that uniquely position me to contribute effectively:

Diverse Expertise in Oligonucleotide Research – Over the years, I have led drug discovery programs spanning cardiorenal, metabolic, pulmonary, hematological, and rare diseases, alongside core research efforts in non-coding RNA, nonsense-mediated mRNA decay, and autophagy. My experience spans multiple modalities—including single-strand ASO, double-strand siRNA, and CRISPR gene editing—and I have published over 80 scientific articles on oligonucleotide-related topics.
Robust Network Across Academia and Industry – I have built collaborations with academic investigators and pharmaceutical partners, fostering connections across diverse therapeutic areas and enhancing my ability to navigate and contribute meaningfully within the expanding OTS community.
Leadership and Service –
- Mentorship: Guiding postdocs, sponsoring internship programs, and serving on career development panels.
- Conference Organization: Co-organizer for OTS 2025, advisory committee member for TIDES and session organizer/chair for multiple scientific conferences.
- Results-Oriented Leadership & Community Engagement: – A consistent track record of delivering high-quality outcomes through strategic thinking and collaborative execution, including co-chairing the OTS Education and Community Building Committee (including the Journal) to launch impactful initiatives such as free access to the OSWG whitepapers and other programs that foster education and connection within the global oligonucleotide community.

In summary, my transition to Vertex broadens my perspective on how oligonucleotide therapeutics can be advanced across different platforms and organizations. My qualifications, combined with a deep passion for the OTS mission, position me to help the Society grow and thrive. I am eager to leverage my experience to support OTS in shaping the future of oligonucleotide science and therapeutic innovation.

BIOGRAPHY

My name is Shuling Guo. I earned my Ph.D. in Molecular Cancer Biology from Duke University and completed my postdoctoral training at HHMI/UCLA. I began my career in oligonucleotide therapeutics at Ionis Pharmaceuticals in 2008, where I held positions of increasing responsibility and most recently served as Vice President of Drug Discovery.

In 2025, I joined Vertex Pharmaceuticals as Head of Biology and Associate Site Head at the San Diego research site. In this role, I lead research efforts in nucleic acid therapeutics and targeted drug delivery, guiding discovery programs from early biology through candidate selection and fostering collaboration across multidisciplinary teams. My work spans multiple modalities—including antisense oligonucleotides (ASO), small interfering RNA (siRNA), CRISPR gene editing, and small molecules—with a focus on advancing next-generation medicines for patients.

Over the course of my career, I have contributed to the discovery and development of numerous oligonucleotide therapeutics, including compounds that have achieved regulatory approval (e.g., Tegsedi® and Wainua™), or are in various stages of clinical development. My research leadership has also encompassed exploratory areas such as noncoding RNA, autophagy/mitophagy, and nonsense-mediated mRNA decay. To date, I have authored or co-authored over 80 scientific publications, most focused on the mechanisms and therapeutic applications of RNA-targeting technologies.

AFFILIATIONS

Vice President and Associate Site Head, Vertex – San Diego
Member, Oligonucleotide Therapeutics Society (OTS)
Director, BoD OTS
Advisor, TIDES

Gang Han, PhD

Tianjin Medical University

PERSONAL STATEMENT/MOTIVATION:

As a young researcher who has devoted nearly fifteen years to the study of oligonucleotide therapeutics, I am deeply motivated to serve on the OTS Board of Directors. My main goal is to help increase the visibility and participation of scientists from China in the OTS community. Despite China being one of the largest contributors to oligonucleotide research and manufacturing, and also one of the most important potential markets for oligonucleotide-based therapies, awareness and engagement with OTS remain limited. I hope to bridge this gap and foster stronger connections between researchers, industry, and OTS.

Since my PhD studies, my research has focused on developing novel strategies to improve the therapeutic efficacy of oligonucleotides for Duchenne muscular dystrophy. Over the years, I have published related work in Molecular Therapy, EMBO Molecular Medicine, Nucleic Acids Research, and other journals, and have also filed five patents in this field. I plan to continue focusing my career on oligonucleotide therapeutics, a field that I truly love and believe has tremendous potential for transforming human health.

I have maintained close collaborations with research groups in multiple countries, working together on oligonucleotide synthesis, modification, and delivery. These experiences have strengthened my belief that global collaboration is key to innovation in this field.

My connection with OTS began ten years ago, when I attended my first OTS Annual Meeting as a PhD student supported by a travel grant. It was my first international conference, and the experience of sharing my work and meeting inspiring scientists left a lasting impression. Since then, I have attended OTS meetings regularly, presenting my progress almost every year. In 2023, I had the honor to give a talk for the first time, which was a milestone moment for me. Every step of my scientific growth has been closely tied to OTS, and I am truly grateful for the opportunities and inspiration it has provided.

Serving on the Board would be a way for me to give back to the society that has shaped my career. I would like to help OTS expand its global reach, promote communication among young scientists, and strengthen engagement from Asia, especially China. I believe my passion, experience, and long-term dedication to oligonucleotide therapeutics will allow me to make a meaningful contribution to the OTS community.

BIOGRAPHY

I received my PhD degree from Tianjin Medical University in 2016 under the supervision of Professor Haifang Yin. In the same year, I joined the School of Medical Technology at Tianjin Medical University as a lecturer and became an associate professor in 2020. I am currently a member of the Preclinical Evaluation Committee of the Chinese Society of Biotechnology, as well as a member of the International Society for Extracellular Vesicles (ISEV) and the Oligonucleotide Therapeutics Society (OTS).

My research focuses on developing novel strategies to enhance the therapeutic efficacy of antisense oligonucleotide drugs for Duchenne muscular dystrophy (DMD). My work mainly involves two areas. First, I have identified and characterized small-molecule adjuvants such as GF and glycine that significantly improve the activity of phosphorodiamidate morpholino oligomers (PMOs) in DMD models. Mechanistic studies revealed that enhancing mitochondrial function and promoting muscle satellite cell proliferation can increase PMO efficacy in vivo.

Second, I am developing targeted delivery systems for antisense oligonucleotides using peptides, aptamers, antibodies, and exosomes. I discovered the muscle-targeting peptides M12 and BV2, which improve PMO uptake in muscle, and identified several exosome-anchoring molecules, including Exp, EAA, and Nexo3, that enable efficient nucleic acid loading and targeted delivery of exosomes to muscle tissue.

These studies have resulted in publications in Molecular Therapy, EMBO Molecular Medicine, and Nature Communications, and I have filed five related patents. I have also been the principal investigator of several national and regional research grants, including the National Natural Science Foundation of China (Youth and General Programs), the Tianjin Municipal Science Foundation (Youth and General Programs), and key projects from the Tianjin Education Commission.

AFFILIATIONS

Member, Oligonucleotide Therapeutics Society (OTS)
Member, International Society for Extracellular Vesicles (ISEV)
Committee Member, Preclinical Evaluation Committee, Chinese Society of Biotechnology

Reka Agnes Haraszti MD PhD

University Hospital Tübingen

PERSONAL STATEMENT/MOTIVATION:

I am deeply committed to advancing the potential of oligonucleotide therapeutics and fostering education about these therapies among a diverse audience, including students, physicians, patients, and researchers from other fields. As a physician-scientist, I bridge basic research, translational science, and patient care, providing a perspective that integrates the potential of RNA therapeutics with the real-world needs of patients.

I represent groups that are currently underrepresented in the oligonucleotide therapeutics community, including European scientists, physician-scientists actively engaged in both research and patient care, and specialists in immunomodulation and immuno-oncology.

I have actively contributed to the RNA therapy field through scientific leadership, education, and community-building initiatives. I served as session chair for early preclinical research at the OTS Meeting 2025. I am a co-founder and speaker of the RNA therapy division in the Gene and RNA Therapy Center Tübingen. I am organizing the GeneNovate Workshop in Tübingen – an initiative of the German national strategy for gene and cell therapies to educate researchers about innovation and startups in the gene, RNA and cell therapy field. I have developed and taught courses on siRNAs and RNA-based therapeutics at several graduate programs in Tübingen, engaging Master’s and PhD students in the fundamentals and translational aspects of oligonucleotide science.

My scientific training provides a robust foundation for these contributions. I earned a PhD in the Khvorova Lab at UMass Chan, where I developed expertise in chemical biology, pharmacokinetics, and delivery of therapeutic siRNAs, and my subsequent work as a junior group leader in immuno-oncology has deepened my understanding of therapeutic immune modulation. As a physician-scientist, I bring a unique perspective by asking questions about clinical application and patient impact that might be overlooked by researchers not involved in patient care.

I am committed to fostering a collaborative, inclusive, and forward-looking community, ensuring that OTS continues to lead in education, innovation, and the responsible development of RNA-based therapies worldwide.

BIOGRAPHY

I completed my medical studies at Semmelweis University, Hungary (2006–2013), with academic stays at the University of Heidelberg, Germany, and the UC Davis School of Medicine, USA. As a student research assistant at Semmelweis I studied the effects of circadian preference on human health. In Heidelberg (2011–2012), I focused on the circadian regulation of mRNA polyadenylation: how constantly expressed mRNAs generate circadian protein profiles.

I joined the Morningside Graduate School of Biomedical Sciences at UMass Chan Medical School in 2013, where I completed my PhD in the Khvorova Laboratory in 2018. My doctoral research centered on the chemical biology and PK/PD of therapeutic siRNAs, as well as the reverse engineering of extracellular vesicles (EVs) for siRNA delivery. One of my major contributions was demonstrating that 5′-vinylphosphonate significantly improves siRNA accumulation in extrahepatic organs. I adapted and optimized a PNA hybridization assay for PK measurements of therapeutic siRNAs and extensively characterized the chemical mechanisms underlying siRNA loading into EVs.

I joined the University Hospital Tübingen, Department of Hematology, Oncology, Clinical Immunology, and Rheumatology in 2018 as a clinical resident. Witnessing the clinical need inspired my current research focus: developing siRNA-based strategies for transient, targeted immune modulation in oncology. In 2021, I established my junior research group, funded by the German Cancer Aid, dedicated to enabling RNA-mediated modulation of therapeutic immune responses, with a particular emphasis on optimizing on-tumor selectivity.

AFFILIATIONS

Gene and RNA Therapy Center, Faculty of Medicine, University of Tübingen

Krystal Johnson, Ph.D.

Alnylam Pharmaceuticals

PERSONAL STATEMENT/MOTIVATION:

My first OTS meeting was unlike any other professional gathering I had ever attended. It felt like walking into a family reunion—where people from competing companies and labs greeted one another with hugs and genuine warmth. The talks were inspiring, the conversations were open, and the spirit of collaboration was palpable. I knew immediately that I had found my scientific home.

During my two years on the OTS Board of Directors as a trainee representative from academia, I had the privilege of contributing to and learning from this remarkable community. I served on the Organizing Committee as co-chair of the NextGen Early Career Session in Barcelona (2023), where we created a platform for rising scientists to share their work. While I was co-chair of the Website Committee, we helped launch an initiative to improve information exchange among industry, academia, and regulatory authorities—culminating in free OTS member access to critical white papers from the Oligonucleotide Safety Working Group (OSWG). These papers provide consensus guidance on oligonucleotide safety assessment, helping to advance the successful development and approval of oligonucleotide-based therapeutics.

In 2024, I also created the first OTS Video Time Capsule—capturing the energy and innovation of our field by asking attendees one question: “What are you most excited about for the future of oligo therapies?” This project invited participation from every level of our community to be part of OTS history, fostering inclusion while documenting the evolving story of our science and vision for the future.

Now, as an industry professional, it would be an honor to once again serve on the OTS Board of Directors. I am eager to bring my creativity, energy, and collaborative spirit to support our society’s mission—advancing the frontiers of oligonucleotide science, promoting the open exchange of ideas, and organizing our annual family reunion that continues to inspire and unite us all.

BIOGRAPHY

I joined Alnylam Pharmaceuticals in 2024 where I currently work in the RNA Biology department. I was recently invited to speak about my transition to Alnylam on a panel at the first OTS Career Event at the 2025 OTS meeting in Budapest. I served as chair of the FEBS Student Session at the 5th International Short and Long Noncoding RNA conference in 2024. I was first recognized by OTS with the NextGen Best Talk Award at the 2022 Annual Meeting in Phoenix, and then, I was awarded the Alan M. Gewirtz Memorial Graduate Student Scholarship at the 2023 OTS meeting in Barcelona. I was elected to the OTS Board of Directors as a Trainee Representative in 2023 where I served as co-chair of the OTS Website Committee.

I earned my Ph.D. in Biochemistry from David Corey’s Lab at UT Southwestern Medical Center (UTSW). I was awarded a NIGMS F31 Diversity Fellowship for my proposed thesis work that deepened our understanding of nuclear localization of Argonaute proteins and microRNA regulation in colon cancer model systems. I received the Alfred Gilman Memorial Student Award for Excellence in Research from UTSW’s Department of Pharmacology, and I recently returned to my alma mater to speak on a panel as an industry representative in 2024 for UTSW’s Biotechnology Club.

Anastasia Khvorova, PhD

UMASS Chan Medical School / RTI

PERSONAL STATEMENT/MOTIVATION:

I have dedicated my scientific career to understanding the fundamental principles behind the development of RNA and siRNA, envisioning them as the next class of therapeutics with the potential to revolutionize how we practice medicine.

I served as an OTS board member from 2010 to 2018, contributing to the definition of the OTS as it stands today. I believe that our field is on the verge of transformative change, and the role the OTS plays is crucial. My unique blend of experiences as an academic PI, pharma executive, co-founder of a small biotech, and adviser to numerous young scientists positions me ideally to guide society into the next chapter.

I am a firm believer in the importance of academia-industry integration and collaboration for our future progress. I hope to serve as a liaison, offering a clear historical perspective and an intimate understanding of the needs and challenges in both worlds.

BIOGRAPHY

Anastasia Khvorova, Ph.D., has over 20 years of experience developing oligonucleotide technology and therapeutics. She is the Remondi Family Chair in Biomedical Sciences and professor at the RNA Therapeutics Institute and Program in Molecular Medicine at the University of Massachusetts (UMass) Chan Medical School. Her lab combines hardcore organic and oligonucleotide chemists with RNA biologists and pharmacologists to develop novel approaches and solutions to understanding natural and therapeutic RNA trafficking and delivery. Dr. Khvorova founded the UMass Nucleic Acid Chemistry Center, the only nonprofit facility in North America with expertise and capacity for complex RNA molecule synthesis at scale and numbers. Dr Khvorova’s laboratory has trained dozens of next-generation scientists expanding the progress of oligonucleotide therapeutics in North America and worldwide.

Before UMass Chan Medical School, Dr. Khvorova spent 12 years in industry in different executive positions. She has co-founded several biotechnology companies.

Dr. Khvorova is a National Academy of Inventors member with more than 150 patents and 300 patent applications. She has authored more than 100 peer-reviewed publications, including seminal articles in Cell, Nature, and Nature Biotechnology, defining the field of RNAi drug design and development. She has served as one of the early directors of the Oligonucleotide Therapeutics Society and as a Director-at-Large, and Scientific & Research Council Chair of the American Society of Gene and Cell Therapy.

AFFILIATIONS:

ASGCTDirector at large(2020-2023)
Editor (NAR, MTNA, NAT)
SAB (Atalanta, Evox, Comanche, Alltrna, Prime Medicine, Aldena)

Audrius Kilikevicius, PhD

Eli Lilly and Company

PERSONAL STATEMENT/MOTIVATION:

Since joining OTS in 2018, I have witnessed remarkable growth in membership, the quality of meetings, and the society’s influence within the nucleic-acid therapeutics community. My primary motivation for serving on the Board of Directors is to build on the excellent work accomplished by previous board members, with particular emphasis on fostering inclusion as we pursue the OTS mission. Given the evolving landscape of scientific funding and global affairs, it is essential to remain attentive and respond thoughtfully to ensure the continued diversity and integrity of the OTS community.

My transition from a lengthy academic career to industry, along with my shift into the nucleic acids field from a different discipline, gives me a distinct perspective and strategic thinking to offer the board. I am a goal-oriented colleague who can adapt well to the rapidly changing needs of the field and society. I maintain a reputation for executing my responsibilities at a high standard of excellence and enjoy going above and beyond.

At the 21st Annual Meeting of OTS (2025, Budapest), I had the honor of serving on the Scientific Organizing Committee and co-chairing the Oligonucleotide Therapeutics Educational Workshop. Two years prior, I contributed to the organization and leadership of the RNA Biology session at the 2nd Midwest Symposium on Oligonucleotide Therapeutics, which was partially supported by the OTS Local Delivery Grant.

BIOGRAPHY

I have spent most of my professional career in academia serving as a research scientist with some teaching roles as an associate professor for molecular biology and genetics courses as well as biochemistry labs. The foundation of my expertise in nucleic acids research and therapeutics came from training as a post-doc in David R. Corey’s lab at UT Southwestern. Currently, I am a Research Advisor for RNA Preclinical Pharmacology at Eli Lilly and Company.

My professional background covers essential areas for developing oligonucleotide therapeutics. My career in this field began with identifying and selecting therapeutic targets, where I used quantitative genomic methods to find genetic factors affecting polygenic traits of skeletal muscle, including structure, function, and metabolism. Later, I dove in endogenous mechanism of RNAi, studying how RNAi functions in both the cytoplasm and nucleus under various stress conditions—insights that could help improve RNAi-based drugs. My experience further extends to preclinical development of diverse ASO modalities – including cleavage, blocking, and RNA editing for rare diseases, as well as the optimization of extrahepatic delivery shuttles.

AFFILIATION

Guest Editor for NAR Molecular Medicine (Nucleic Acids Therapeutics focused special collection).

Marc Lemaitre, Dr. Sc.

ML_Consult LLC

PERSONAL STATEMENT/MOTIVATION:

I would like to serve in the OTS Board of Directors, to bring to the Board more than 30 years of experience in oligo synthesis, development (from N=1 to large applications), CMC starting materials, regulatory, discussions with authorities as well as mentorship for young colleagues. Besides CMC activity I like preparing and giving trainings on oligonucleotide technologies and development I have been CEO and Vorstand of Girindus America Inc and Girindus AG [now Avecia Cincinnati), as well and Chairman of the board of Link Technologies (now part of Axolabsl). I could use my networking in the field to continue helping the organization team to improve fundraising also to help supporting fellowship budget. I’ve been in the board of OTS between 2015 and 2021. As a chemist by training, I try to be a problem solver and I like to think that as a scientist I would like to bring leadership through science and knowledge.

BIOGRAPHY

After a graduate degree in organic synthetic chemistry at the University of Liege [Belgium] I got a Dr. Sc. in Biochemistry from the same university in 1985. I then started a post-doc at the University of Montpellier [FR] where I made my first oligonucleotide the same year. I published my first paper on ASOs degradation of mRNA in 1987 [PNAS, 84, 648-652] and have now published over 50 papers in peer-reviewed journals. Since 1985, my entire professional endeavor was about oligos with a 100% focus on therapeutic oligos since 2009. I collaborated to the development of many oligos including 3 approved ones with more than 35 companies from Fortune 500 companies to young start-ups and mid-sized companies. I’ve experience in almost all aspects of oligonucleotide therapeutics development and CMC from supply chain, manufacturing, analytical methods, regulatory, QA.

AFFILIATIONS:

Member of the USP expert committee Bio 1 since 2020, second term till 2030
Voluntary Member of the OSWG
Member of N=1 collaborative
Member of the ACS
Member of IS3NA

Shabnam Mohandessi

CordenPharma International

PERSONAL STATEMENT/MOTIVATION:

As an active member of the Oligonucleotide Therapeutics Society and a long-standing advocate for advancing oligonucleotide and peptide-based drug discovery, I am honored to express my interest in serving on the OTS Board of Directors. My professional journey—spanning leadership roles in business development and strategic partnerships across global CDMO and CRO organizations—has been dedicated to building bridges between cutting-edge science and real-world therapeutic impact. I am eager to contribute this perspective to further the Society’s mission of fostering innovation, collaboration, and education in the field of oligonucleotide therapeutics.

Over the past decade, I have led North American business development teams specializing in oligonucleotide and peptide therapeutics, serving as Director of Business Development for TIDES Discovery at WuXi AppTec for nearly four years. In this capacity, I cultivated strategic alliances with emerging biotech, mid-size biotech, and big pharma key opinion leaders—driving early discovery partnerships and accelerating translation from concept to clinic. I also had the privilege of mentoring four business development professionals as they transitioned from postdoctoral research to commercial leadership.

In my current role as Director of Business Development at CordenPharma, I lead strategic initiatives across oligonucleotide, lipid, LNP, peptide, and small-molecule platforms. This position requires not only commercial and scientific acumen but also a deep commitment to ethical collaboration, financial stewardship, and cross-functional leadership—qualities that align closely with the governance and oversight responsibilities of the OTS Board.

Beyond my professional leadership, I am deeply committed to mentorship and community development. Having managed and guided multiple direct reports throughout my career, I take pride in mentoring emerging professionals and scientists entering the oligonucleotide field. I would welcome the opportunity to contribute to OTS’s mentorship programs and professional development initiatives, sharing practical industry perspectives to help the next generation of leaders thrive.

In addition, my extensive network across U.S. oligonucleotide and peptide therapeutic companies—particularly within the Boston/Cambridge biotech cluster—positions me well to support OTS initiatives such as identifying distinguished speakers, fostering cross-sector collaboration, and expanding engagement with both established and emerging industry players. I believe this connectivity can help strengthen OTS’s presence and impact in one of the world’s most vibrant innovation hubs.

My vision for serving on the OTS Board centers on fostering collaboration between academia and industry, promoting transparent governance and accountability, and elevating OTS’s global visibility as the leading voice in oligonucleotide therapeutics. With a deep respect for the Society’s mission and a commitment to advancing its goals, I am eager to contribute my leadership experience, strategic insight, mentorship, and professional network to the continued success of OTS.

Sullenger Head Shot copy – Bruce Sullenger

Bruce A. Sullenger, PhD

Duke University

PERSONAL STATEMENT/MOTIVATION:

Since its inception, the Oligonucleotide Therapeutics Society (OTS) has been the leading scientific society promoting the development of oligonucleotide-based therapeutics. Since joining the OTS in 2005, I have witnessed how our field has grown from a concept that many thought was an impractical if not impossible approach to combat diseases to a flourishing field leading the biomedical worlds response to many unmet medical needs ranging from treating rare genetic disorders to addressing a global pandemic and common human illnesses such as cardiovascular disease, cancer and stroke.

I have served on the OTS Board of Directors from 2007-2014 and 2022-2023 and was the Treasurer of the Society from 2008-2010. I have also served as the Co-Editor-in-Chief for OTS’s journal Nucleic Acid Therapeutics from 2011-present, a time span which OTS has continuously grown and moved to the forefront of translational medicine. In addition, I have served on the Board of Directors for the American Society of Gene and Cell Therapy as well as multiple sub-committees including heading the ASGCT committee on oligonucleotide therapeutics. I have been an organizer of multiple aptamer meetings, the OTS Meeting in Naples and was a founding organizer of the Cold Spring Harbor Meeting on RNA Therapeutics. Finally, I was asked to testify about the importance of biomedical research including the use of oligonucleotide therapeutics by Senators Lamar Alexander and Patty Murray before the United States Senate in 2015 and have been a founder of five oligonucleotide-based biotechnology companies. Thus, I believe that I bring considerable experience to the OTS Board about translation of oligonucleotide research from the academic laboratory to the clinical and commercial sectors and also working with governments and our elected leaders on the importance of biomedical research for society. Therefore, I seek your support for election to the OTS Board so that I can serve our Society as ever more oligonucleotide therapeutics progress from discovery to the clinic and to the market and thereby ultimately positively impact our world community.

BIOGRAPHY

I am a translational researcher who has been at Duke University for 30 years working on the discovery and development of RNA/oligonucleotide therapeutics. I obtained my PhD from Weill Cornell University working at Sloan-Kettering Cancer Institute and postdoctoral training in RNA biochemistry with Tom Cech at the University of Colorado. My research program focuses upon the generation and development of nucleic acid therapeutics for a variety of medical applications. We have worked on 1.) translational medicine for treating thrombosis in the acute care settings of cardiovascular disease and stroke and 2.) therapeutic targeting of malignancies with RNA aptamer-drug conjugates. More recently my research group has also worked upon developing the use of RNA and DNA-containing DAMP/PAMP inhibitors to limit pathological inflammation and the use of combinatorial chemistry approaches to generate novel guide RNAs for improved CRISPR gene editing. Three different RNA/oligo-based drugs discovered/invented in my laboratory have been translated into the clinic an accomplishment recognized by the awarding of the 2015 ASGCT Outstanding Achievement award and election into the National Academy of Inventors (2018) and the AAAS (2014). In addition, I have served as the Founding Director of the Duke Translational Research Institute (DTRI; now Duke CTSI) as well as the Associate Director for Translational Research for the Duke Cancer Institute. Finally, I am a scientific founder of five biotechnology companies, two of which completed IPOs and one currently enrolling patients in a large, international phase 2b clinical study evaluating a novel thrombolytic RNA aptamer created in my lab for treatment of ischemic stroke. Thus, I have extensive experience building research teams spanning the academic-private sectors to move important oligo/RNA discoveries and inventions from the laboratory to the bedside to improve patient care and health.

AFFILIATIONS:

Board of Directors, AZURNA Therapeutics Inc Scientific Advisory Board, Rznomics Inc Scientific Advisory Board, SomaLogic Inc (acquired by Illumina)

Hélène Tran, PhD

Servier

PERSONAL STATEMENT/MOTIVATION:

I am deeply honored to express my interest in serving on the OTS Board of Directors. My journey in RNA science began with a PhD in France, where I studied RNA metabolism dysfunction in myotonic dystrophy. I then pursued with a post doctoral research experience at UMASS Medical School, on RNA repeat expansion in the pathogenesis of amyotrophic lateral sclerosis (ALS). During this time, I contributed to the development of potential antisense oligonucleotide (ASO) therapeutics for this devastating rare neurological disorder. These experiences solidified my commitment to advancing RNA-targeted therapies and shaped my belief in the power of cross-disciplinary collaboration, both within academia and in partnership with biotechs.

I then joined Servier, a mid-sized pharmaceutical company based in Paris-Saclay; France, where I was fortunate to be given the opportunity to build from the ground up a team dedicated to oligonucleotide therapeutics. What began as a small initiative has steadily grown into a fully cross-functional team, covering the entire spectrum of ASO discovery, from target identification to clinical candidate nomination. This experience has reinforced my conviction that scientific innovation flourishes when diverse expertise is brought together around a shared vision.

What I hope to bring to the OTS Board is a combination of scientific depth, strategic thinking, and a strong commitment to community building. In my current role, I must understand, communicate, and embark a wide range of stakeholders, from scientists to regulatory, business, legal, patient advocacy and beyond. This requires the ability to interpret complex data, translate it into clear and compelling narratives, and align diverse perspectives toward a common goal. Leading multiple projects across disciplines also demands strong organizational skills and a clear sense of purpose.

Beyond my professional responsibilities, I am actively engaged in shaping the future of oligonucleotide science as a therapeutic modality of choice for rare diseases and ultra-rare neurological disorders. I am committed to increasing visibility and momentum for this field globally but with a focus in Europe, and France. I currently participate in the N=1 Collaborative non clinical task force which brings together international scientists dedicated to advance the field of ultra rare diseases. I serve on the board of ERDERA (European Rare Diseases Research Alliance), a public private partnerhip aiming to strengthen Europe’s position as a global leader in rare disease , and am co-founding the French Oligo Association, an initiative aimed at uniting national stakeholders and advocating for governmental investment in
oligonucleotide research and development.

I view the OTS not only as a scientific society but as a platform to educate, connect, and empower the next generation of innovators. I am particularly committed to training young scientists to understand the fundamentals of oligonucleotide therapeutics to better tackle the current challenges.

Serving on the OTS Board would be a privilege and an opportunity to give back to a global community that has already given me so much. I am eager to bring my experience, energy, and collaborative spirit to help shape the future of our field

BIOGRAPHY

Hélène Tran is Research Director in the Neurology Therapeutic Area at the Servier Research & Development Institute in Paris-Saclay, where she leads efforts in antisense oligonucleotide (ASO) therapeutics development. Her team is dedicated to advancing ASO therapies for brain disorders addressing key technological challenges related to central nervous system delivery, safety and stability.

Prior to joining Servier in late 2018, Hélène held an academic appointment as Instructor at UMASS Medical School, Worcester, USA in the laboratory of Professor Robert Brown. Her research focused on (1) investigating the role of RNA repeat expansions in the pathogenesis of C9ORF72-related amyotrophic lateral sclerosis (ALS) using a diverse set of disease models ranging from flies to patient derived cells and (2) advancing ASO therapeutic approaches in collaboration with the Watts lab at the RNA Therapeutic Institute and the team of Wave Life Sciences. Hélène began her scientific career at the University of Lille, France, where she completed her doctoral studies investigating the contribution of repeat RNA to neuronal toxicity in myotonic dystrophy.

Her contributions to the field have been recognized with several prestigious awards, including the 2024 Mary Ann Liebert Publishers, Inc. Young Investigator Award from the Oligonucleotide Therapeutics Society (OTS); the inaugural Servier Group Award from the Servier Executive Committee for her team’s pioneering work on a potential ASO therapy for a rare neurodevelopmental disorder, and the Milton Safenowitz Postdoctoral Fellowship Award from the ALS Association.

Hélène serves on the advisory board of ERDERA (European Rare Disease Research Alliance) supporting efforts to accelerate research in prevention, diagnosis and treatment for rare disease patients. She is also an active member of the N1Collaborative, a global initiative committed to advancing individualized medicine and ensuring safe and rapid access to patients worldwide.

AFFILIATIONS:

Research Director, Neurology therapeutic area, antisense oligonucleotide therapeutics head
Erdera
Nof one collaborative
Effect (A European Training Program to Foster the Full Therapeutic Potential of Antisense
Technology across Tissues)

Jonathan Watts, PhD

RNA Therapeutics Institute,
UMass Chan Medical School

PERSONAL STATEMENT/MOTIVATION:

I have been grateful for the OTS community since I was a trainee, and strongly believe in the vision of the OTS to bring academic and industry scientists together in a science-centric forum to support the development of future leaders and advance the quality and depth of science in our field.

I was previously on the OTS Board of Directors from 2015-2021, serving as Secretary through much of that time. I was a member of the organizing committees for the 2014, 2015, 2017 and 2020 annual meetings, including serving as Chair for the 2020 meeting.

During that first stretch serving on the OTS Board, I helped establish the Trainee Rep program, in which students and postdocs serve on the Board for two-year terms. This initiative has proven to be good for trainees, giving them key experience and access to leaders in the field—but even more clearly, it has been great for the OTS since our trainee reps have contributed so much to the mission of the society. In particular, they have really helped us raise our game in supporting other trainees, a key part of our mission. I have also contributed to the OTS work on rare disease, first through the Rare Disease Task Force and then in partnership with the N=1 Collaborative, a group established to share data and best practices to ensure that more single-patient drugs reach patients quickly, safely and affordably.

I am interested in serving on the Board again as the OTS is in a season that brings both challenge and opportunity: Oligonucleotide medicines have now proven themselves to be commercially successful, and each year brings new approvals. As such, a major challenge is to keep and build the collegial, almost familial feel of the OTS community and conference, while our field expands and we have a larger number of trainees and researchers to serve. If we are to achieve our potential as a society and add the most value to the field, we will need to empower even more of our members—both established scientists and trainees—to share their gifts and support each other through mentoring initiatives and through creating a culture that encourages openly sharing data, including both successes and failures, at our meetings.

BIOGRAPHY

I completed my PhD in nucleic acids chemistry from McGill University (working with Masad Damha) and carried out postdoctoral work in biochemistry and pharmacology at UT Southwestern Medical Center (working with David Corey). In 2012, I moved to the UK to start my independent group at the University of Southampton. In 2015, my group and I moved to the RNA Therapeutics Institute of UMass Chan Medical School (Worcester, Massachusetts, USA) where I am Professor.

Work in the Watts lab is centered on the application of oligonucleotide chemistry to improve platform technologies for nucleic acid therapeutics. Major areas of interest for us include the optimization of oligonucleotide therapeutics for use in the brain and lung, and increasing the efficacy and versatility of genome editing for therapeutic use. We also work on specific disease applications, and we have contributed to the development of three oligonucleotide drugs that have reached ALS patients on a compassionate use basis, plus additional licensed technologies and therapies in more traditional preclinical development. We have published over 90 papers which have been cited over 6000 times. I also co-edited the textbook Nucleic Acids in Chemistry and Biology, 4th Edition (Blackburn, Egli, Gait and Watts, 2022).

AFFILIATIONS:

I serve on the editorial boards of Nucleic Acid Therapeutics, Nucleic Acids Research and Molecular Therapy: Nucleic Acids. I also serve as part of the N=1 Collaborative (n1collaborative.org) and regularly advise biotech companies developing genetically targeted therapies. I am a co-founder of Nucyrna and member of Scientific Advisory Boards including PepGen and EnPlusOne. I am also currently on the Oligonucleotide and RNAi Therapeutics Committee of the ASGCT.

Marie Wikström Lindhom, PhD

Silence Therapeutics GmbH

About Marie Wikström Lindhom, PhD

PERSONAL STATEMENT/MOTIVATION:

It has been a pleasure to serve two periods on the OTS BoD; I was on the team starting the mentoring program, and also served as a mentor myself – it is very rewarding to share my perspective as a person with both industry and academic experience, good and bad, and if this can help younger people in our field to find their way that is worth a lot. During my second period I have co-chaired the outreach and education subcommittee, where among other things we have set up access to the different oligonucleotide safety working group whitepapers and industry recommendations as open access through the OTS home page. I have also happily contributed in other ways such as reviewing travel grant applications, posters for poster awards, abstracts for the annual meeting, and I have been chairing and co-chairing sessions at three annual meetings. Our field is evolving very rapidly across the globe and being part of the OTS BoD means I can contribute to different ways of “spreading the word”. I would be honored to continue to do this for another period, and I hope we can continue to work internationally as well as support local efforts to further connect industry and academic scientists working on different oligo therapeutics.

BIOGRAPHY:

I started out with a BSc in chemistry, a PhD in medicinal chemistry, and post-doctoral research projects in Berkeley and London before I returned to Sweden to be part of a research team working on a vaccine against atherosclerosis and eventually qualified as Associate Professor in Experimental Cardiovascular Medicine. I wanted to work on more applied science and started at Santaris Pharma, a biotechnology company developing RNA-targeting drugs where I worked on LNA oligonucleotide drug discovery: molecule design, in vitro screening, in vivo activity and metabolism studies, and finally as lipid metabolism expert when the apoB, PCSK9, and miR-122 targeting programs reached clinical development. After Santaris was acquired by Roche I was appointed Expert Scientist in Discovery Technology and Head of Targeted Delivery of oligonucleotide conjugates. In that role I headed interdisciplinary teams across Roche sites, led collaborations with academia, and frequently participated in evaluations of outside opportunities for targeted delivery.

December 2017 I returned to biotech when I was recruited to Silence Therapeutics where I am leading a skilled team focusing on fine-tuning design of GalNAc-conjugated siRNA and exploring extra-hepatocyte siRNA targeting, and I have been the CSO of Silence since December 2023. All in all this means that I started out as an academic specialized in cardiometabolic diseases and then got a chance to work on different oligonucleotide therapeutics intended for these and other patients in need.

AFFILATIONS::

Co-founder and co-owner of Maclawili Hb, Sweden.

Timothy Yu, MD, PhD

Harvard/Boston Children’s Hospital

About Timothy Yu, MD, PhD

Division of Genetics & Genomics and Department of Neurology,
Boston Children’s Hospital

PERSONAL STATEMENT/MOTIVATION:

Millions of children worldwide are affected by over 8,000 different genetic diseases, many of which are severely debilitating and/or fatal. I have had the privilege of alongside many in the OTS community since 2017 to use oligonucleotide-based therapies to bring to bear genetic interventions for even the smallest of these patient populations.

BIOGRAPHY:

Dr. Yu is a neurogeneticist at Boston Children’s Hospital, Harvard Medical School, and the Broad Institute. He leads a cross-disciplinary research group that works in genomics, neurobiology, and bioinformatics to understand and treat childhood disorders of brain development. Beginning with a young girl with Batten disease (a progressive, fatal, neurodegenerative disorder), his team demonstrated it was possible to go from genetic diagnosis to development, testing, and deployment of a novel investigational drug, using an intrathecally delivered, patient-customized oligonucleotide, in a year’s time. His laboratory continues to work with clinicians, scientists, industry, policymakers, rare disease advocates, and the FDA to streamline this path as an option for children suffering from rare, orphan diseases, and currently has five clinical therapeutic programs in the IND or pre-IND phase for individuals with ultra-rare, orphan neurogenetic conditions.

AFFILIATIONS:

Staff Physician, Division of Genetics & Genomics and Department of Neurology, Boston Children’s Hospital
Associate Professor, Harvard Medical School
Associate Member, Broad Institute of MIT and Harvard

Current OTS members are encouraged to participate in the BOD election which will be open through Friday, December 12, 2025. All current members have been emailed a link to the ballot which can also be reached here.

If you did not receive the email or have questions about the status of your membership, contact us at info@oligotherapeutics.org.

2025 BOD Election – Candidate Profiles

Annemieke Aartsma-Rus, PhD

Marc Abrams, PhD

Julia Alterman, PhD

Sritama Bose, PhD

Ronald Buijsen, PhD

Michael J Byrne, PhD

Masad J. Damha, PhD, FCIC

Sarah L DeVos, PhD

MariJean Eggen, PhD

Hassan H. Fakih, PhD

Alex Garanto, PhD

Felix Gnerlich, PhD

Bruno Miguel da Cruz Godinho, PhD

Aurelie Goyenvalle, PhD

Shuling Guo, PhD

Gang Han, PhD

Reka Agnes Haraszti MD PhD

Krystal Johnson, Ph.D.

Anastasia Khvorova, PhD

Audrius Kilikevicius, PhD

Marc Lemaitre, Dr. Sc.

Shabnam Mohandessi

Bruce A. Sullenger, PhD

Hélène Tran, PhD

Jonathan Watts, PhD

Marie Wikström Lindhom, PhD

Timothy Yu, MD, PhD

Subscribe to our Newsletter

Resources

Stay Connected

Search

2025 BOD Election – Candidate Profiles

Annemieke Aartsma-Rus, PhD

Marc Abrams, PhD

Julia Alterman, PhD

Sritama Bose, PhD

Ronald Buijsen, PhD

Michael J Byrne, PhD

Masad J. Damha, PhD, FCIC

Sarah L DeVos, PhD

MariJean Eggen, PhD

Hassan H. Fakih, PhD

Alex Garanto, PhD

Felix Gnerlich, PhD

Bruno Miguel da Cruz Godinho, PhD

Aurelie Goyenvalle, PhD

Shuling Guo, PhD

Gang Han, PhD

Reka Agnes Haraszti MD PhD

Krystal Johnson, Ph.D.

Anastasia Khvorova, PhD

Audrius Kilikevicius, PhD

Marc Lemaitre, Dr. Sc.

Shabnam Mohandessi

Bruce A. Sullenger, PhD

Hélène Tran, PhD

Jonathan Watts, PhD

Marie Wikström Lindhom, PhD

Timothy Yu, MD, PhD

Subscribe to our Newsletter

Resources

Stay Connected

Search

Login to your account

Reset Password

Signup to your Account

Answers

Job Alerts

Account Activation