Patient Advocacy and Therapeutic Development for Huntington’s Disease
Time: 11am - 12pm EST Date: August 11, 2022 Description: Dan O'Reilly, PhD, a former OTS Trainee BoD member, focuses his research on developing siRNA therapeutics for Huntington’s Disease. Join us as Dr. O'Reilly discusses patient advocacy and Huntington’s Disease research. ...
FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients
In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in the pipeline. As oligonucleotide therapeutics continue to develop as an emerging and promising treatment for ...
OTS President’s Pick Paper – July 2022
By: David Corey Establishing an environment in which rigorous scientific inquiry is practiced: A personal journey. Stanley Crooke Nucleic Acids Research, gkac526, https://doi.org/10.1093/nar/gkac526 Published: 08 July 2022. https://academic.oup.com/nar/advance-article/doi/10.1093/nar/gkac526/6633881?login=true The Story of Ionis Pharmaceuticals For those new to the field, Ionis Pharmaceuticals has always been ...
A New Conjugated siRNA provides broad delivery in the CNS, Lung, and Eye
Ever since the groundbreaking discovery of RNA interference and its role as a gene-silencing mechanism in mammalian cells, an ongoing process of discovery has been underway to harness its potential in treating disease. A key mechanism that induces gene-silencing in the RNA pathway is ...
Transitioning from Trainee to Faculty – A conversation with Prof. Bethany Powell Gray
Time: 11am - 12pm EST Date: July 14, 2022 Description: Join us for an open discussion with Dr. Bethany Powell Gray, who is an Assisstant Professor at the John Hopkins University School of Medicine, where we will talk about the postdoc to faculty ...
Positive Results from Tofersen VALOR Trial and OLE Integrated Data
People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective treatment that could delay the onset of symptoms or extend their lifespan. However, as a result of recent advances in science and medicine, multiple oligonucleotide therapeutics are being developed ...
Trainee Spotlight Series: Yan Li and Roberto El Khoury
Title: Trainee Spotlight Series: Yan Li and Roberto El Khoury Time: 11am EST Date: July 7, 2022 Description: This Trainee Spotlight features Yan Li, a Ph.D. student at the University of California, Los Angeles, and Roberto El Khoury a Ph.D. candidate at McGill ...
OTS President’s Paper Pick – June 2022
Successful nucleic acid therapeutic projects have one thing in common - rigor. Lack of controls has many consequences ranging from failed companies, seminars that waste the time of all involved, and lost hopes of patients. In this “President’s Pick”, Past President Annemieke Aartsma-Rus lays ...
CRISPR Babies and the Scientist Behind Their Editing
At an international genome-editing summit in Hong Kong, MIT Technology Review reporter Antonio Regalado uncovered the existence of the CRISPR baby project. The now infamous scientist, He Jiankui, had used CRISPR-Cas9, a genetic engineering tool, to edit the CCR5 gene of three embryos with ...
Interview with M.Sc. Philipp Niklas Ostermann, PhD Student
M.Sc. Philipp Niklas Ostermann PhD Student Heiner Schaal Lab Institute of Virology University Hospital Düsseldorf How did you become interested in the field of oligonucleotides? I first encountered the use of antisense oligonucleotides as potential therapeutics during my master’s thesis, which was about inhibition of ...
