2026 Dr. Alan M. Gewirtz Memorial Scholarship – Postdoctoral Fellow
Mitsugu Yanagidaira, MD, PhD Tokyo Medical and Dental University Talk Title: Heteroduplex Oligonucleotides: A Platform for Enhanced Efficacy and Improved Safety of Antisense Oligonucleotides Mitsugu Yanagidaira, MD, PhD, is a Postdoctoral Research Fellow at Brigham and Women’s Hospital and Harvard Medical School. He received his ...
2026 Dr. Alan M. Gewirtz Memorial Scholarship – Graduate Student
Sai Pallavi Pradeep University of Connecticut Talk Title: Developing Next-Generation Peptide Nucleic Acids for Antigene Therapy Sai Pallavi Pradeep earned her B.Sc. in Chemistry, Zoology, and Microbiology from Bangalore University and her M.Sc. in Applied Microbiology from Vellore Institute of Technology in India. Following ...
Mei’s Story: Investigation Reveals Unreported Fatal Outcome After In Vivo Base Editing of CHD3
In March 2025, a six-year-old girl received the first reported in vivo base-editing treatment directed at the human CNS. The girl, given the pseudonym “Mei” — meaning “beautiful” in Chinese — had Snijders Blok-Campeau syndrome, an extremely rare genetic condition associated with mutations in ...
Roche Ends Two HD Clinical Trials as Other Huntingtin-Lowering Therapies Advance
On July 9, Roche announced it would discontinue two ongoing clinical studies investigating treatments for Huntington's disease (HD) after separate analyses showed the programs would not achieve their intended goals. While the news is a significant disappointment for the HD community, both studies have ...
Antisense Oligonucleotide Therapy in Chronic Hepatitis B: Clinical Progress and Phase 3 Outcomes
When Sheree was 26, she kept wondering why she was always so tired. Though she was a mom and a nurse working multiple double shifts, she was still young and knew she shouldn’t be this fatigued. Then the nausea and abdominal pain began. Soon, the ...
Al3Cas12f RKK: A New, Enhanced Compact Editor that is Highly Efficient
CRISPR-based gene-editing tools have transformed the biomedical field, but delivering these systems directly into human tissues — known as in vivo gene editing — remains a major challenge. Many therapeutic CRISPR approaches still rely on ex vivo editing, where the patient’s cells are removed, ...
OTS Member Highlight – Keith T. Gagnon, PhD
As a child, before Keith Gagnon knew anything about science, he told his mom he wanted to be a scientist when he grew up. Now, decades later, Dr. Keith Gagnon is a biochemist and molecular biologist specializing in RNA biology, nucleic acid therapeutics, repeat ...
Interesting Findings on the Impact of Antisense Oligonucleotides on Genome Stability and DNA-Repair Enzyme Activation
Antisense oligonucleotides (ASOs) have emerged as promising drug candidates for the treatment of genetic diseases. Because of their ability to target virtually any disease-related gene product, several ASO drugs have been approved, and many more are in development (1). While they remain among the most ...
Beyond the “Too Rare” Barrier: Platforms for Rare Mutations Leverage New Regulatory Tool
In November, Food and Drug Administration (FDA) officials introduced the Plausible Mechanism Pathway, a regulatory tool designed to make it easier to approve treatments — specifically investigational gene therapies — for rare conditions. An article published in The New England Journal of Medicine (1) ...
Bioanalysis of Oligonucleotide Therapeutics via Nucleic Acid Nanorobotic Platform
Date: May 21, 2026 Time: 11-12pm EDT / 5-6pm CEST Title: Bioanalysis of Oligonucleotide Therapeutics via Nucleic Acid Nanorobotic Platform Description: Dynamic DNA nanotechnology enables the isothermal and enzyme-free exchange of nucleic acid strands. The underlying mechanism of action in this ...
