President’s Pick Article – April 2024
True to the vision of OTS, we worked with our journal publisher, Mary Ann Liebert publishers, Inc., through our BOD Committees to compile a list of important open-access Nucleic Acid Therapeutics (NAT) publications relating to the development of safe oligonucleotides. Finding high-quality publications to guide ...
RNA Single-base Editing Therapy that Treats Genetic Lung and Liver Disease Entered Clinical Trials
When Peggy's mom was in her forties, she started to have trouble breathing. Although she had never smoked, she had been exposed second-hand earlier in her life. Eventually, her mother ended up at the Mayo Clinic, where she was the 36th patient to be ...
Jennifer Doudna – Seeking to Improve the World with CRISPR
At 10:53 p.m. on Oct. 7, Jennifer Doudna woke to a buzzing sound. Picking up her phone — which she noticed had multiple missed calls and messages — she was greeted by the voice of Nature journalist Heidi Ledford. The day before, Doudna had ...
FDA Approved the First CRISPR Treatment – Potential Cure for People with Sickle Cell Disease
Victoria Gray was diagnosed with sickle cell disease (SCD) when she was just three months old; since then, episodes of pain and frequent hospitalizations have been a part of her life. Many of her dreams seemed like far-off impossibilities when the smallest things, like ...
RNA Targeting Technology – Chemistry and Delivery
Time: 8am PDT / 11am EDT / 4pm CET Date: March 21, 2024 Title: RNA Targeting Technology – Chemistry and Delivery Description: Chemistry coupled with pharmacology, both preclinical and clinical, forms the basis for the pipeline we manage at Ionis today. ...
2023 Annual Meeting Poster Awardee Spotlight
Time: 8AM PST | 11AM EST | 5PM CET Date: February 29, 2024 Title: Towards Allele-Selective Gapmer ASO Design Description:Â Allele selective gapmer antisense oligonucleotides (ASOs) are a promising strategy for the treatment of rare monogenic neurodevelopmental disorders caused by gain-of-function or ...
Edward M. Kaye, MD
Edward M. Kaye, M.D. Chief Executive Officer and Director of Stoke Therapeutics How did you become interested in the field? My interest in genetic medicine began as a biochemical geneticist and pediatric neurologist. Back in the 1990s, when I was treating patients, I saw first-hand ...
2023 OTS Lifetime Achievement Award Winner Cy A. Stein, MD, PhD
Cy A. Stein, MD, PhD Cy A. Stein, MD, PhD was born in New York City. He received a BA degree from Brown University (1974) and a PhD in chemistry from Stanford (1978) under Henry Taube (Nobel Laureate, 1983). A career change took him ...
2022 OTS Lifetime Achievement Award Winner Sudhir Agrawal, D.Phil.
Sudhir Agrawal, D.Phil.Founder and President, Arnay SciencesDr. Agrawal is the founder and President of Arnay Sciences, a life science research company pursuing RNA therapeutics. Dr. Agrawal started his work on antisense in the late eighties in the laboratory of Paul Zamecnik, a pioneer of antisense ...
Follow-up Data Confirms That Inclisiran Provides Long-Term Reduction of LDL-Cholesterol
Cardiovascular disease is the leading cause of death in the United States, exceeding all types of cancer, unintentional injury, and stroke combined. However, updated data from Novartis's open-label trial shows promising long-term results for inclisiran (Leqvio), the first and only small interfering RNA (siRNA) ...
