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: 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) ...
Meet OTS’ New BOD Trainee Reps
Time: 8AM PST | 11AM EST | 5PM CET Date: February 22, 2024 Title: Advancing Therapeutic Approaches for Erythropoietic Protoporphyria Description: Currently, no curative treatment is available for the rare disease erythropoietic protoporphyria (EPP). The genetic background of EPP is well ...
The First FDA Approval for a GalNAc-conjugated ASO
It starts with a pins-and-needle sensation in your feet, or maybe your gastrointestinal tract has become easily irritated, or you've started to lose weight or develop heart problems. Hereditary ATTR amyloidosis can present differently among patients and with symptoms found in far more common ...
Base Editing in Clinical Trials to Treat Acute Lymphoblastic Leukemia
Base editing has experienced a rapid rise in use since it first came on the scene in 2016, with multiple trials underway, testing its ability to treat conditions with precise, single-letter changes to DNA. The technique presents a potentially more accurate and safer method ...
Interview with Roberto El-Khoury, PhD
Roberto El-Khoury, PhD Eli Lilly and Company How did you become interested in the field? I recall being drawn to the field of oligonucleotide therapeutics in high school biology classes, but it was only years later, when I was offered the opportunity to pursue my ...
