From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis
On April 25 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to tofersen (trade name Qalsody) for the treatment of Superoxide Dismutase 1 (SOD1) associated amyotrophic lateral sclerosis (ALS) [1]. The clinical trial failed to meet its primary endpoint; however, approval ...
President’s Pick Article – August 2023
During the 2022 OTS annual meeting in Phoenix it was easy to listen to the talks announcing successful clinical trial results and assume that obviously favorable data were becoming routine. Willeke van Roon-Mom, Chantal Ferguson, and Annemieke Aartsma-Rus have just published a commentary (Nucleic ...
Investigating the Biology and Potential of CircRNA
In the past few years, mRNA has become a mainstream word with the swift creation of mRNA vaccines to treat SARS-CoV-2. This new technology was already in development, placing it in the perfect position to meet an urgent global need. Yet, there are still ...
Interview with Leonora Abdullahu, PhD
Leonora Abdullahu, PhD Genetic Medicines Search and Evaluation Eli Lilly & Company How did you become interested in the field?In September 2016, I joined Prof. Masad Damha’s lab at McGill University as a graduate student. To my luck, the Annual OTS meeting was being held ...
Hydrogels and Nucleic Acids – An Innovative Combination
An incredible amount of time, effort, and money have gone into finding increasingly better ways to safely and effectively deliver nucleic acid based therapeutics. Many solutions have been developed that do indeed provide safe and effective delivery, and we have had many occasions to celebrate ...
The Oligonucleotide Therapeutics Society is Pleased to Announce the 2023 Annual Meeting in Barcelona, Spain
The Oligonucleotide Therapeutics Society (OTS) is excited to bring together those in the field of oligonucleotide therapeutics for its 19th Annual Oligo Meeting. Anyone interested in oligonucleotide therapeutics is welcome to join the event, which will be held virtually and in person on Oct. ...
Non-viral DNA delivery: Opportunities and challenges
Time: 8AM PST | 11AM EST | 5PM CET Date: October 12, 2023 This presentation will cover defining the key hurdles for non-viral DNA delivery, innate immune stimulation, and delivery to post-mitotic nuclei, as well as strategies to address these challenges. ...
ASO activity and distribution after LNP delivery
Time: 8AM PST | 11AM EST | 5PM CET Date: Thursday, September 14, 2023 Webinar title: Assessing antisense oligonucleotide delivery to the brain What is your research about? Amy Byrnes, PhD, focuses her research on understanding how delivery vehicles impact antisense oligonucleotide (ASO) ...
Muscle targeted siRNA Conjugates for Pompe Disease
Time: 8AM PST / 11AM EST / 5PM CET Date: August 3, 2023 Aro Biotx is focused on developing Centyrin-siRNA conjugates for treatment of extrahepatic genetic diseases. Our lead program employs a CD71 Centyrin for targeting to muscle together with an siRNA for ...
Developing Oligonucleotides Into Therapies: Opportunities and Challenges
Time: 8AM PST / 11AM EST / 5PM CET Date: July 6, 2023 Speakers: Shalini Andersson, PhD Vice President of Oligonucleotide Discovery AstraZeneca View Bio
