2023 Paper of the Year Award – Late Discovery Category Winner Jinkuk Kim, PhD
Jinkuk Kim, PhD KAIST - Korea Advanced Institute of Science and Technology A framework for individualized splice-switching oligonucleotide therapy https://pubmed.ncbi.nlm.nih.gov/37438524/ Biography: Jinkuk Kim is an Assistant Professor at KAIST in South Korea. He obtained a B.S. in computer science and a Ph.D. ...
2023 Annual Meeting Highlights
We are pleased to note that this year’s annual Oligonucleotide Therapeutics Society meeting in Barcelona, Spain had the highest involvement of any previous meeting, with 800 in-person attendees, 297 abstract submissions, 260 posters, 47 travel grants, 36 Sponsors, and 29 Exhibitors. We hope all ...
Fanzor: A Programmable RNA-Guided System In Eukaryotes Similar to CRISPR
Researchers have uncovered the first programmable RNA-guided system in eukaryotes that could be even more precise than CRISPR gene-editing. The discovery — led by Feng Zhang at the McGovern Institute for Brain Research at MIT and the Broad Institute of MIT and Harvard — demonstrates ...
RNAi: The Persistence of a Few Leads to Success
On August 10, 2018, the U.S. Food and Drug Administration (FDA) approved the first RNAi therapy. The approval marked a significant milestone not just for the drug but the entire field of RNA interference (RNAi). The success of the technology was never guaranteed, and ...
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 ...
