The Oligonucleotide Therapeutics Society (OTS) is an open, nonprofit forum to foster research and development of oligonucleotide therapeutics.
The Founders’ vision was to bring together the expertise from different angles of oligonucleotide research to create synergies and to bring the field of oligonucleotides to its full therapeutic potential. A new era of oligonucleotide drugs was envisioned that would change the landscape of therapeutic modalities.
Oligonucleotide therapeutics are now being used to successfully treat diseases and have the potential for widespread application. Over 100 treatments are in development for common conditions, including cancer and Alzheimer’s disease, as well as rare diseases. Incredibly, oligonucleotide therapeutics have also been used to create an individualized treatment for one single person with a rare, fatal disease.
All the latest news
Martin Egli and Muthiah Manoharan’s Review of Approved Oligonucleotide Therapeutics
An ASO Is Granted Accelerated Approval By the FDA
Tofersen, an antisense oligonucleotide treatment for SOD1-ALS, a rare form of amyotrophic lateral sclerosis (ALS), was recently granted accelerated ...
Innovation, Versatile Gene Editing Technologies, and Patent Battles
Last June, at a genome editing summit in Lisbon, following a much-anticipated presentation by Tessera Therapeutics about their new ...
Can There Be International Agreement on How to Navigate the Future of Genome Editing?
Our world is changing at an astonishingly rapid pace and, as described a few weeks ago, CRISPR is poised ...
Steven F. Dowdy, Ph.D.
Department of Cellular & Molecular Medicine,
University of California San Diego (UCSD), School of Medicine
In 2004, we started addressing the siRNA (and ASO) delivery problem, which is really just the endosomal escape problem, that plagues all RNA therapeutics. We were the first lab to synthesize neutral, bioreversible phosphotriester groups on siRNAs…
Our work over the last 10 years has solely been focused on enhancing endosomal escape in a clinically acceptable non-toxic manner, which remains highly elusive.