SAN DIEGO, Oct. 09, 2021was held virtually the second year in a row and was once again phenomenally successful. Over 1,000 attendees around the world engaged in lively discussions and learned about groundbreaking developments in a broad range of oligonucleotide-based disciplines.
The Rare Diseases Session featured progress in the development of ASO and RNAi therapeutics to treat rare genetic mutations that cause diseases such as ALS and Angelman Syndrome. The session also emphasized the vital role of patient advocacy groups for patients with rare diseases and highlighted the formation of N=1 Collaborative and n-Lorem. The N=1 Collaborative's mission is to make safe, individualized genetic medicines that are rapidly accessible worldwide. n-Lorem is a non-profit which develops and provides ASO therapeutics free for life to patients with extremely rare genetic mutations.
Presenters shared fascinating information on progress in innovative areas such as enhancing oligonucleotide chemistry and delivery, enabling oligos to reach additional cell and tissue types, new approaches of genome editing, and incredible imaging technologies that display organelles in cells or system-wide distribution of ASOs.
The extraordinary lifesaving applications of oligonucleotide therapeutics were demonstrated in updates on progress in Ionis's pulmonary program, Alnylam's Phase 1 study using RNAi therapeutics to provide prolonged reductions in blood pressure and the creation of Moderna's Covid-19 vaccine.
This year's award winnerswere selected for their outstanding contributions to the field, and OTS would like to congratulate Hassan Fakih, Kotaro Yoshioka, MD, PhD, and Alex Garanto, PhD.
Hassan Fakih from the Department of Chemistry, McGill University was awarded the Dr. Alan M. Gewirtz Memorial Scholarship - Graduate Students for his work on designing and testing DNA-based nanocarriers to deliver nucleic acid therapeutics with characteristics that enable them to move faster into clinical trials.
Kotaro Yoshioka, MD, PhD of the Department of Neurology and Neurological Science, Tokyo Medical and Dental University, was awarded the Dr. Alan M. Gewirtz Memorial Scholarship - Postdoctoral Fellows and Junior Industrial Professionals. He has developed unique double-stranded ASO technologies that enable highly efficient delivery to multiple tissues, including skeletal muscles, peripheral lymphocytes, and intestines via systemic injections, and to the brain via intraventricular injection.
Alex Garanto, PhD of the Radboud University Medical Center, was awarded the Mary Ann Liebert, Inc. publishers Young Investigator Award. His work focuses on the design and characterization of antisense oligonucleotides (ASOs) as a potential approach to treat inherited retinal diseases. Part of his preclinical work in targeting an intronic mutation in CEP290 led to a clinical trial that is currently in phase 3.
The Oligonucleotide Therapeutics Society thanks everyone involved in this year's meeting for contributing to the wonderful success and looks forward to hosting an in-person event next yearin Montreal, Canada, from Oct. 2-5, 2022. All interested in attending the OTS Annual Meeting are welcome as it is open to both members and nonmembers.
Media Contact:
Geri Beaty
Phone: (619) 795-9458
Email: info@oligotherapeutics.org
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