Targeting ALS-FUS: Early Insights from the Jacifusen (ION363) Case Series
Amyotrophic lateral sclerosis (ALS) remains one of the most devastating diseases and is the most common motor disease among adults, affecting around 55,000 people in the US, Europe, and Japan. More than 40 genes have been linked to ALS, providing a valuable opportunity for ...
New Approaches for Functional Oligonucleotides: RNA Activation and Non-Nucleosidic Aptamers.
Date: November 20, 2025Time: 11-12pm EST / 5-6pm CET Register Here Title: New Approaches for Functional Oligonucleotides: RNA Activation and Non-Nucleosidic Aptamers Description: This talk will highlight two complementary approaches aimed at expanding the functional scope of oligonucleotide therapeutics. First, I will present ...
Financing the Future of Individualized Medicine: Funding Models for Rare Genetic Therapies
Date: September 18, 2025 Time: 11-12pm EDT / 5-6pm CEST Title:Financing the Future of Individualized Medicine: Funding Models for Rare Genetic Therapies Description: Individualized medicine has emerged as an approach offering bespoke therapeutics to patients with rare genetic conditions. In spite ...
From Discovery to Deals: The Neurological Oligonucleotide Landscape at a Glance
Date: September 4, 2025 Time: 11-12pm EDT / 5-6pm CEST Title: From Discovery to Deals: The Neurological Oligonucleotide Landscape at a Glance Description: A deep dive on the drug landscape, displaying critical data-driven therapeutic and clinical trial highlights, as well as ...
Behind the Rapid Development of an Individualized CRISPR Therapy for a Deadly Rare Disease
A baby is the first person to receive a bespoke CRISPR therapy designed specifically for him that directly fixes a disease-causing mutation in his genome. It wasn't long after KJ was born in the summer of 2024 that doctors noticed something was wrong. Within ...
2024 OTS Travel Grant Recipients
Muna Abedrabbo, The University of British ColumbiaUnnur J. Björgvinsdóttir, Technical University of DenmarkMathilde Blitek, U1179 UVSQRut Bryl, UT Southwestern Medical CenterJessica Cale, PhD, Centre for Molecular Medicine and Innovative TherapeuticsMyriam Cerezo-Magaña, PhD, Lund UniversityDavid Cheerie, The Hospital for Sick ChildrenMarguerite Colin, MRC Toxicology Unit, University ...
2024 Patient Advocacy Award
Katie Gross Research Associate, RNA Therapeutics Institute, UMASS Chan Medical School Biography Katie Gross is a Research Associate in the laboratory of Dr. Julia Alterman and Dr. Anastasia Khvorova in the RNA Therapeutics Institute at the University of Massachusetts Chan Medical School. She began ...
2024 Dr. Alan M. Gewirtz Memorial Scholarship – Graduate Students
Jillian Belgrad, M.D./Ph.D. Candidate, MS3/GS4 Khvorova Lab, RNA Therapeutics Institute, UMass Chan Medical School Biography: Jillian Belgrad is a sixth-year MD/PhD student in the RNA Therapeutics Institute at UMass Chan Medical School. Before joining UMass Chan, Jillian graduated from Colgate University with a ...
2024 Dr. Alan M. Gewirtz Memorial Scholarship – Postdoctoral Fellows and Junior Industrial Professionals
Hassan H. Fakih, Ph.D. Post-Doctoral Associate, Prof. Anastasia Khvorova Research Group RNA Therapeutics Institute | UMASS Chan Medical School Biography: Hassan is a Postdoctoral Associate in the lab of Prof. Anastasia Khvorova at the RNA Therapeutics Institute at UMass Chan Medical School. His research focuses on ...
