VY9323

VY9323 is an investigational gene therapy developed by Voyager Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. It is an intravenously administered small interfering RNA (siRNA) therapeutic delivered via an adeno-associated virus (AAV)-based vector, designed to silence expression of the mutant SOD1 gene through RNA interference. The goal was to reduce toxic gain-of-function effects from mutant SOD1 protein in ALS patients. Preclinical studies showed that higher doses could significantly reduce SOD1 mRNA levels in brain and spinal cord, but also led to late-onset neurotoxicity due to off-target effects of the siRNA payload. As a result, development was discontinued before clinical trials commenced[2][3][5][7].

Other names

SOD1 gene therapy - Voyager TherapeuticsSOD-1 gene therapy - Voyager TherapeuticsSOD 1 gene therapy - Voyager TherapeuticsSOD1 siRNA therapeutic - Voyager TherapeuticsSOD-1 siRNA therapeutic - Voyager TherapeuticsSOD 1 siRNA therapeutic - Voyager TherapeuticsSOD1-ALSSOD-1-ALSSOD 1-ALSVY9323-SOD1VY-9323-SOD1VY 9323-SOD1