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SOD1 silencing gene therapy
- Development stage
- Discontinued
- Lead developer
- Voyager Therapeutics
- Modality
- Gene Therapies
- Administration
- Intrathecal, Intraspinal, Intracerebral, Intrapleural
01
Overview
SOD1 silencing gene therapy is a gene therapy program developed by Voyager Therapeutics, leveraging their TRACER AAV platform. It is designed to silence the SOD1 gene, which is implicated in certain forms of amyotrophic lateral sclerosis (ALS). The program is being co-developed and commercialized in collaboration with Novartis for the treatment of amyotrophic lateral sclerosis.
02
Targets
SOD (Manganese Superoxide Dismutase)
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