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INS1202
- Development stage
- Unknown
- Lead developer
- Insmed
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intrathecal
01
Overview
INS1202 is an investigational, intrathecally delivered gene therapy designed for the treatment of amyotrophic lateral sclerosis (ALS). Developed by Insmed, it utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a short hairpin RNA (shRNA) that targets endogenous human superoxide dismutase 1 (SOD1) mRNA. By reducing the expression of SOD1, the therapy aims to decrease the accumulation of misfolded SOD1 protein aggregates, which are associated with motor neuron death in both genetic (SOD1-mutated) and sporadic forms of ALS. Preclinical studies in nonhuman primates have demonstrated broad central nervous system biodistribution and dose-dependent knockdown of SOD1 mRNA and protein, supporting its advancement into clinical development.
- Other names
- AAV9-SOD1-shRNAAAV-9-SOD1-shRNAAAV 9-SOD1-shRNA
02
Targets
SOD (Manganese Superoxide Dismutase)
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