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AAVrh10-miRNA-SOD1
- Development stage
- Discontinued
- Lead developer
- uniQure
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Intraparenchymal
01
Overview
AAVrh10-miRNA-SOD1 (also known as AMT-160) is an experimental gene therapy developed by uniQure for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The therapy utilizes an adeno-associated virus serotype rh10 (AAVrh10) vector to deliver a microRNA (miRNA) expression cassette designed to silence the expression of the superoxide dismutase 1 (SOD1) gene through RNA interference (RNAi). By reducing the levels of mutant SOD1 protein, which is known to exert toxic gain-of-function effects in motor neurons, the therapy aims to slow or halt the progression of the disease. Development was discontinued by uniQure in 2020 to prioritize other pipeline assets.
- Other names
- AAVrh10-miSOD1AAVrh-10-miSOD1AAVrh 10-miSOD1
02
Targets
SOD (Manganese Superoxide Dismutase)
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