Drug intelligence / Profile preview

VY-SOD102

Development stage
Discontinued
Lead developer
Voyager Therapeutics
Modality
Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intraparenchymal
01

Overview

**VY-SOD102** is an investigational **AAV-based gene therapy** developed by **Voyager Therapeutics** for **familial amyotrophic lateral sclerosis caused by superoxide dismutase 1 mutations**. The therapy uses an **AAVrh10 capsid** to deliver a proprietary **RNA interference transgene** designed to selectively reduce **SOD1 mRNA** in the spinal cord, thereby lowering production of toxic mutant SOD1 protein implicated in SOD1-associated ALS. It was intended as a **one-time intraparenchymal administration to the cervical spinal cord after laminectomy**. Preclinical studies reportedly showed substantial SOD1 mRNA knockdown throughout the spinal cord, but the program was later discontinued and Voyager indicated in 2019 that it would seek a partner rather than file an IND that year.

02

Targets

SOD (Manganese Superoxide Dismutase)

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