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LY3884961

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AAV9 GBA1 gene therapy in Phase 1/2 follow-up for GBA1-associated Parkinson's disease and Gaucher disease.

Modality
single-administration AAV9 gene therapy delivering a functional human GBA1 gene
Availability (US)
Two Phase 1/2 studies Active, not recruiting in Parkinson's disease with GBA1 mutation and Gaucher disease
Biomarkers
treatment-emergent adverse events; AAV9 and glucocerebrosidase immunogenicity; glucocerebrosidase activity; glycolipid substrates; neurofilament light
Hallmark targeted
impaired autophagy-lysosomal function, loss of proteostasis, neurodegeneration
Kind
investigational gene therapy
Clinical trials
NCT04127578 — Phase 1/Phase 2 PROPEL, Active, not recruiting on September 18, 2026 update; NCT05487599 — Phase 1/Phase 2 PROCEED, Active, not recruiting on August 17, 2026 update