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229Aging Biotech
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LY3884963

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AAV9 progranulin gene-replacement therapy in active Phase 1/2 follow-up for GRN-associated frontotemporal dementia.

Modality
intracisternal AAV9 gene therapy encoding human progranulin
Availability (US)
Phase 1/2 PROCLAIM study Active, not recruiting
Biomarkers
adverse events; AAV9 and progranulin immunogenicity; blood and cerebrospinal-fluid progranulin; neurofilament light
Hallmark targeted
lysosomal dysfunction, impaired autophagy, loss of proteostasis, neuroinflammation
Kind
investigational gene therapy
Clinical trials
NCT04408625 — Phase 1/Phase 2, Active, not recruiting on July 16, 2026 registry update