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PBFT02

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AAV1 progranulin gene therapy in Phase 1/2 follow-up for genetically defined frontotemporal dementia.

Modality
intracisternal AAV1 gene therapy encoding human progranulin
Availability (US)
Phase 1/2 upliFT-D study Active, not recruiting; company evaluating next clinical-development steps
Biomarkers
treatment-related adverse events; cerebrospinal-fluid progranulin; plasma neurofilament light; brain-volume MRI; AAV1 and progranulin immunogenicity
Hallmark targeted
lysosomal dysfunction, impaired autophagy, loss of proteostasis, neuroinflammation
Kind
investigational gene therapy
Clinical trials
NCT04747431 — Phase 1/Phase 2, Active, not recruiting on May 15, 2026 registry update