AGTC-601

**AGTC-601** is an investigational **AAVrh10-based gene therapy** designed to deliver a functional **granulin gene** to restore progranulin expression in **frontotemporal dementia caused by GRN mutations**. The program was originated by **AGTC** and later became part of the portfolio associated with **Beacon Therapeutics** following the Syncona-backed acquisition of AGTC. Publicly available information indicates it was in **advanced preclinical development** around 2022, with toxicology and biodistribution studies planned to support an IND, and later preclinical reports described it specifically as an **AAVrh10-granulin gene therapy** for **GRN-mutant frontotemporal dementia**. Its intended therapeutic rationale is gene replacement to increase progranulin levels and thereby modify the underlying biology of progranulin haploinsufficiency rather than provide only symptomatic treatment.