AGTC-801

AGTC-801 is an experimental **AAV-based gene therapy** program developed by Applied Genetic Technologies and later associated with Beacon Therapeutics after AGTC's acquisition. Publicly available pipeline references identify it as a preclinical central nervous system program for **amyotrophic lateral sclerosis** linked to **C9orf72**, but detailed construct-level information has not been clearly disclosed. Based on the available information, the program appears intended to modulate or replace dysfunctional C9orf72 biology in C9orf72-associated ALS, but its exact therapeutic design, transgene payload, dosing route, and clinical formulation remain undisclosed. Development appears to have remained preclinical and was later deprioritized or discontinued.