FDA grants RMAT designation to MeiraGTx's AAV-GAD for Parkinson's disease

MeiraGTx Holdings plc

RMAT designation based on data from three clinical studies (Phase 1 n=12, Phase 2 n=45, dose-ranging n=14).
AAV-GAD showed significant benefit on UPDRS Part 3 motor endpoint in double-blind sham-controlled trials.
Hologen AI analysis indicated disease-modifying brain circuitry changes, first in sham-controlled Parkinson's study.
RMAT includes Fast Track and Breakthrough Therapy benefits, rolling review, and potential Priority Review for BLA.
MeiraGTx plans to work closely with FDA to advance this one-time gene therapy for Parkinson's disease.

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