ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

Phase 1 Active not recruiting 6 enrolled Atamyo Therapeutics

Non-randomizedSequentialQuadruple-blindTreatment

Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up

Evaluation	Drug	Modality	Dose	Route
Subject	ATA-100	Gene therapy (AAV / viral vector)	9e+12 vg/kg	Intravenous
Subject	ATA-100	Gene therapy (AAV / viral vector)	2.7e+13 vg/kg	Intravenous