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

A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)

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

Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.

This study is an open-label dose escalation phase with long-term follow-up (LTFU) period.

Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.

All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.

Inclusion Criteria:

• 1. Female and male ambulant patients
• 2. Patients ≥ 16 years old
• 3. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
• 4. Moderate diaphragmatic muscle impairment

Exclusion Criteria:

• 1. Detectable serum neutralizing antibodies against AAV9
• 2. Cardiomyopathy