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

NCTID	NCT05224505 (View at clinicaltrials.gov)
Description	Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up (Show More)
Development Status	Active
Indication	Limb-Girdle Muscular Dystrophy, Type 2I/R9
Disease Ontology Term	DOID:0110299
Drug Product Name	GNT0006
Drug Product Alias	ATA-100
Sponsor	Atamyo Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	6 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	FKRP
Therapeutic Modality	Gene transfer
Therapy Route	in vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Gene Delivery System Type	Viral transduction
Vector Type	AAV2/9
Dose 1	9.0E12 vg/kg
Dose 2	2.7E13 vg/kg

Eligible Age	16 Years - 99 Years
Standard Ages	Child, Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	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 View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	3
Locations	Denmark,United Kingdom,France

Has US IND	True
FDA Designations	Fast Track, Rare Pediatric Disease Designation
Recent Updates	Dose-finding study completed April 2025