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
Compound Name	GNT0006
Compound 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
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Delivery System	Viral transduction
Vector Type	AAV2/9
Editor Type	none
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