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Clinical Trial Report

Gene Therapy Trial Report

Summary

GNT0006 Gene Therapy Trial in Patients With LGMDR9

NCTID NCT05224505 (View at clinicaltrials.gov)
Description
Indication Limb-Girdle Muscular Dystrophy, Type 2I/R9
Compound Name ATA-100 (GNT0006)
Sponsor Atamyo Therapeutics
Funder Type Industry
Status
Recruiting
Enrollment Count 39

Therapy Information

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
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2/9
Editor Type
Dose 1 9.0 x 10^12 vg/kg
Dose 2 2.7 x 10^13 vg/kg
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2021-12-15
Completion Date 2030-10
Last Update 2024-07-31

Participation Criteria

Eligible Age 16 Years - 99 Years
Standard Ages Child, Adult, Older adult
Eligible Sex ALL

Locations

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

Regulatory Information

Has US IND False
Recent Updates FDA granted Fast Track designation 6/24/24

Resources/Links

Patents
  • US20230321277A1
  • EP4031673A1
  • EP4198047A1
News and Press Releases
Preclinical Publications