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# Atamyo OK’d for Gene Therapy Trial
- URL: https://www.fdaweb.com/atamyo-okd-for-gene-therapy-trial/
- Published: 2024-11-14T12:00:00.000Z
- Updated: 2026-09-14T14:47:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5158184

FDA has approved an Atamyo Therapeutics IND for a Phase 1b/2b clinical trial involving ATA-200, a one-time gene therapy for treating γ-sarcoglycan-related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5). The serious and debilitating condition is said to affect children and leads to loss of ambulation before adulthood. The company says its open-label, dose-escalation study ([NCT05973630](https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT05973630%3Fterm%3DAtamyo%26rank%3D2&esheet=54150539&newsitemid=20241112690214&lan=en-US&anchor=NCT05973630&index=2&md5=1f554b66f8cf6aaad7cd4733e16ac348&ref=fdaweb.com)) will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose adeno-associated virus vector carrying the human γ-sarcoglycan transgene.

LGMD-2C/R5 is described as a rare genetic disease caused by mutations in the gene that produces γ-sarcoglycan, a transmembrane protein that is involved in the connection between muscle fibers and their environment. About 2,000 people are affected by the disorder in the U.S. and Europe.