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ATA 100

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Drug Profile

ATA 100

Alternative Names: ATA 100; GNT-0006; LGMD2I/R9 gene therapy - Atamyo Therapeutics; rAAV9-hFKRPco_miR-208a

Latest Information Update: 16 Apr 2025

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At a glance

  • Originator Atamyo Therapeutics
  • Class Gene therapies
  • Mechanism of Action FKRP protein replacements; Gene transference; Glycosyltransferases replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Limb girdle muscular dystrophies
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Limb girdle muscular dystrophies

Most Recent Events

  • 03 Apr 2025 ATA 100 receives Fast Track designation for Limb girdle muscular dystrophies [Parenteral] in USA
  • 03 Apr 2025 ATA 100 receives Rare Pediatric Disease Designation forLimb girdle muscular dystrophies in the USA
  • 03 Apr 2025 Updated efficacy data from the phase I/II trial in Limb girdle muscular dystrophies released by Atamyo Therapeutics

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