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ATA 200

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Drug Profile

ATA 200

Alternative Names: ATA-200; GNT-0007; LGMD-R5/LGMD2C gene therapy - Atamyo Therapeutics

Latest Information Update: 01 Jun 2026

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At a glance

  • Originator Atamyo Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; Sarcoglycan 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

  • 13 May 2026 Efficacy and pharmacodynamics data from phase I/II clinical trials in Limb girdle muscular dystrophies released by Atamyo Therapeutics
  • 09 Mar 2026 Efficacy and adverse events data from a phase Ib/II trial in Limb girdle muscular dystrophy released by Atamyo Therapeutics
  • 06 Jun 2025 ATA 200 receives Orphan Drug status for Limb girdle muscular dystrophies in USA

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