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TSHA 102

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Drug Profile

TSHA 102

Alternative Names: TGTX 104; TSHA-102

Latest Information Update: 09 Jun 2025

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At a glance

  • Originator University of Texas Southwestern Medical Center
  • Developer Taysha Gene Therapies
  • Class Gene therapies
  • Mechanism of Action Gene transference; Methyl-CpG-binding protein 2 gene replacements
  • Orphan Drug Status

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

    Yes - Rett syndrome
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Rett syndrome

Most Recent Events

  • 28 May 2025 Efficacy and adverse events data from a phase I/II REVEAL trial in Rett syndrome released by Taysha Gene Therapies
  • 15 May 2025 Taysha Gene Therapies plans to file an amendment to IND application with the US FDA for phase-I/II trial for Rett syndrome in Q2 2025
  • 13 Nov 2024 Taysha Gene Therapies announces intention to submit Biologics License Application to the US FDA for Rett syndrome

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