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TSHA 101

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TSHA 101

Alternative Names: TGTX-101; TSHA 101

Latest Information Update: 28 May 2024

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At a glance

  • Originator University of Texas Southwestern Medical Center
  • Developer Queens University; Taysha Gene Therapies; University of Texas Southwestern Medical Center
  • Class Gene therapies
  • Mechanism of Action Gene transference; Hexosaminidase A replacements; Hexosaminidase B replacements
  • Orphan Drug Status

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

    Yes - GM2 gangliosidoses
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II GM2 gangliosidoses

Most Recent Events

  • 28 May 2024 No recent reports of development identified for preclinical development in GM2-gangliosidoses in USA (Intrathecal)
  • 05 May 2022 Taysha Gene Therapies suspends a phase I/II trial in GM2 gangliosidoses (In infants, In neonates) in Canada because the company need to submit updated regulatory information (Intrathecal) (NCT04798235)
  • 27 Jan 2022 Preliminary efficacy and adverse events data from a phase I/II trial in GM2 gangliosidoses released by Taysha-Gene-Therapies

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