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Gene therapy - ASC Therapeutics/University of Massachusetts

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Drug Profile

Gene therapy - ASC Therapeutics/University of Massachusetts

Alternative Names: Dual-function gene replacement vector therapy; MSUD AAV9 gene therapy replacement vector; MSUD gene therapy - ASC Therapeutics/University of Massachusetts Medical School

Latest Information Update: 25 May 2022

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At a glance

  • Originator ASC Therapeutics; University of Massachusetts Medical School
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Maple syrup urine disease

Most Recent Events

  • 16 May 2022 Pharmacodynamics data from preclinical trials in Maple syrup urine disease released by ASC Therapeutics and University of Massachusetts Medical School
  • 16 May 2022 Preclinical trials in Maple syrup urine disease in USA (Parenteral)
  • 16 May 2022 Pharmacodynamics data from preclinical trials in murine models and a calf presented at the 25th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT-2022)

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