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Haematopoietic stem cell gene therapy - Foundation for Angelman Syndrome Therapeutics/TransformaTx Biotherapeutics/University of California at Los Angeles

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Drug Profile

Haematopoietic stem cell gene therapy - Foundation for Angelman Syndrome Therapeutics/TransformaTx Biotherapeutics/University of California at Los Angeles

Alternative Names: ube-cel; ube-cel therapy; UBE3A lentiviral therapy program - FAST

Latest Information Update: 13 Aug 2026

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At a glance

  • Originator University of California, Davis
  • Developer TransformaTx Biotherapeutics
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action UBE3A protein expression stimulants
  • 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 Angelman syndrome

Most Recent Events

  • 05 Aug 2026 Foundation for Angelman Syndrome Therapeutics in-licenses TfR1 CapX™, Apertura's blood-brain barrier-crossing capsid technology from Apertura Gene Therapy
  • 30 Jan 2025 Preclinical trials in Angelman syndrome in USA (Parenteral)
  • 30 Jan 2025 TransformaTx Biotherapeutics and UCLA plan to file an IND application with the US FDA for Angelman syndrome

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