Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Trial Profile

Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 01 Dec 2017

At a glance

  • Drugs ABO 102 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man
  • Sponsors Abeona therapeutics
  • Most Recent Events

    • 09 Nov 2017 According to an Abeona Therapeutics media release, the first patient was enrolled at the Hospital Clinico Universitario of Santiago de Compostela, Spain. A total of nine subjects have been infused in this clinical trial. Safety and efficacy data have been reviewed and enrolliment in Cohort 3 continues at all three active clinical sites (US, Spain, and Australia).
    • 11 Oct 2017 Results presented in an Abeona Therapeutics media release.
    • 11 Oct 2017 According to an Abeona Therapeutics media release, two patients were enrolled for cohort 3 at sites in Australia and the US.
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