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Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB

Trial Profile

Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 15 Jul 2019

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At a glance

  • Drugs ABO-101 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Acronyms Transpher B
  • Sponsors Abeona therapeutics
  • Most Recent Events

    • 14 May 2019 According to an Abeona Therapeutics media release, the Company has completed dosing cohort 1 and treated the first patient in cohort 2 in the trial. The company expects to report interim data from the trial in the second half of 2019.
    • 04 Apr 2019 According to an Abeona Therapeutics media release, the patients are being enrolled in the trial at sites in the U.S. and Spain and expects to activate additional sites globally this year to accelerate enrollment.
    • 12 Sep 2018 According to an Abeona Therapeutics media release, the company announced authorization to move forward this trial in Spain. The clinical study was approved by the Agencia Espanola de Medicamentos y Productos Sanitarios and is being conducted at Hospital Clinico Universitario of Santiago de Compostela, Spain. The company plans to add sites for the trial in three European countries including France, Germany and the United Kingdom.
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