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Gene Therapy for Danon Disease (DD): A Clinical Study Evaluating the Infusion of a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease

Trial Profile

Gene Therapy for Danon Disease (DD): A Clinical Study Evaluating the Infusion of a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 05 Dec 2019

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At a glance

  • Drugs RP A501 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; First in man; Pharmacodynamics; Pharmacogenomic; Therapeutic Use
  • Sponsors Rocket Pharmaceuticals
  • Most Recent Events

    • 07 Aug 2019 According to a Rocket Pharmaceuticals media release, patient dosing continues in first cohort.
    • 18 Jun 2019 According to a Rocket Pharmaceuticals media release, patient dosing has commenced in this trial.
    • 08 May 2019 According to a Rocket Pharmaceuticals media release, data from this trial is expected in 2020.
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