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A phase 1 dose-escalation study of RP-A501 in patients with Danon disease

Trial Profile

A phase 1 dose-escalation study of RP-A501 in patients with Danon disease

Phase of Trial: Phase I

Latest Information Update: 22 Jan 2019

At a glance

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

    • 22 Jan 2019 According to a Rocket Pharmaceuticals media release, the U.S. Food and Drug Administration (FDA) has cleared the IND application for RP-A501. This trial is expected to start in the second quarter of 2019.
    • 07 Dec 2018 New trial record
    • 26 Nov 2018 According to a REGENXBIO media release, the company has granted Rocket Pharmaceuticals worldwide rights to NAV AAV9 for the development and commercialization of gene therapy treatments (exclusive options for two additional undisclosed NAV AAV vectors) for Danon Disease, through the new license agreement.The IND application (NAV AAV9 vector) is expected to be filed in 2019, which will be followed by a clinical trial.
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