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A registrational trial of RP-L102 for Fanconi's Anemia

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Trial Profile

A registrational trial of RP-L102 for Fanconi's Anemia

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 08 Nov 2023

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At a glance

  • Drugs Mozafancogene autotemcel (Primary)
  • Indications Fanconi's anaemia
  • Focus Registrational; Therapeutic Use
  • Sponsors Rocket Pharmaceuticals

    • Most Recent Events

    • 06 Nov 2023 According to a Rocket Pharmaceuticals media release, Product filings for RP-L102 anticipated in the first half of 2024 in the U.S. and Europe. The company is finalizing the CMC package with the FDA.
    • 19 May 2023 According to a Rocket Pharmaceuticals media release, positive, updated data (cut-off April 17, 2023) from this study presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, California.
    • 19 May 2023 According to a Rocket Pharmaceuticals media release, based on the safety and efficacy data from this study, company anticipates filing the Biologics License Application (BLA) with the FDA in the fourth quarter of 2023.
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