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Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene

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Trial Profile

Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 13 Nov 2024

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At a glance

  • Drugs Marnetegragene autotemcel (Primary)
  • Indications Leukocyte-Adhesion Deficiency Syndrome
  • Focus Adverse reactions; First in man; Registrational; Therapeutic Use
  • Sponsors Rocket Pharmaceuticals

Most Recent Events

  • 07 Nov 2024 According to a Rocket Pharmaceuticals media release, company is progressing on FDA-approval of KRESLADI, U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLAD. Approval anticipated in 2025.
  • 28 Jun 2024 According to a Rocket Pharmaceuticals media release, The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to Rocket's Biologics License Application for KRESLAD wherein the FDA requested limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review.
  • 10 May 2024 According to a Rocket Pharmaceuticals media release, data from this trialpresented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting.

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