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CD34-positive cells edited by autologous CRISPR-Cas9 (RM-001) Treatment of transfusion-dependent β-thalassemia Safety and effectiveness exploration

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Trial Profile

CD34-positive cells edited by autologous CRISPR-Cas9 (RM-001) Treatment of transfusion-dependent β-thalassemia Safety and effectiveness exploration

Status: Recruiting
Phase of Trial: Phase 0

Latest Information Update: 26 Sep 2024

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At a glance

  • Drugs RM 001 Reforgene Medicine (Primary) ; Busulfan; Granulocyte colony-stimulating factors; Plerixafor
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; First in man; Therapeutic Use

Most Recent Events

  • 12 Dec 2023 Results (n=7, As of July 31, 2023) from ChiCTR2100053406 and ChiCTR2100052858 assessing safety and efficacy results from 7 patients that have been dosed with RM-001 were presented at the 65th American Society of Hematology Annual Meeting and Exposition.
  • 15 Jun 2023 Results assessing safety and efficacy from patients that received RM-001 infusion from two ongoing clinical trials (ChiCTR2100053406 & ChiCTR2100052858), including a recent treated transfusion-dependent beta -thalassemia patient who also carries two alpha-globin genes deletion presented at the 28th Congress of the European Haematology Association
  • 13 Dec 2022 Preliminary results from 2 patients presented at the 64th American Society of Hematology Annual Meeting and Exposition

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