RM-004 / Reforgene Medicine - LARVOL DELTA

Curing Hemoglobin H-Constant Spring through Base Editing of Autologous CD34+ Hematopoietic Stem and Progenitor Cells (ASH 2025) - P1 | "Peripheral CD34+ HSPCs werecollected by apheresis after mobilization with G-CSF and plerixafor...The safety profilewas generally consistent with busulfan myeloablation and autologous hematopoietic stem celltransplantation...These results present the first successful application of gene editing in treating a patient with transfusion-dependent α-thalassemia. The patient treated with RM-004 demonstrated successful engraftment and aclinically meaningful increase in hemoglobin levels, leading to transfusion independence. This proof-of-concept study indicates that RM-004 is a promising therapy for the treatment of HbH-CS."

Bone Marrow Transplantation • Genetic Disorders • CD34 • HBA2

First-in-Human Study of Autologous HBA2-Edited CD34+ Hematopoietic Stem and Progenitor Cells in Alpha-Thalassemia with Constant Spring Mutation (ASH 2024) - P1 | "Peripheral CD34+ HSPCs were collected by apheresis after mobilization with G-CSF and plerixafor...Prior to RM-004 infusion, patient received myeloablative conditioning with busulfan form day-7 to day-3...This is the first-ever clinical report of patients with α-thalassemia treated successfully with gene editing and indicates that RM-004 is a promising approach for the treatment of HbH-CS. Data will be updated for the presentation."

First-in-human • P1 data • Bone Marrow Transplantation • Genetic Disorders • CD34 • HBA2

FIRST-IN-HUMAN STUDY OF AUTOLOGOUS HBA2-EDITED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN ALPHA-THALASSEMIA WITH CONSTANT SPRING MUTATION (EHA 2025) - P1 | "Peripheral CD34+ HSPCs were collected by apheresis after mobilization with G-CSF and plerixafor...Prior to RM-004 infusion, patient received myeloablative conditioning with busulfan form day-7 to day-3... The first patient treated with RM-004 demonstrated successful engraftment and clinical meaningful increase in Hb level. Although the follow-up time is relatively short, available data have shown that the patient has entered a transfusion-independence state. This is the first-ever clinical report of patients with α-thalassemia treated successfully with gene editing and indicates that RM-004 is a promising approach for the treatment of HbH-CS."

P1 data • Bone Marrow Transplantation • Genetic Disorders • CD34 • HBA2

First-in-Human Study of Autologous HBA2-Edited CD34+ Hematopoietic Stem and Progenitor Cells in Alpha-Thalassemia with Constant Spring Mutation (ASGCT 2025) - P1 | "Peripheral CD34+ HSPCs were collected by apheresis after mobilization with G-CSF and plerixafor...The safety profile was generally consistent with busulfan myeloablation and autologous hematopoietic stem cell transplantation... The first patient treated with RM-004 demonstrated successful engraftment and clinical meaningful increase in Hb level. Although the follow-up time is relatively short, available data have shown that the patient has entered a transfusion-independence state. This is the first-ever clinical report of patients with α-thalassemia treated successfully with gene editing and indicates that RM-004 is a promising approach for the treatment of HbH-CS."

P1 data • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • CD34 • HBA2

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease (clinicaltrials.gov) - P1 | N=5 | Recruiting | Sponsor: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

New P1 trial • Genetic Disorders • Hematological Disorders