RM-001 / Reforgene Medicine - LARVOL DELTA

All 19 subjects with transfusion-dependent β-thalassemia achieved transfusion independence after treatment with RM-001 (autologous HBG1/2 promoter-modified CD34+ hematopoietic stem and progenitor cells) (ASH 2025) - P=N/A, P1/2 | "The data from 19 TDT patients infused with RM-001 demonstrated clinically meaningful and sustainedincreases in total Hb and HbF, leading to transfusion independence in all subjects. The safety profile ofRM-001 was excellent and no product-related serious adverse events were reported during thestudy. These results indicate that RM-001 has potential to cure TDT with one-time treatment."

Clinical • Beta-Thalassemia • Genetic Disorders • CD34 • HBG1

Safety and Efficacy of RM-001 (Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells) in Patients with Transfusion-Dependent β-Thalassemia (ASH 2023) - P=N/A | "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... This updated data reported here from 7 patients with TDT infused with RM-001 demonstrated clinically meaningful increases in total hemoglobin (Hb) and HbF levels. All patients stopped receiving pRBC transfusions within 1 month after RM-001 infusion and remained transfusion-free through the time of this analysis. The safety profile of RM-001 is generally consistent with myeloablative conditioning and autologous hematopoietic stem cell transplantation."

Clinical • Beta-Thalassemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Transplantation • CD34 • HBA2

Updated Safety and Efficacy Results of RM-001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia (ASH 2024) - P=N/A, P1/2 | "The safety profile of RM-001 is very well and no product-related serious adverse event was reported during the study. These results indicate that RM-001 has the potential to cure TDT with one-time treatment."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • CD34

UPDATED SAFETY AND EFFICACY RESULTS OF RM-001, AUTOLOGOUS HBG1/2 PROMOTER-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS, IN TREATING TRANSFUSION-DEPENDENT ß-THALASSEMIA (EHA 2025) - P=N/A, P1/2 | "The data from 19 TDT pts infused with RM-001 demonstrated clinically meaningful and sustained increases in total Hb and HbF, leading to transfusion-free in all of subjects. The safety profile of RM-001 is very well and no product-related serious adverse event was reported during the study. These results indicate that RM-001 has the potential to cure TDT with one-time treatment."

Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Oncology • CD34

Updated Safety and Efficacy Results of RM-001, Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia (ASGCT 2025) - P=N/A, P1/2 | "The data from 19 TDT pts infused with RM-001 demonstrated clinically meaningful and sustained increases in total Hb and HbF, leading to transfusion-free in all of subjects. The safety profile of RM-001 is very well and no product-related serious adverse event was reported during the study. These results indicate that RM-001 has the potential to cure TDT with one-time treatment."

Clinical • Beta-Thalassemia • Genetic Disorders • Oncology • CD34

SAFETY AND EFFICACY OF RM-001, AUTOLOGOUS HBG1/2 PROMOTER-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS, IN TRANSFUSION-DEPENDENT Β-THALASSEMIA (EHA 2024) - "Peripheral CD34+ HSPCs were collected by apheresis aftermobilization with G-CSF and plerixafor...Prior to RM-001 product infusion (day 0), patientsreceived myeloablative conditioning with Busulfan from day-7 to day-4... All subjects treated with RM-001 demonstrated sustained expression of HbF to a level that means transfusionindependence. The subjects who received RM-001 treatment early have reached the safety and efficacyendpoints. This study provides clinical evidence supporting the safety and efficacy of RM-001 in treating TDT."

Clinical • Beta-Thalassemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Transplantation • CD34