Reforgene Medicine to Present First-in-Class β-Thalassemia Gene-Editing Therapy RM-001 at EHA 2022
The 27th European Hematology Association (EHA) Congress will be held in Vienna, Austria, from June 9 to 17, 2022. Guangzhou Reforgene Medicine Co., Ltd. (Reforgene Medicine, hereinafter referred to as “Reforgene Medicine”), a leading Chinese enterprise in gene-editing therapeutics, will present the preliminary clinical results of its gene-editing therapy product RM-001 for β-thalassemia at the 2022 EHA Congress, marking its first public disclosure.This is the first clinical study on thalassemia involving gene-editing modification of the γ-globin promoter reported worldwide, and also the first time a Chinese pharmaceutical company has presented clinical results of gene editing for thalassemia at an international conference and the EHA.
The European Hematology Association (EHA) Congress is one of the largest international conferences in the field of hematology worldwide, attracting more than 10,000 professionals from over 100 countries each year to share and discuss the latest scientific and clinical research advances in hematology. In light of the COVID-19 pandemic, Ruifeng Biopharma has opted to present the preliminary clinical study results of “RM-001” in poster format, showcasing safety and efficacy data that demonstrate the product’s internationally leading standards.
Abstract No.：P1465
Title: A Preliminary Study on the Safety and Efficacy of Autologous CD34+ Hematopoietic Stem/Progenitor Cells Modified with the HBG1/2 Promoter (RM-001) in the Treatment of Transfusion-Dependent β-Thalassemia
INITIAL SAFETY AND EFFICACY STUDY OF RM-001, AUTOLOGOUS HBG1/2 PROMOTER-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS, IN TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Time: June 10 (Central European Time)
Thalassemia is the most widely distributed and prevalent monogenic inherited disease globally, with no effective curative drugs available, representing a significant unmet clinical need. RM-001 is an autologous hematopoietic stem cell gene-editing therapy/product independently developed by Ruifeng Bio for the treatment of transfusion-dependent β-thalassemia. This therapy utilizes CRISPR/Cas gene-editing technology to permanently modify the γ-globin promoter, activating the synthesis of naturally occurring fetal hemoglobin (HbF) in the human body. It provides sustained relief from hemolytic anemia caused by β-globin deficiency, aiming to achieve a one-time curative treatment for β-thalassemia.
Ruifeng Biologics has conducted highly effective clinical studies, with all patients successfully achieving transfusion independence and being discharged within 1–2 months after cell infusion, thereby embarking on a new chapter in their lives. At this year’s EHA Annual Meeting, we report the clinical observational data from the first two patients, both of whom have been followed up for more than five months and are in good condition.
Currently, pharmaceutical companies in China, Europe, and the United States are all developing curative therapies based on gene-edited autologous hematopoietic stem cells. CTX-001, a product from CRISPR Therapeutics in the U.S. that is undergoing Phase I/II clinical trials, works by editing the BCL11A erythroid enhancer to suppress BCL11A expression, thereby indirectly reactivating γ-globin production. In contrast, Rainbo Bio’s RM-001 adopts a novel strategy of directly reactivating γ-globin gene expression. This approach avoids editing BCL11A, an important multifunctional transcription factor, thus offering a better safety profile.
Guangzhou Ruifeng Biotechnology is a leading innovative gene therapy company in China, driven by core gene-editing technologies and dedicated to the development of groundbreaking therapeutics. Recognized with numerous honors by industry media, Ruifeng Biotechnology was the youngest company listed among the “Top 10 Cell and Gene Therapy Companies” in the 2021 Biopharmaceutical Value Sector rankings. The team comprises internationally renowned scientists in gene editing, returning scholars with research experience at top-tier overseas clinical research institutions, entrepreneurs with successful track records in the gene industry, and senior executives from multinational pharmaceutical companies. As one of the earliest teams globally to apply gene editing for exploring genetic disease treatments, Ruifeng has achieved pioneering results in hematology and ophthalmology. The company has established robust technical expertise in areas including innovative gene-editing tools, druggability strategy analysis, novel animal model development, and multi-level efficacy and safety assessments, with two major innovative drug platforms focusing on in vivo and ex vivo therapies. Currently, Ruifeng maintains a pipeline spanning genetic diseases, ophthalmology, complex diseases, and oncology.