Reforgene presents novel-target gene editing thalassemia clinical results at EHA 2022

The 27th European Hematology Association Congress (27th EHA Congress) will be held in Vienna, Austria, from June 9 to 17, 2022. Guangzhou Reforgene Medicine Co., Ltd. ("Reforgene"), a leading Chinese gene editing drug company, will publicly report for the first time at the 2022 EHA Congress the preliminary clinical research results of its beta-thalassemia gene editing therapy product RM001.

This is the first globally reported clinical study of thalassemia with gene editing modification of the gamma-globin promoter, and also the first time a Chinese pharmaceutical company has reported thalassemia gene editing clinical results at an international conference and at EHA.

The EHA Congress is one of the largest international conferences in the global hematology field, with more than 10,000 professionals from over 100 countries attending each year to share and discuss the latest scientific and clinical research results in hematology. Considering the COVID-19 pandemic, Reforgene chose to report the preliminary clinical research results of "RM001" in the form of a poster this time, which will present internationally leading safety and efficacy data for the product.

Abstract number: P1465. Title: Initial Safety and Efficacy Study of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Transfusion-Dependent Beta-Thalassemia. Time: June 10 (European local time).

About RM001

Thalassemia is the monogenic genetic disease with the widest distribution and largest affected population worldwide, lacking effective curative drugs and with huge unmet clinical needs. RM001 is an autologous hematopoietic stem cell gene editing therapy/product independently developed by Reforgene for the treatment of transfusion-dependent beta-thalassemia. The therapy uses CRISPR/Cas gene editing technology to permanently modify the gamma-globin promoter, activating the body's natural synthesis of fetal hemoglobin (HbF), continuously alleviating the hemolytic anemia symptoms caused by beta-globin deficiency, and achieving the goal of a one-time administration to cure beta-thalassemia.

Reforgene has carried out highly effective clinical research, and all patients smoothly escaped transfusion dependence and were discharged within 1-2 months after cell reinfusion, opening a new chapter of life. At this EHA Congress, we report the clinical observation data of the first two patients, both of whom have been followed up for more than 5 months and are in good condition.

At present, pharmaceutical companies in China, Europe and the United States are all developing curative products based on gene-edited autologous hematopoietic stem cells. The CTX-001 product of CRISPR Therapeutics in the United States, which is in Phase I/II clinical research, indirectly releases gamma-globin by editing the BCL11A erythroid enhancer to suppress BCL11A expression. Reforgene's RM001 instead adopts a novel strategy of directly reactivating gamma-globin gene expression, avoiding editing BCL11A, an important multifunctional transcription factor, and thus has better safety.