Reforgene completes dosing of all patients in the Phase I trial of its thalassemia gene editing drug RM001

On December 26, 2023, Guangzhou Reforgene Medicine Co., Ltd. ("Reforgene") announced that the Phase I clinical study of its beta-thalassemia gene editing product RM001 (HBG gene-modified autologous CD34+ hematopoietic stem cell injection) has successfully completed drug reinfusion for all patients, marking a stage clinical achievement for the world's first novel-target thalassemia gene medicine developed by Reforgene, which is about to reach a new level.

RM001 is Reforgene's lead pipeline product, developed based on a safer editing strategy targeting the HBG gene. The product obtained approval from the National Medical Products Administration (NMPA) in November 2022 to conduct registration clinical research in China. It is the first novel-target gene editing medicine approved for clinical trials for thalassemia in China.

The Phase I clinical study of the RM001 cell injection is a multicenter, open-label clinical study aimed at evaluating the safety and efficacy of a single dose of RM001 in beta-thalassemia patients, enrolling 12 subjects aged 6 to 35, covering children, adolescents and adults.

"The completion of the infusion of the 12th and last patient in the multicenter Phase I clinical trial of RM001 is an important milestone in our development of gene editing medicines for patients with transfusion-dependent beta-thalassemia," said Dr. Liang Junbin, CEO of Reforgene. "We sincerely thank all the patients, their families and investigators who participated in this clinical trial. The results of the Phase I clinical and earlier IIT studies demonstrate the excellent safety and clinical efficacy of the RM001 product. We look forward to our efforts enabling more beta-thalassemia patients to be liberated from thalassemia as soon as possible."

Reforgene's RM001 product has achieved globally leading clinical progress. At present, a total of 19 subjects have received RM001 treatment in the Phase I clinical and IIT clinical studies. The first 5 treated patients have been continuously transfusion-free for about 2 years and have returned to normal life.

Among evaluable subjects (with reinfusion time greater than 1.5 months), 100% (17/17) of patients achieved successful engraftment and rapidly escaped transfusion. The average time to successful neutrophil engraftment was 15 days, and the average time to successful platelet engraftment was 21 days, with an average of less than one month (24 days) to stop and escape transfusion. Both the time required for hematopoietic system reconstruction and escape from the high-risk transplant period, and the time required to stop and escape transfusion, are at internationally leading levels. The first 17 subjects have been followed up for more than 1.5 months (1.5-25 months), all in a stable transfusion-free state with hemoglobin reaching normal levels, and no product-related serious adverse events occurred throughout the treatment and follow-up.

About RM001

RM001 is a beta-thalassemia gene editing medicine independently developed by Reforgene. Using CRISPR gene editing technology to modify the patient's autologous hematopoietic stem cells, it activates the body's natural fetal hemoglobin (HbF) synthesis, restoring normal physiological function of red blood cells and freeing patients from blood transfusion, achieving the goal of a one-time administration to cure beta-thalassemia. RM001 adopts an editing strategy that directly targets the promoter of the gamma-globin genes (HBG1/2), which does not affect the expression of other functional genes of hematopoietic stem cells and their differentiated functional cells, and is safer.

About Reforgene

Reforgene Medicine is one of China’s leading innovative gene editing drug companies, driven by gene editing technology and dedicated to bringing novel medicines to life. Reforgene has a high-caliber team that applied gene editing to explore the treatment of genetic diseases at an early stage internationally, and has accumulated pioneering achievements in hematology, ophthalmology and other disease areas, with strong technical capabilities in gene editing tool innovation, drug development strategy, novel animal model construction, and multi-level efficacy and safety assessment, operating both in vivo and ex vivo drug development. Reforgene’s mission is to deliver accessible, curative medicines for patients with serious diseases through innovative genetic technologies. The company currently has pipeline programs across genetic diseases, complex diseases and oncology. Its innovative beta-thalassemia medicine has achieved globally leading clinical progress, and it achieved the world’s first cure of an alpha-thalassemia patient.