World's first cure of an alpha-thalassemia patient with a gene editing drug: another Reforgene breakthrough

Guangzhou Reforgene Medicine Co., Ltd. ("Reforgene") recently announced that its innovative gene editing medicine RM004 has achieved a breakthrough result in clinical research for alpha-thalassemia: the first patient was successfully cured and discharged smoothly. This is the first cure case of the world's first alpha-thalassemia gene editing medicine in application, and another milestone new chapter opened by Reforgene in the thalassemia drug field, following the world's first novel-target beta-thalassemia medicine successfully completing Phase I clinical trials and curing all patients.

Thalassemia is one of the most prevalent monogenic genetic diseases worldwide, affecting more than 350 million people. Patients suffer anemia due to impaired hemoglobin synthesis, seriously affecting their health. Thalassemia is mainly divided into alpha-thalassemia and beta-thalassemia, of which alpha-thalassemia is caused by deletion or mutation of alpha-globin genes. The disease is particularly common in China, Southeast Asia, the Mediterranean coast, the Middle East and Africa, posing a serious threat to the health of millions of people worldwide. Alpha-thalassemia patients usually require long-term blood transfusions to sustain life, which not only brings economic burden but also seriously affects quality of life, causing long-term damage to organs and systems and significantly shortening life expectancy. Therefore, thalassemia poses a major challenge to global public health systems.

Reforgene's RM004 is a world-first (first-in-class) alpha-thalassemia hematopoietic stem cell gene editing medicine. It uses advanced CRISPR gene editing technology to efficiently and precisely edit the patient's gene sequences, achieving correction of the alpha-globin genes and thereby restoring hemoglobin content and oxygen-carrying function to normal. Principal investigator Professor Zhang Xinhua introduced that after receiving the drug reinfusion, the patient engrafted rapidly and achieved transfusion independence within 26 days. The successful engraftment times for neutrophils and platelets were 14 and 20 days, respectively. This is a new achievement in the field of thalassemia treatment, and it is believed that more patients will benefit in the future.

Reforgene has always been committed to solving unmet medical needs through innovative life science technologies. This success marks a major global breakthrough for Reforgene's gene editing technology in treating alpha-thalassemia. It not only helps patients escape long-term transfusion dependence, but also greatly improves the quality of life of patients and their families.

As a leading enterprise in the gene editing technology field, Reforgene focuses on the development of curative medicines for genetic diseases and high-incidence diseases to address global health challenges. Previously, Reforgene's beta-thalassemia gene editing medicine RM001 achieved globally leading clinical data and completed early clinical trials first in 2023, with all subjects cured. This was the world's first clinical report of a novel-target thalassemia gene medicine. This breakthrough in the alpha-thalassemia field further proves Reforgene's outstanding scientific research strength in drug innovation.

This milestone progress not only demonstrates the great potential of gene editing technology in treating major diseases, but also marks a solid step for humanity in conquering thalassemia, this stubborn disease. The era of cure for thalassemia patients worldwide is accelerating its arrival.

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.