All 28 participants have achieved transfusion independence, each with more than one year of follow-up. China's first HBG promoter-targeted gene-editing therapy for thalassemia demonstrates best-in-class potential.

GUANGZHOU, China, October 21, 2026 — Reforgene Medicine today announced major progress in the pivotal Phase 2 clinical study of RM001, its internally developed investigational gene-editing therapy for transfusion-dependent β-thalassemia (TDT). All 28 participants in the trial have achieved transfusion independence, with more than one year of follow-up for every participant. RM001 has demonstrated outstanding clinical efficacy and safety, placing it among the leading gene-editing therapies for TDT worldwide. It also has the world's largest publicly reported clinical dataset for an HBG promoter-targeted gene-editing therapy.

Across the investigator-initiated trial (IIT) and the Phase 1 and Phase 2 studies, a total of 47 participants have received RM001. All have achieved hematopoietic reconstitution and remain transfusion-independent, with the longest follow-up reaching five years. The accumulating clinical data further strengthen RM001's competitive profile and provide a solid foundation for its continued clinical development and for addressing the treatment needs of patients with thalassemia.

Gene Editing Offers the Prospect of a Cure

Thalassemia is a group of inherited blood disorders with high carrier frequencies and a substantial disease burden in many regions worldwide. It is particularly prevalent in the Mediterranean, the Middle East, South Asia and Southeast Asia, and is also common in southern China. With population mobility and migration, thalassemia has become a shared public health challenge across many countries and regions. Patients with TDT cannot produce enough functional β-globin, experience persistent ineffective erythropoiesis and rely on regular, long-term blood transfusions to survive. Repeated transfusions lead to iron overload, which can damage multiple organs and add substantially to the disease burden. Allogeneic hematopoietic stem cell transplantation offers a potential cure, but its use is limited by donor matching requirements, risks during the transplant period and long-term complications. A treatment using a patient's own hematopoietic stem cells edited with CRISPR technology to restore effective erythropoiesis after reinfusion and achieve lasting transfusion independence could address a major unmet medical need in β-thalassemia.

Reforgene initiated the pivotal Phase 2 study of RM001 in 2025, enrolling 28 participants. The study continues to evaluate RM001's clinical efficacy and safety in a larger cohort with more complex baseline clinical characteristics. As of October 2026, all 28 participants had achieved sustained transfusion independence, each with more than one year of follow-up, and continued to derive clinical benefit.

The larger pivotal Phase 2 cohort provides further evidence of rapid and sustained hematopoietic recovery following RM001 treatment. To date, all 47 treated participants in the program have achieved sustained transfusion independence. RM001 has demonstrated outstanding performance relative to other therapies in the same class on key measures, including neutrophil engraftment, platelet engraftment and time to transfusion cessation. The median time to transfusion cessation was approximately 18 days. All participants maintained stable hemoglobin levels and remained transfusion-independent. As of the current follow-up assessment, no RM001-related serious adverse events had been reported. No cases of hepatic veno-occlusive disease, deaths, treatment interruptions or malignancies had been observed. The Phase 2 efficacy and safety findings were consistent with those from the IIT and Phase 1 study. This consistency across cohorts reflects the robustness and technological sophistication of RM001's therapeutic approach, process design and manufacturing.

RM001 has shown clinical benefits in participants across different ages and disease backgrounds. The program delivered China's first gene-editing treatment for an adult with thalassemia and its first for a patient with thalassemia who had undergone splenectomy. Both achieved transfusion independence. Clinical experience across children and adults, including participants with more complex baseline characteristics, further broadens the potential patient population for RM001.

RM001 and Its HBG1/2 Promoter-Targeted Mechanism of Action

RM001's core innovation is its targeting of the HBG1/2 promoters, an approach supported by human genetic evidence. This autologous hematopoietic stem cell gene-editing therapy developed by Reforgene received clinical trial clearance from China's National Medical Products Administration (NMPA) in November 2022. It was the first HBG1/2 promoter-targeted gene-editing therapy for thalassemia to receive clinical trial clearance in China and, based on publicly disclosed clinical information, among the first autologous hematopoietic stem cell gene-editing therapies worldwide to enter clinical development targeting these promoters. Since clinical studies began in 2022, clinical updates have been presented at the annual meetings of the European Hematology Association (EHA), the American Society of Hematology (ASH) and the American Society of Gene and Cell Therapy (ASGCT), including invited presentations on multiple occasions.

Unlike strategies that indirectly increase fetal hemoglobin (HbF), RM001 directly targets the HBG1/2 promoters to precisely regulate γ-globin gene expression and increase HbF production. The target design draws on naturally occurring protective variants in the HBG promoters. Individuals carrying these variants can maintain elevated HbF levels over the long term while retaining normal hematopoiesis. RM001 mimics this natural protective mechanism without editing the BCL11A locus, preserving BCL11A's normal physiological role in hematopoietic regulation and providing a biological basis for RM001's efficacy and long-term safety.

Efficient Gene Editing Helps Reduce Iron Overload, Organ Damage and the Burden of Iron Chelation

RM001 is administered as a single dose. Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) are edited ex vivo using CRISPR gene-editing technology and infused back into the patient. This approach links high editing levels to sustained HbF production and the restoration of effective erythropoiesis. Clinical data showed ex vivo on-target editing efficiencies of 80%–90% in CD34+ cells. After infusion, editing levels in bone marrow samples remained at 65.6%–85.4% over long-term follow-up. These findings support the long-term persistence of edited hematopoietic stem cells and the sustained production of healthy, HbF-rich red blood cells, promoting a shift from ineffective to effective erythropoiesis. HbF can account for the vast majority of total hemoglobin in participants' peripheral blood.

Persistently high levels of HBG1/2 promoter editing observed in bone marrow samples provide a key biological basis for correcting ineffective erythropoiesis and improving iron metabolism. Iron chelation is an important part of thalassemia care to help prevent organ damage from iron accumulation, making improvements in iron metabolism after gene therapy clinically meaningful. Among the 19 participants in the Phase 1 study and IIT, more than 50% experienced a gradual decline in serum ferritin to a low-risk range after treatment, without additional iron chelation therapy. These findings suggest that RM001's benefits extend beyond improvements in laboratory measures: high levels of HBG1/2 promoter editing in bone marrow and the sustained HbF production they support together block excessive intestinal iron absorption driven by ineffective erythropoiesis, thereby reducing the risk of ongoing organ damage from iron deposition.

RM001's differentiation comes from the combined strengths of its novel target, mechanism of action, editing efficiency, clinical trial design and manufacturing processes, rather than any single technical measure. Reforgene's proprietary, data-driven TIPBAIG platform supports multiple gene-editing toolsets backed by the Company's intellectual property. Its integrated research and development capabilities span tool innovation, target validation, druggability assessment, animal-model evaluation, and efficacy and safety assessment.

Reforgene is the only company worldwide to have brought gene-editing therapies for both α- and β-thalassemia into clinical development. The Company will continue to advance RM001's late-stage clinical development and work toward registration, with plans to expand development to sickle cell disease. With its differentiated gene-editing platform, Reforgene remains focused on major unmet medical needs and seeks to compete internationally through innovation, bringing high-quality new treatment options to patients worldwide.

Notes

RM001 is an investigational therapy in clinical development and has not been approved for marketing. The data reported in this release are from clinical trials and should not be taken as a definitive indication of efficacy following any future marketing approval.

This release contains forward-looking statements. These statements are subject to uncertainties, including clinical development progress and regulatory approval, and do not constitute a guarantee of therapeutic efficacy or business performance.