Reforgene gene editing drug program approved under the National Key R&D Program disruptive technology innovation special project

Recently, the project "Autologous Hematopoietic Stem Cell Gene Editing Therapy for One-Time Cure of Thalassemia", led by Reforgene, was officially approved under the disruptive technology innovation special project of the National Key R&D Program, on the strength of its outstanding innovation and breakthrough clinical progress. The project is dedicated to the development of gene editing medicines, providing broad-spectrum curative medicines for thalassemia patients, achieving a lifelong cure through a single administration and bringing new hope to patients and families.

Thalassemia is the monogenic disease with the largest affected population worldwide, facing a huge unmet clinical need. For a long time there has been no universally applicable curative drug or treatment, and the vast majority of patients have had to rely on lifelong blood transfusions and medication. The gene editing medicine developed by this project is key to breaking this predicament: through precise modification of autologous hematopoietic stem cells, it restores the patient's own hematopoietic function and thereby achieves a true one-time cure, is suitable for a wide age range, involves no immune rejection, and has extremely high drug safety. In terms of application prospects, the project is expected to completely change the existing treatment model for thalassemia, providing a Chinese solution with outstanding competitiveness for the treatment of thalassemia worldwide, and holds unique international leading advantages. Based on the company's own innovation capability and national support, the project will achieve leapfrog technological innovation and foster new industries.

The disruptive technology innovation special project is an important part of the National Key R&D Program during the 14th Five-Year Plan period, dedicated to cultivating disruptive technologies and fostering new industries, new models, new drivers and new quality productive forces. The birth of disruptive technologies is the product of the deep integration of scientific drive and demand pull; like "jewels in the crown", they have major strategic value and unique technical routes, forming the great potential to change the rules of the game and continuously sparking new waves of scientific and technological revolution. The successful approval of Reforgene's project represents the nation's high recognition and support of the company's technological innovation capability.

Reforgene is the only innovative biomedical enterprise in the world that has simultaneously achieved clinical cures of both alpha-thalassemia and beta-thalassemia, with the potential to be an international leader in this field. Its beta-thalassemia gene editing medicine RM001 has achieved leading clinical results worldwide, with all treated patients rapidly cured, including the first cure of an adult beta-thalassemia patient in China. Its innovative medicine for alpha-thalassemia is also a world-first breakthrough, being the first and only alpha-thalassemia gene editing medicine to successfully cure a patient. Reforgene's research results have been presented and reported at authoritative international hematology annual meetings for three consecutive years, voicing the representative voice of Chinese companies on the global academic stage.

Reforgene will continue to anchor its core technological innovation in gene editing, contribute to disruptive technological innovation in China's life science field, actively integrate into global biomedical competition, and grow into a technology enterprise with global competitiveness and influence.

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, it achieved the world's first cure of an alpha-thalassemia patient, and the IND application for its first-in-class Usher syndrome gene editing medicine has been approved by regulators in both China and the United States.