World Thalassemia Day: Reforgene contributes China's innovative medicine solutions toward a thalassemia-free world

On May 6, as "World Thalassemia Day" approached, the International Thalassemia Prevention and Treatment Forum was grandly held in Qionghai City, Hainan Province. Guided by the Thalassaemia International Federation (TIF) and hosted by the Beijing Angel Mama Charity Foundation and the Hainan Thalassemia Prevention and Care Association, the forum brought together experts, scholars and medical professionals from the global thalassemia field. As the only gene editing innovative drug leading enterprise in the world to have achieved cures for both alpha- and beta-thalassemia, Reforgene was invited to attend and deliver a special report, contributing wisdom and strength to the global thalassemia cause.

The forum attracted more than 200 participants including clinical institutions, biomedical enterprises, public welfare organizations, and government and patient representatives from the domestic and international thalassemia field. Dr. Androulla Eleftheriou, President of TIF, Wang Zhenyao, Director of the China Philanthropy Research Institute, Qiu Lili, founder of the Angel Mama Foundation, Lu Zhiyuan, President of the Hainan Charity Federation, and other experts and leaders gathered to discuss new technologies and advances in thalassemia prevention and treatment, and to chart a health blueprint for a thalassemia-free world, injecting new hope for human health and well-being.

As an inherited blood disease, thalassemia traps countless patients and their families in the struggle of long-term blood transfusions, medication maintenance and complications, and its high incidence constitutes a major public health burden in some regions. Breakthroughs in curative therapies have become the key to changing the status quo. It is worth noting that gene editing technology is revolutionizing the field by offering thalassemia patients a one-time treatment that achieves permanent "functional cure", gradually reshaping the traditional treatment paradigm of transfusion dependence.

As a specially invited enterprise representative, Professor Huang Junjiu, Chief Scientific Advisor of Reforgene, delivered a keynote report titled "Progress in Gene Editing Treatment of Alpha- and Beta-Thalassemia from a Global Perspective", systematically sharing Reforgene's innovative breakthroughs in alpha- and beta-thalassemia gene editing medicines. Relying on its independently developed gene editing technology platform and a dual-drive strategy of underlying technology breakthroughs and drug R&D, Reforgene has become the only enterprise in the world to simultaneously develop alpha/beta thalassemia gene editing therapies and achieve clinical cures. To date, it has helped a cumulative total of 19 beta-thalassemia patients and 1 alpha-thalassemia patient escape transfusion dependence, contributing a Chinese solution to global thalassemia treatment.

Reforgene's core product, the beta-thalassemia gene editing medicine RM001, has entered a critical clinical stage. Based on in-depth research into thalassemia genomics, the product adopts a proprietary HBG-targeted editing strategy that does not affect the proliferation and differentiation of hematopoietic stem cells, ensuring long-term safety. Through optimized manufacturing processes, RM001 effectively protects the stemness of hematopoietic stem cells, laying the foundation for excellent efficacy. Clinical data show that the product has successfully cured China's first adult beta-thalassemia patient, the first patient who had undergone splenectomy, and the first patient with combined beta- and alpha-thalassemia, with rapid hematopoietic reconstitution, fast platelet recovery and short hospital stays, demonstrating the potential to rewrite the existing treatment landscape.

In addition, Professor Huang Junjiu participated as a guest in the roundtable forum, offering unique insights on topics including the clinical translation and challenges of thalassemia gene therapy, the clinical application safety of gene editing technology, and future trends in thalassemia diagnosis and treatment.

The forum built a multi-party cooperation platform among government, enterprises, public welfare organizations and international institutions, calling on society to pay attention to thalassemia prevention and treatment and promote the upgrading of the global thalassemia prevention and treatment system. As a frontrunner in China's gene editing medicine field, Reforgene will continue to practice the philosophy of "Innovation for Life", work with clinical experts, accelerate the clinical translation of thalassemia gene therapies, and strive for early market launch to benefit patients worldwide.