Guangzhou Ruifeng Biotechnology

Reforgene operates at the forefront of the gene therapy market, which was valued at over USD 2 billion in 2020 and is projected to grow significantly. The company's business model is centered on the research, development, and eventual commercialization of innovative gene-editing drugs. Its revenue streams will be derived from these proprietary drug products upon regulatory approval and market launch. Reforgene has established two primary drug development platforms: an ex vivo platform for cell therapies and an in vivo platform for direct gene therapies. The company's operations are supported by nearly 7,000 square meters of research and development space and pilot workshops.

The company's lead product, RM-001, is a gene-editing drug for transfusion-dependent β-thalassemia. This treatment involves modifying a patient's own hematopoietic stem and progenitor cells and has shown promising results in early clinical studies, with treated patients becoming transfusion-independent. RM-001 received investigational new drug (IND) approval from China's National Medical Products Administration in November 2022 and is currently in Phase I clinical trials. Reforgene has also made advancements in treating α-thalassemia, achieving a breakthrough with its RM-004 drug. The pipeline extends to other genetic diseases, including ophthalmology, with its RM-101 drug for Usher syndrome expected to receive clinical trial approvals in both the U.S. and China. The company has successfully raised significant capital to fund its clinical trials and pipeline expansion, including a Series A+ round in 2021, a Pre-B round in 2023, and another multi-hundred million RMB financing round in late 2024 led by Guangzhou Industrial Investment and Capital Group.