Multiple Innovative Cell and Gene Therapies Receive Clinical Trial Approvals in China’s February Regulatory Wave

　　【Pharmaceutical Network Industry DynamicsGene therapy can treat and prevent diseases by altering human genes, showing promising clinical prospects in treating genetic disorders, tumors, and certain viral infectious diseases. "Cell and gene therapy and clinical translation" has been regarded as one of the major topics in China's 14th Five-Year Plan for health security development. In recent years, under regulatory norms and policy support, China’s cell and gene therapy industry has experienced rapid growth with an expanding market size. Data shows that by 2025, the market size of this industry is expected to grow to 1,788.53 billion yuan.
 

Meanwhile, the research and development of pharmaceutical products in China is in full swing. Statistics show that from 2015 to 2020, China has carried out approximately 250 clinical trials related to cell and gene therapy.
 

According to the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China, in February just ended, multiple cell and gene therapies (CGT) received implied permission for clinical trials.
 

For example, CHIGENOVO's ZVS203e injection received implied permission for clinical trials in February. This drug is a self-developed gene-editing therapeutic targeting RHO gene mutations that cause autosomal dominant retinitis pigmentosa (RHO-adRP). It obtained the FDA Orphan Drug Designation (ODD) in June 2022 and completed the first IIT subject dosing in September 2023.
 

IMC001, an autologous CAR-T cell injection targeting EpCAM independently developed by Immunofoco, was approved for clinical trials in February and obtained FDA approval for clinical research in the United States in the same month. It is intended for treating advanced digestive system tumors with positive EpCAM expression, including but not limited to advanced gastric cancer (GC) and gastroesophageal junction adenocarcinoma (GEJ). Reportedly, this product, which received IND approval in both China and the U.S., has demonstrated good safety and efficacy in IIT studies. Previously, the product was granted Orphan Drug Designation (ODD) by the U.S. FDA in August 2023.
 

Immunotech Applied Science Limited-B announced in February that the company had received approval from the Center for Drug Evaluation of the National Medical Products Administration regarding the Investigational New Drug (IND) application for Phase I clinical trial of aT19 injection. The aT19 injection is a chimeric antigen receptor T-cell (CAR-T cell) therapy targeting CD19, used sequentially after treating B-cell-derived acute lymphoblastic leukemia (B-ALL) patients under 25 years old with relapsed or refractory disease, aiming to prevent CD19-positive relapse. This injection aims to address the challenge of potential CD19-positive relapse following CD19-targeted CAR-T cell treatment for B-ALL. The Group submitted the IND application to the Center for Drug Evaluation in November 2023. It is expected that the Phase I clinical trial of aT19 injection will commence by the end of 2024.
 

Beijing Weiyuan Likang Biological Technology Co., Ltd.'s newly developed gene therapy drug WG1025 for external application on wound sites was approved for clinical trials in February. This product uses a modified type I herpes simplex virus (HSV-1) gene therapy vector to deliver the full-length COL7A1 gene, enabling the expression of intact type VII collagen protein in patients' keratinocytes and fibroblasts. This forms anchoring fibrils that anchor the epidermis to the dermis, restoring normal skin structure, promoting wound healing, and thereby exerting a therapeutic effect.
 

In addition, JWK001 Injection, independently developed by Jinweike Biotechnology, has also been approved for clinical trials. This is an innovative gene therapy drug targeting neovascular age-related macular degeneration (nAMD). It delivers AAV carrying a newly self-designed anti-VEGF protein expression cassette to ocular cells through subretinal administration, enabling sustained and efficient expression of the anti-VEGF protein. The ongoing IIT follow-up results show that JWK001 Injection has good safety and tolerability, with a low clinically effective dose. Patients only need a single administration without adjuvant therapy, avoiding risks associated with traditional nAMD treatments.
 

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