Recently, GC101 Adeno-Associated Virus Injection (hereinafter referred to as "GC101 Injection"), the first intrathecally administered AAV gene therapy drug independently developed by Genecradle Therapeutics, has officially received approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration to conduct a Phase III pivotal clinical trial for Type 2 5q spinal muscular atrophy (SMA). This Phase III pivotal clinical trial will be led by the Seventh Medical Center of the People's Liberation Army and jointly conducted by multiple centers across the country, including Beijing, Shanghai, Shenzhen, Wuhan, and Suzhou. The trial aims to further verify the efficacy and safety of GC101 Injection, providing key evidence for the drug's market application.

GC101 Injection: Revolutionizing the SMA Treatment Landscape

GC101 Injection is the first domestically developed AAV gene therapy product for the treatment of SMA via single intrathecal administration. The indications cover Types 1, 2, and 3 of 5q SMA. The successful completion of the I/II phase clinical trial of the GC101-2 Type IND project has laid the foundation for the Phase III pivotal clinical trial. Clinical trial data have shown positive and significant therapeutic effects in Type 2 SMA subjects treated with GC101 Injection, with several subjects achieving breakthroughs in motor milestones. Compared to existing treatments, GC101 Injection's potential for "one-time treatment, long-term efficacy" demonstrates a differentiated advantage. In December 2024, GC101 Injection was included in the Breakthrough Therapy drug directory by the CDE, in recognition of its outstanding performance.

Information on the recruitment of subjects for this Phase III pivotal clinical trial will be released successively through research hospitals, official Genecradle channels, and related non-profit organizations. Please stay tuned for subsequent announcements.