GeneThera Biotech's GC101, China’s First Intrathecal AAV Gene Therapy for SMA, Enters Pivotal Phase III Clinical Trial

Recently, GC101, the first intrathecal AAV gene therapy drug independently developed by Genecradle in ChinaAdeno-Associated Virus Injection (hereinafter referred to as GC101 Injection) has officially received approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) to commence research onType 2 5q Spinal Muscular Atrophy (Type 2 SMA)Phase ⅢPivotal Clinical Trial. This itemPhase ⅢCriticalityClinical TrialTest, will be handled byThe Seventh Medical Center of the Chinese People's Liberation ArmyAs the lead organization, in collaboration withMulticenter in China, including Beijing, Shanghai, Shenzhen, Wuhan, Suzhou, etc.Jointly carry out.Aims toStep inTo further verify the effectiveness and safety of GC101, providing critical evidence support for drug marketing application.。

GC101 Injection: Revolutionizing the SMA Treatment Landscape

GC101 Injection is independently developed by Genecradle.ChinaInFirst SingleIntrathecal AdministrationAAV gene therapy product for treating SMA. Indications cover Type 1, Type 2, and Type 3 5qSMA.The Phase I/II clinical trial of the successfully completed GC101-2 IND project isThe Phase III pivotal clinical trial has laid a solid foundation. Clinical trial data indicate that Type 2 SMA subjects treated with GC101 injection have shown positive and significant efficacy, with multiple subjects achieving breakthroughs in motor milestones. Compared to existing treatments, the therapeutic potential of GC101 injection — "single treatment, long-term efficacy" — demonstrates a differentiated advantage. Based on its outstanding performance, GC101 injection was included by the CDE in December 2024.Breakthrough TherapyDrug Catalog.

About Genecradle

Beijing Genecradle Therapeutics Co., Ltd. is a national high-tech enterprise with core business in the development of gene therapy drugs mediated by AAV vector delivery technology. It aims to advance China's rare disease gene therapies from basic research to clinical and market applications, benefiting patients and their families. The company focuses on developing gene therapy drugs for hereditary neuromuscular diseases, inherited metabolic disorders, lysosomal diseases, and ophthalmic conditions. By promoting the research, development, and clinical application of rare disease gene drugs, the company seeks to deepen the understanding of life and health, transitioning gene therapy technologies and products from rare diseases to the treatment and rehabilitation of chronic and other major diseases.

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