By Fineline Cube 
China-based Gene Cradle has received approval from the National Medical Products Administration (NMPA) to proceed with a pivotal Phase III study for its adeno-associated virus (AAV)-based gene therapy GC101 in type II 5q spinal muscular atrophy (SMA). This marks a significant milestone in the development of this novel therapeutic approach.
GC101: First AAV Gene Therapy for SMA in China
GC101 is the first AAV gene therapy in China administered via a single intrathecal injection for the treatment of SMA. It is designed to address type I, II, and III 5qSMA. Previous Phase I/II clinical trials demonstrated significant therapeutic effects in type II SMA patients, showcasing differentiated advantages over existing treatments. Notably, multiple subjects achieved milestone breakthroughs in mobility.
Clinical Recognition and Advancement
GC101 was awarded Breakthrough Therapy Designation by the NMPA in December last year, underscoring its potential to address unmet medical needs in SMA treatment. The initiation of the Phase III study brings this innovative therapy one step closer to potential commercialization and broad availability for patients.-Fineline Info & Tech