SK pharmteco Advances AAV Gene Therapy to Phase III with 20 Clinical Batches for Genethon DMD Program
- SK pharmteco's Yposkesi French facility has manufactured 20 AAV8 batches (18 cGMP) at 400 L scale to support Genethon's GNT-0004 DMD gene therapy through Phase III authorization by EMA and MHRA in 2025.
- The partnership demonstrates viral vector CMC, analytical validation, and clinical supply capabilities; SK pharmteco is now manufacturing additional batches while preparing commercial-scale documentation and process validation.
- Long-term efficacy and safety data presented at ASGCT 2026 showed sustained improvements in muscle function over two years, supporting continued enrollment in the 72-patient Phase III trial.
Operational Impact
SK pharmteco's demonstrated ability to manufacture 400 L cGMP AAV batches and transition from clinical to commercial-scale documentation strengthens its competitive position in viral vector CDMO services for late-stage gene therapy programs in Europe and beyond.
Source: PharmaSource RSS