SK Pharmteco and Genethon Advance DMD Gene Therapy with French Manufacturing Milestone
Event summary
- SK Pharmteco's French subsidiary Yposkesi has manufactured 20 batches of AAV8 micro-dystrophin for Genethon’s GNT-0004 DMD gene therapy, including 18 cGMP-compliant batches at a 400 L scale.
- The collaboration supported the launch of a pivotal Phase III clinical trial in Europe and the UK, authorized by EMA and MHRA in 2025.
- Long-term data from the ASGCT 2026 meeting showed sustained efficacy and favorable safety profile for GNT-0004 at two years post-treatment.
The big picture
This collaboration highlights SK Pharmteco's growing role as a key player in viral vector manufacturing, particularly for complex AAV programs. The success of this partnership positions the company to support additional late-stage gene therapy programs in Europe and beyond, while Genethon's progress with GNT-0004 could set new benchmarks for DMD treatments.
What we're watching
- Regulatory Momentum
- Whether the EMA and MHRA approvals will accelerate broader regulatory acceptance of AAV8-based gene therapies.
- Manufacturing Scale-Up
- The pace at which SK Pharmteco can transition from clinical supply to commercial-scale production for GNT-0004.
- Therapeutic Efficacy
- How the lower dose of GNT-0004 will compare in efficacy and safety against other DMD gene therapy candidates.