- SK pharmteco highlighted its ongoing manufacturing partnership with Genethon, supporting the development and production of GNT-0004, an investigational AAV8-micro-dystrophin gene therapy for Duchenne muscular dystrophy (DMD).
- The collaboration has produced 20 AAV8 batches, supported Phase I/II studies, and enabled the launch of a Phase III clinical trial in Europe and the UK, while SK pharmteco continues manufacturing clinical supply for the study.
SK pharmteco has announced details of its ongoing strategic manufacturing partnership with Genethon to support the development of GNT-0004, an investigational AAV8-micro-dystrophin gene therapy for Duchenne muscular dystrophy (DMD). Through its French viral vector manufacturing operations, Yposkesi, an SK pharmteco company, has provided viral vector manufacturing, chemistry, manufacturing and controls (CMC) activities, analytical validation, and clinical supply to support the program’s progression from Phase I/II studies to a pivotal Phase III clinical trial. The collaboration demonstrates the company’s CDMO and contract manufacturing capabilities in viral vector production.
The partnership has resulted in the manufacture, testing, and release of 20 batches of AAV8 micro-dystrophin at a 400 L scale, including 18 current Good Manufacturing Practice (cGMP) batches. The companies also completed master and working cell banks, stability studies, and validation of analytical methods. According to the announcement, these activities supported regulatory submissions that enabled the Phase III trial in Europe and the UK, authorized in 2025 by the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA).
The ongoing randomized, placebo-controlled, double-blind, multicenter Phase III study is expected to enroll 72 ambulatory boys aged 6 to 10 years with DMD. SK pharmteco said it is manufacturing additional clinical batches for the study while preparing documentation and process validation activities to support future commercial manufacturing. The company also stated that long-term data presented at the 2026 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting demonstrated sustained efficacy, improvements in muscle function, and a favorable safety profile after two years in the first patients treated with GNT-0004.
“Our partnership with Genethon embodies SK pharmteco’s core mission: translating complex scientific innovation into reliable, scalable therapies. This program demonstrates our ability to support complex AAV manufacturing not only through pivotal clinical supply, but also through the quality, technical, and operational readiness required for future commercial manufacturing. It also reinforces the strength of our viral vector capabilities in France as part of the broader SK pharmteco viral vector organization.”
Joerg Ahlgrimm, CEO of SK pharmteco
The companies said the collaboration also highlights the continued expansion of SK pharmteco’s viral vector manufacturing operations in France, positioning the facility to support additional late-stage viral vector programs in Europe and other regions.
