Merck Signs MoU with Gene Therapy Research Institution to Accelerate Viral Vector-based Gene Therapy for Parkinson’s Disease

News Release

• One of first Sf-RVN^® Insect Cell Line applications for clinical stage gene therapy production
• Company to support safe and robust production of viral vectors
• Collaboration aims to bring new therapies to market faster

Tokyo, Japan, July 11, 2024 - Merck, a leading science and technology company, has signed a non-binding Memorandum of Understanding (MoU) with Gene Therapy Research Institution Co., Ltd. (GTRI), a Japanese clinical-stage biotech start-up specializing in gene therapy using adeno-associated virus (AAV) vectors. GTRI will utilize Merck’s Sf-RVN^® Insect Cell Line platform for the Good Manufacturing Practices (GMP) production of its viral vector-based gene therapy for Parkinson’s Disease.

“In Japan, clinical trials of gene therapy medicines are underway in the hope of developing new treatments to fight and cure diseases,” said Isao Hatano, Head of Process Solutions, Japan, Life Science business of Merck. “With our broad portfolio of products and services, we can better equip and support GTRI in accelerating process development and manufacturing of viral vector-based gene therapy. We believe this collaboration will ultimately help save many patients suffering from serious diseases.”

As part of the MoU, Merck will partner with GTRI to build a robust AAV production platform to scale up production. The collaboration will also include the use of Merck’s cell line and platform technologies for developing viral vector-based gene therapy. Additionally, Merck will provide GTRI with technical support, training and process optimization for process development at the M Lab™ Collaboration Center in Tokyo.

“Since 2014, we have been working to bring our unique gene therapy techniques to refractory diseases where effective therapies have not been established, primarily focusing on Parkinson's disease and Amyotrophic Lateral Sclerosis (ALS),” said Katsuhito Asai, CEO, GTRI. “Our biggest challenge is developing robust and safe manufacturing processes for viral vectors used to introduce DNA into patients, especially on a large scale. Producing viral vectors is much more complex and larger in scale than making typical antibody drugs. Our partnership with Merck will optimize this process and help bring therapies to market faster.”

Earlier this year, Merck announced a partnership with The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance research aimed at slowing progression of Parkinson’s disease.

About Gene Therapy Research Institute Co., Ltd.
Gene Therapy Research Institution Co., Ltd. (GTRI) is a Japanese clinical-stage biotech start-up specializing in gene therapy using adeno-associated virus (AAV) vectors. Established in 2014, GTRI aims to realize the innovative research in practice, in the fastest way from basic research to clinical studies with the goal of popularizing gene therapy. https://www.genetherapy-ri.com/en/