MilliporeSigma Announces Signing of MoU with Gene Therapy Research Institution 

• 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

MilliporeSigma, the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany, a leading science and technology company, announced that its Life Science business sector 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 MilliporeSigma’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, Process Solutions, Japan, Life Science business of Merck KGaA, Darmstadt, Germany. “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, MilliporeSigma will partner with GTRI to build a robust AAV production platform to scale up production. The collaboration will also include the use of MilliporeSigma’s cell line and platform technologies for developing viral vector-based gene therapy. Additionally, MilliporeSigma 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 MilliporeSigma will optimize this process and help bring therapies to market faster.”

Earlier this year, Merck KGaA, Darmstadt, Germany 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.