Vita Therapeutics Collaborates with MilliporeSigma to Advance Preclinical Program for Vita’s Preclinical Cell Therapy for Limb-Girdle Muscular Dystrophy (LGMD2A)

Licensing agreement utilizing MilliporeSigma’s CRISPR genome-editing technology supports the advancement of Vita’s pipeline
Baltimore, MD, July 18, 2023 Vita Therapeutics, Inc. today announced a licensing agreement between Vita and MilliporeSigma, the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany. Under the agreement, Vita will leverage MilliporeSigma’s foundational CRISPR patents to advance the development of its preclinical asset VTA-100 for the treatment of limb-girdle muscular dystrophy (LGMD2A). Financial and other terms of the agreement were not disclosed.

“Our purpose is to impact life and health with science, empowering customers to deliver breakthroughs such as Vita's impactful, health-advancing research,” said Christopher Arnot, Director of Licensing and Business Development, Gene Editing, MilliporeSigma.  

“For people with limb-girdle muscular dystrophy, the calpain 3 (CAPN3) gene is mutated in satellite cells,” said Douglas Falk, MS, Chief Executive Officer at Vita Therapeutics. “MilliporeSigma’s proprietary CRISPR genome-editing technology will allow us to insert a functional copy of the gene into those cells, taking us an important step forward in advancing the development of VTA-100 as a potential cell therapy.”

About Limb-Girdle Muscular Dystrophy
Limb-girdle muscular dystrophy (LGMD) is a group of disorders that cause weakness and wasting of muscles closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. The severity, age of onset, and disease progression of LGMD vary among the more than 30 known sub-types of this condition and may be inconsistent even within the same sub-type. As the atrophy and muscle weakness progresses, individuals with LGMD begin to have trouble lifting objects, walking, and climbing stairs, often requiring the use of assistive mobility devices. There is currently no cure for LGMD, with treatments limited to supportive therapies such as corticosteroids.

About Vita Therapeutics
Vita Therapeutics is a biotechnology company developing state-of-the-art cellular therapeutics for the treatment of debilitating neuromuscular diseases and cancers. Vita Therapeutics uses induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients. The Company is progressing its lead program VTA-100 for the treatment of limb-girdle muscular dystrophy (LGMD2A) with the goal of filing Investigational New Drug Applications with the U.S. Food and Drug Administration in the next 18 months. Long term, the Company is developing its pipeline of cellular therapies following a dual development strategy beginning with autologous-derived cells before moving to a universal hypoimmunogenic cell line. Vita Therapeutics is currently working with numerous partners, including PanCella, Wyss Institute, and Johns Hopkins University, to advance their clinical programs. Learn more about the company at

About the Life Science business of Merck KGaA, Darmstadt, Germany
The Life Science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has more than 28,000 employees and more than 55 total manufacturing and testing sites worldwide, with a portfolio of more than 300,000 products focused on scientific discovery, biomanufacturing and testing services. Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and electronics.  

More than 64,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2022, Merck KGaA, Darmstadt, Germany, generated sales of € 22.2 billion in 66 countries.  

The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as MilliporeSigma in life science, EMD Serono in healthcare and EMD Electronics in electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. For more information about Merck KGaA, Darmstadt, Germany, visit

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Media Inquiries for Vita
Mike Beyer
Sam Brown Inc. Healthcare Communications

Media Inquiries for MilliporeSigma
Livia Guadaim
Millipore Sigma