CRISPR collaboration will advance cell therapy for muscular dystrophy

CRISPR gene editing

A new licensing agreement between Vita Therapeutics and MilliporeSigma (the US and Canada Life Science business of Merck) will see Vita leverage MilliporeSigma’s foundational CRISPR patents to advance its preclinical asset VTA-100 for limb-girdle muscular dystrophy (LGMD2A).

VTA-100 utilises this CRISPR genome editing technology to insert a functional copy of the Calpain 3 (CAPN3) gene, which is mutated in patients with LGMD2A, into Human Satellite Cells, generated using Vita’s proprietary production technology, for use as a cell therapy.

‍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.

“For people with limb-girdle muscular dystrophy, the CAPN3 gene is mutated in satellite cells,” said Douglas Falk, 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.”

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