Скачать или смотреть Regenerative Therapies for Muscular Dystrophy - Douglas Falk | Maryland Stem Cell Research Fund

Muscular dystrophy is a group of 30+ diseases that is characterized by loss of muscle mass referred to as muscle wasting. A common form of muscular dystrophy is Limb-girdle muscular dystrophy (LGMD), which is a diverse subgroup of genetic disorders that usually manifests as weakness in the muscles around the hips and shoulders.

In the US alone, there are approximately 4000 patients with up to 100 new patients identified each year. The onset and progression of LGMD varies and can manifest during adulthood and childhood. In most cases, LGMD is associated with early disability, such as difficulty climbing stairs and walking, resulting in many patients being wheelchair-bound. The current focus is on disease management to improve the patient’s quality of life, with no disease-modifying treatments available.

Vita Therapeutics is a cell engineering company, founded at Johns Hopkins University in 2019 that seeks to develop life-transformative treatments. The company utilizes induced pluripotent stem cell (iPSC) technology to develop regenerative therapies designed to support muscle regeneration and treat patients with muscle disorders.

Vita is getting ready to launch its first-in-human clinical trial to evaluate its leading product, VTA-100, for the treatment of LGMD 2A/R1. VTA-100 is designed as an autologous treatment that combines gene correction and iPSC technology to help repair and replace muscle cells. It is envisioned that transplanted Vita Therapeutics’ muscle stem cells (satellite cells) will not only replace and repair damaged muscle tissue but also, upon homing to the satellite cell niche in a quiescent state, support long-lasting homeostasis, and repair of future muscle damage.

Douglas Falk, Vita’s CEO, credits MSCRF with providing support early on to enable the development and preclinical testing of its muscle satellite cells, which led to the development of its lead product, VTA-100.

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