CMTA and ToolGen Announce Collaboration to Develop Potential Treatment for CMT1A Using Gene Therapy

GLENOLDEN, PA — The Charcot–Marie–Tooth Association (CMTA) and ToolGen announced a collaboration to investigate a potential therapy for CMT type 1A (CMT1A), the most common subtype of the disease, which affects approximately 1.5 million people, using CRISPR gene-editing technology.

The primary goal of the collaboration is to facilitate the development of a novel gene-editing therapy for CMT1A that seeks to suppress overactivity of the causative disease gene, PMP22.

The CMTA’s Strategy to Accelerate Research (STAR) connects leading CMT clinicians and researchers with pharmaceutical partners committed to developing treatments and a cure for CMT. Strategic alliances with pharmaceutical partners like ToolGen support drug development efforts to deliver therapies to CMT patients.

“We are excited to establish this partnership with ToolGen as they work to advance gene-editing technology to treat CMT1A,” said CMTA’s CEO Amy Gray. “Developing strategic research partnerships with companies like ToolGen is a central part of our strategy to accelerate treatments for the CMT community. Since launching STAR 12 years ago, the CMTA has been able to establish research partnerships with almost 40 pharmaceutical and biotech companies, and leading research labs around the world.”

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The testing alliance with ToolGen involves access to the STAR Scientific and Clinical advisors of the CMTA, animal testing of the approach in the CMTA preclinical testing alliance network, and assistance with the planning of clinical trials in the USA. The CMTA’s preclinical testing alliance: www.cmtausa.org/our-research/for-researchers/cmta-preclinical-testing-network/ makes this possible.

“We are delighted to have this collaboration to strengthen our drug development program for CMT1A where no approved therapy is available,” said ToolGen’s Director of R&D Therapeutics Jae young Lee. “This alliance with the Charcot-Marie-Tooth Association will enable us thorough testing of our drug candidate in suitable CMT1A models and lead us closer to initiating clinical trials.”

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