Neuromuscular Disease Foundation announces 24 month plan toward human dosing

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The Neuromuscular Disease Foundation (NDF) announced today that they will be moving forward with a two year plan to achieve the human dosing phase of a new drug to treat the rare autosomal recessive genetic disease GNE Myopathy (“GNEM”), a progressive condition that causes deterioration of muscle tissues. GNEM is a genetic disease that exists in races and nationalities worldwide, with elevated carrier rates in certain populations of the Middle East, Eastern Europe and Asia.

NDF is a global leader in critical research focused on treatments and a cure for GNEM. The nonprofit has made swift and smart progress in its drug development process, by taking two major steps. First, NDF onboarded two world renowned scientific experts in
rare neuromuscular diseases, Doctors Angela Lek and Rich Horgan. Secondly, NDF established a strategic partnership with Charles River Laboratories, known for its expertise in expediting the path to IND and subsequent clinical trials.

Doctors Angela Lek and Rich Horgan Join NDF Dr. Angela Lek, Associate Research Scientist at Yale University’s School of Medicine,
and a part of Yale’s LEK LAB, has been appointed to be NDF’s next Scientific Director and, and will coordinate NDF’s continued work with its impressive global consortium of scientists. This year NDF has funded more studies than ever before, totaling over $700,000, all of which will help fulfill the necessary steps for gene therapy, the most promising method of treatment being investigated for this and other rare diseases. Dr. Lek has been part of the NDF team since 2017, having served as a scientific consultant.

She received her PhD in Cell/Cellular and Molecular Biology from the University of Sydney and is the recipient of many prestigious fellowships and awards from the Muscular Dystrophy Association, which honored her with the MDF Development Award,
the Hood Foundation and the American Australian Association.

NDF is also pleased to bring Rich Horgan on board as a special consultant tasked with guiding its gene therapy process. Horgan is the founder and president of Cure Rare Disease, a nonprofit biotech that develops custom-made drugs. He was named in Forbes’ “30 Under 30 to Watch,” and has been working on a Crispr-based therapy for his brother Terry, who has Duchenne muscular dystrophy.
His experience as the founder of Cure Rare Disease, with the biopharmaceutical industry, and his passion for transforming the paradigm of rare disease drug development, make him uniquely qualified to help NDF as it continues to move closer to making dosing patients a reality. Rich received his MBA from Harvard Business School, where he was a recipient of the Blavatnick Fellowship in Life Science Entrepreneurship.

NDF Partners with Charles River Laboratories
Charles River Laboratories International, a Biotech and contract research organization (CRO), specializes in preclinical and clinical laboratory, gene therapy and cell therapy services for the Pharmaceutical, Medical device and Biotechnology industries. By
bringing Charles River Labs into its drug development process, NDF will gain access to its extensive reach in the drug discovery and safety fields, to accelerate its progress toward a GNEM cure. With its globally recognized expertise in pharmaceuticals
development, safety and manufacturing, Charles River Labs is well positioned to help lead NDF through this exciting next phase of its scientific progress.

Doctor Lek said of this next step in delivering a GNEM cure “We at NDF are excited to embark on a partnership with regulatory and translational experts at Charles River Laboratories to expedite the development and testing of gene therapies for GNE myopathy.