NTrans Technologies (NTrans), a leader in gene editing technologies, announced a Series A investment of 2 million Euros, led by Essential Investments, together with BOP Capital, Utrecht Health Seed Fund, and Starfish Innovations. Through this investment, NTrans aims to advance the development of its gene editing-based therapy for Duchenne Muscular Dystrophy (DMD). The funds will be used to demonstrate preclinical efficacy and safety of the therapy in DMD disease models, in preparation for its progression to the clinic.<\/p>\n\n\n\n
NTrans, a spin-off of the Hubrecht Institute of the Netherlands Royal Academy of Arts and Sciences, is a leader in developing novel gene editing technologies for the treatment of inheritable diseases and cancer. Its proprietary therapeutic platform to treat genetic muscle disease, triggers a cell\u2019s natural uptake mechanism to safely deliver gene editing systems, like CRISPR-Cas, into the target cell, where it efficiently corrects the function of the defective gene.<\/p>\n\n\n\n
Duchenne Muscular Dystrophy (DMD) is a devastating genetic disease characterized by progressive muscle degeneration, for which there is no cure. Onset occurs usually in boys between the ages of 2 and 3 years, whose average life expectancy is 26 years. DMD is caused by mutations in the dystrophin gene and affects all voluntary muscles in the body. NTrans\u2019 iTOP DMD gene editing treatment will be used to preserve the critical arm function in DMD patients. Sustained upper extremity function is extremely important as it allows a patient to independently control his wheelchair, computer, phone and feed himself. It preserves a great level of independence and thereby significantly enhances the quality of life. NTrans aims to offer a treatment that will permanently repair the dystrophin gene mutation with a single intramuscular administration.<\/p>\n\n\n\n