Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing personalized therapies for rare genetic diseases.
The company was founded based on the pioneering work of Prof. Maria Siemionow, a world-renowned scientist and surgeon who led the team that performed the first near-total face transplantation in the United States. Professor Siemionow’s research focused on the creation of chimeric cells which have a role in modulation of the immune system's response to a transplant. This led to the development of Dystrophin Expressing Chimeric (DEC) cell therapy. DECs are engineered cells and belong to a family of therapeutic technologies called Advanced Therapeutics Medicinal Product (ATMP). Using Dystrogen’s patented cell engineering technology, DECs are made by combining a malfunctioning cell of the Duchenne patient with a normal, working cell from a healthy donor. This novel chimeric cell, is composed of both the donor’s and the recipient’s structures, and interacts with the surroundings like a normal cell. This offers a unique advantage and allows the patient's body and immune system to accept the chimeric cell without rejection. In such a way, we have created dystrophin producing cells that can engraft in the patient's muscles (such as heart, diaphragm, skeletal muscles) and, as demonstrated in our peer-reviewed publications, increase their dystrophin levels. Increased dystrophin levels have been shown to correlate with improved functional outcomes. This technology was originally developed at the University of Illinois in Chicago.
Dystrogen Therapeutics is currently focused on the development of a chimeric cell-based therapy platform. The company is testing dystrophin expressing chimeric (DEC) cells, cell-based therapy for the treatment of Duchenne muscular dystrophy.