About / Our mission

Our mission is to create new personalized therapies for rare genetic diseases.

Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing personalized therapies for rare genetic diseases. The company has two technology platforms, which focus on treating patients with rare diseases such as Duchenne muscular dystrophy, sickle cell anemia, and neurodegenerative disorders such as Huntington’s disease.

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. This led to the development of Dystrophin Expressing Chimeric (DEC) cell therapy. DECs work 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 and the recipient 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 the patient's muscles (such as heart, diaphragm, skeletal muscles) and increase their dystrophin levels. Increased dystrophin levels have been shown to correlate with improved functional outcomes. This technology was developed at the University of Illinois in Chicago.

Dystrogen Therapeutics is currently focused on the development of two therapeutic platforms: a cell-based therapy platform and gene therapy platform. The company is testing dystrophin expressing chimeric (DEC) cells, a cell-based therapy for the treatment of Duchenne muscular dystrophy. Dystrogen’s RNAi platform focuses on trinucleotide repeat disorders such as Huntington's, Machado-Joseph disease, or Spino-Cerebellar Ataxia.