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 on chimeric cells therapy for transplantation tolerance, developed by Professor Maria Siemionow MD, PhD, a world renowned scientist and surgeon who led a team of surgeons that performed the first near-total face transplantation in the United States. Our technologies are developed in collaboration with the University of Illinois at Chicago and the Institute of Bioorganic Chemistry, Polish Academy of Sciences in Poland.
- Press Releases:
- September 12, 2019: UIC today - Human ‘chimeric’ cells restore crucial protein in Duchenne muscular dystrophy
- April 26, 2019: Positive Opinion from Ethics Committee to perform a First-in-Human trial (pdf) - Duchenne therapy
- April 16, 2019: huntingtonsdiseasenews.com - Dystrogen Therapeutics Gene Therapy Shows Promise for Huntingtons (pdf)
- April 05, 2019: Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington's Disease (pdf)
- In 2019, Dystrogen will be present at the following conferences:
- Bio International Convention
June 3-5 2019, Philadelphia, PA
Come meet us at Booth no. 4133
- Bio International Convention
- Duchenne Therapy: 2019 The Muscular Dystrophy Association (MDA) Clinical and Scientific Conference
April 13-17 2019, at the Hyatt Regency, Orlando.
At the conference we will present the newest data from our pre-clinical studies related to systemic delivery of dystrophin expressing chimeric cells “DEC” and how they circumvent the immune response and improve a number of functional characteristics. The new results confirm the feasibility and efficacy of chimeric cell therapy and represent a novel satellite cell-based approach for the treatment of DMD.
Conference poster: Systemically delivering dystrophin expressing chimeric (DEC) cells circumvents an immune response and improves various functional characteristics.(pdf)
- RNAi Platform: The 5th Animal Models of Neurodegenerative Diseases
15-18 September 2019, Chateau Liblice, Czech Republic.
At the conference Prof. Marta Olejniczak will present „UNIVERSAL RNAi TRIGGERS FOR SPECIFIC INHIBITION OF MUTANT HUNTINGTIN, ATAXIN-3, ATAXIN-7 AND ATROPHIN-1 EXPRESSION” in session Neurological Disease, Perspectives from Mammalian cells and Mouse models.
- RNAi Platform: The 44th FEBS Congress, Federation of European Biochemical Societies (FEBS)
6-11 July 2019, Krakow, PL
- RNAi Platform: The 24th Annual Meeting of the RNA Society
11-16 June 2019, Krakow, PL
Scientist from Dystrogen will present genetic tools used for silencing the expression of mutated genes containing expanded CAG repeats targeting short hairpin RNAs used to reduce the level of mutant huntingtin. Presented tools may serve as a universal allele – selective reagents for polyQ diseases such as: Huntington’s disease (HD), spinocerebellar ataxias (SCAs) and dentatorubral-pallidoluysian atrophy (DRPLA).
- Peer-Reviewed Publications on DEC
- Creation of Dystrophin Expressing Chimeric Cells of Myoblast Origin as a Novel Stem Cell Based Therapy for Duchenne Muscular Dystrophy
- Dystrophin Expressing Chimeric (DEC) Human Cells Provide a Potential Therapy for Duchenne Muscular Dystrophy
- Peer-Reviewed Publications on RNAi