Dystrogen Therapeutics

is a clinical stage regenerative medicine company focusing on rare diseases. The company is currently testing DEC therapy for Duchenne Muscular Dystrophy.

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About Dystrogen

Dystrogen Therapeutics was founded based on the decades long work of Professor Maria Siemionow, MD, PhD.

Professor Siemionow’s research focused on creating cell hybrids, referred to as chimeras. This has lead to the development of Dystrophin Expressing Chimeric (DEC) cell therapy. DECs work by combining a malfunctioning cell of the Duchenne patient with a working cell from a healthy donor. This offers a unique advantage and allows the patients’ body and immune system to accept the new cell without rejection. The new cell is composed of both donor and recipient structures and interacts with its surroundings like a normal cell. In such a way we have created dystrophin producing cells that can engraft inside the patient's 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.

How it Works
Human Cells Provide a Potential Therapy for Duchenne Muscular Dystrophy
New Hope for Treatment of Duchene Dystrophy

Dystrogen Therapeutics

Our mission is to advance treatments for patients with rare diseases.

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Dystrogen Therapeutics is a life science company focused on rare diseases. The company has developed dystrophin expressing chimeric (DEC) therapy. DEC therapy is a candidate for the treatment of Duchenne muscular dystrophy. The company is currently preparing to initiate a clinical pilot trial.

The Science

Dystrogen Science

In vivo experiments demonstrated that DEC therapy restored dystrophin expression in dystrophin-deficient hosts, and significantly improved muscle function at 90 days post intramuscular transplant.

Executive Team

Maria Siemionow, MD, PhD, Founder and Chief Scientific Officer

Dr. Siemionow spent decades investigating the effect of chimeric cells – made by fusing donor and recipient bone marrow stem cells – on the immune system’s natural attack response to foreign tissues and donor organs. If the immune system leaves these chimeric cells alone as part “self,” implanted donor tissue may be spared a full-blown rejection response. Dr. Siemionow is president of the American Society for Reconstructive Transplantation and past president of the International Hand and Composite Tissue Allotransplantation Society and the American Society for Peripheral Nerve.  She is a member of the academic-industry team known as the Warrior Restoration Consortium, which focuses on the development of clinical therapies and new treatments for wounded soldiers as part of the Armed Forces Institute for Regenerative Medicine. Dr. Siemionow received the Great Immigrants Award from the Carnegie Foundation. Dr. Siemionow serves on the editorial board of nine professional society journals and is an ad hoc reviewer for six professional society journals. She has published more than 320 scientific articles and has edited three plastic surgery textbooks, two popular science books, and has contributed to 58 published book chapters. She has appeared on the Oprah Winfrey Show, CNN with Sanjay Gupta, Good Morning America, and many others. In 2008 she led a team of surgeons at the Cleveland Clinic Foundation that performed the first face transplant in the United States.

Kris Siemionow, MD, Co-Founder and CEO

Dr. Kris Siemionow is an orthopaedics surgeon and serial entrepreneur. Dr. Siemionow completed residency at the Cleveland Clinic Foundation in orthopedic surgery and a spine surgery fellowship at Rush University Medical Center. Dr. Siemionow is co-founder of Global Spine Outreach a not for profit organization whose mission is to “save children with severe spinal deformities”. Dr. Siemionow co-founded Holo Surgical Inc, a medical technology company developing Synthetic Vision for Surgeons. Dr. Siemionow has published over 50 scientific articles and holds 11 patents in various medical applications.

Edyta Niemczyk PhD, Chief Operating Officer

Dr Edyta Niemczyk is an accomplished regulatory professional, scientist and consultant with a strong life science background and experience in technology transfer across diagnostics, pharma (metabolic diseases), cosmetics and medical products. Edyta holds a Master Degree in biotechnology and a PhD in molecular biology starting her career in science where she honed her skills in human clinical genomics as a postdoc, both, at the University of Liverpool and University of Birmingham, UK. Before joining Dystrogen Therapeutics, she was actively acting in clinical trials regulatory affairs as a Head of the Unit at the Department for Registration of Clinical Trials of Medicinal Products at the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products in Warsaw. There, she effectively managed a team who was responsible for assessment of submitted clinical trial applications in compliance with Polish and EU law. Prior to that, Edyta was consultant at LSBC where she built up considerable experience working with a portfolio of innovative life science projects to drive their potential to the investment stage by being responsible for scientific due diligence, IP, and regulatory affairs analysis, market research, and mentoring. She managed to successfully attract investments in diagnostic and dietary supplements products.

Martin Zajac - Financial Controller

Martin has over 10 years of experience in creation of financial models reflecting company operations, including large international public companies, as well as small innovative start-ups. Martin was previously the financial controler at Giza Polish Ventures. Martin holds a postgraduate diploma in Managment from the Warsaw School of Economics.

Advisory Board

Waldemar Priebe, PhD

Waldemar Priebe is a Founder of Moleculin( NASDAQ:MBRX). Dr. Priebe is also a Professor of Medicinal Chemistry at the Department of Experimental Therapeutics at The University of Texas MD Anderson Cancer Center. As a Founder or Founding Scientist at a number of successful biotechnology firms such as Aronex Pharmaceuticals(NASDAQ: ARNX, acquiered by Antigenics), Houston Pharmaceuticals, Reata Pharmaceuticals (NASDAQ: RETA), and IntertechBio, Dr. Priebe has been integral in advancing several drugs through the pipeline, three of which are currently in clinical development. He has also developed several new small molecule compounds that have been licensed as potential drugs.

News

Dr. Siemionow’s pioneering work featured in National Geographic

September, 2018

Human 'Chimeric' Cells Restore Crucial Protein in Duchenne Muscular Dystrophy

March 16, 2018

Chimeric’ Cells Restore Crucial Protein in Duchenne Muscular Dystrophy

March 16, 2018

UIC researchers launch company to develop ‘chimeric cell’ therapy for Duchenne muscular dystrophy

February 23, 2018