Dystrogen Therapeutics Announces First in Man Dosing of Novel Chimeric Cell Therapy for Duchenne Muscular Dystrophy and Reports 6-week Clinical Outcomes
- Category: DNA RNA and Cells
- Published on Wednesday, 05 January 2022 09:35
- Hits: 765
MIAMI, CA, USA I January 4, 2022 I Dystrogen Therapeutics Corp. a clinical-stage company developing chimeric cell therapeutics to modify the course of rare diseases, today announced dosing of the first patient in its Phase 1 Human Pilot Clinical Study of DT-DEC01, Dystrophin Expressing Chimeric cells, or DEC, for the treatment of Duchenne Muscular Dystrophy (DMD).
The Phase 1 Human Pilot Study is a non-randomized trial enrolling boys between the ages of 5 and 18. The trial investigates the safety, tolerability, and efficacy of DT-DEC01. The pilot trial will enroll a total of 10 patients with DMD across 3 escalating doses. The study will also assess NorthStar Ambulatory Assessment (NSAA), range of motion (ROM), performance of upper limb (PUL), 6MWD, Muscle strength by myometry and 5-grade Lovett scale, and cardiac muscle evaluation by ECHO.
The first patient, a 6-year-old boy with deletion of exons 3-12, received an infusion of DT-DEC01 on November 26th, 2021. The patient tolerated the procedure well and was discharged home. At day 7, day 28, and 6 week follow up, the patient demonstrated no side effects from the treatment. Functional outcomes, including ECHO, demonstrated a non-clinically significant improvement when compared to pretreatment baseline. Subjective parent reported assessment of post - treatment daily activity level demonstrated a significant improvement when compared to pretreatment baseline at 6 weeks. Subjective therapist reported assessment of post - treatment quality of motion as well as patient performance during functional assessment demonstrated a significant improvement when compared to pretreatment baseline at 6 weeks. At 6 weeks, laboratory data demonstrated no signs of inflammation, infection, antibody formation, or other adverse systemic effects on end organs.
Screening and enrollment of patients is expected to continue over the course of the next 12 months in the European Union, with data from this trial expected once patients have been evaluated for one full year post-treatment.
"We are very pleased to begin dosing patients in our pilot study. This is the first time we will assess our chimeric cell therapy in Duchenne patients, which will provide us not only with safety data but could also give us an early look into the potential efficacy of this treatment in possibly halting disease progression and even partly restoring functional loss in boys with muscular dystrophy", said Kris Siemionow, MD, PhD Founder and Chief Executive Officer of Dystrogen Therapeutics.
"This is an important step toward bringing the promise of this novel technology to patients with serious diseases like DMD, and we are thrilled to be leading the effort in what we believe may be a fundamental change in the treatment of debilitating disorders", said Maria Siemionow, MD, PhD Founder and Chief Scientific Officer of Dystrogen Therapeutics.
DT-DEC01 is a chimeric cell therapy. The advanced therapeutic medicinal product (ATMP) is made using Dystrogen's proprietary cell engineering technology which creates a DEC cell. Clinically, DEC cells have been shown to express CD56 at significantly higher levels than myoblasts from Duchenne patients. DEC cells express favorable HLA characteristics which carries multiple advantages. In preclinical studies, DEC cells have also been shown to express clinically significant levels of dystrophin when compared to controls. DEC cell therapy demonstrated significant functional improvement in cardiac, diaphragm, and other skeletal muscle strength and associated function in preclinical trials. Because DEC therapy is designed to prevent triggering an immune system response, a major advantage of DEC therapy is that it does not require immunosuppression. The therapy is not associated with any genetic manipulation and therefore involves no risk of off target mutation, does not use viral vectors, and its use is not dependent on the genetic mutation of the DMD patient, thus making DEC a universal therapy for all DMD patients.
About Dystrogen Therapeutics Corp
Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing 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 initially 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 that is designed to prevent the immune system from attacking the chimeric cells. DECs are engineered cells and belong to a family of therapeutic technologies called Advanced Therapy Medicinal Products (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 cell structures but looks to the patient's immune system like his own cell and thus does not trigger an immune response while it functions (i.e. produces dystrophin) like the patients' 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, Dystrogen has created dystrophin producing cells that can engraft in the patient's muscles (such as heart, diaphragm, skeletal muscles) and, as demonstrated in our research and related peer-reviewed publications, increase their dystrophin levels. Increased dystrophin levels have been shown to correlate with improved functional outcomes which was confirmed in preclinical studies of DEC.
SOURCE: Dystrogen Therapeutics