Dystrogen Therapeutics Investigational Chimeric Cell Therapy DT-DEC01 for the Treatment of Duchene Muscular Dystrophy Shows Safety and Functional Improvements
- Category: DNA RNA and Cells
- Published on Friday, 08 April 2022 11:06
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- Results continue to reinforce safety and tolerability profile of DT-DEC01
- First look at functional outcomes in low dose cohort found improvements 3 months after DT-DEC01 administration
- Therapy not dependent on the genetic mutation of the DMD patient, thus making DT-DEC01 a universal therapy for all DMD patients
MIAMI, FL, USA and WARSAW, Poland I April 7, 2022 I Dystrogen Therapeutics, Corp. the leader in chimeric cell therapies, today announced positive results from an ongoing study conducted in Poland under a hospital exemption protocol of DT-DEC01 (Dystrophin expressing chimeric (DEC) cells), the Company's investigational engineered cell therapy for Duchenne muscular dystrophy (DMD). Results include 1 to 3 month functional and safety data from three clinical trial participants in the low-dose cohort. During this period, no adverse events (AEs and SAEs) associated with DEC therapy were observed. DT-DEC01 is in development for the treatment of DMD, a devastating neuromuscular disease associated with a lack of dystrophin protein. DT-DEC01 is an engineered chimeric cell that engrafts in skeletal and cardiac muscle, delivering a full-length dystrophin gene and related components of a healthy muscle cell, the absence of which is closely associated with the progressive degeneration and a shortened lifespan characteristic of the disease.
"There are currently no approved treatments for people with DMD that result in a cure or significant attenuation of the disease – one that causes significant disability in boys and young men and usually leads to early mortality. It's very encouraging that we continue to see consistent, positive data from our investigational DT-DEC01 engineered cell therapy across several measures, as we know the community needs more options," said Maria Siemionow, M.D. Ph.D., Chief Scientific Officer, Dystrogen Therapeutics. "The improvements in functional measures at 1 and 3 months in participants from the low dose cohorts who received DT-DEC01 are distinctly different from what an age-matched, natural history group would predict with DMD. When coupled with the strong and sustained dystrophin expression results in preclinical studies and encouraging safety profile seen to date, today's results increase our confidence in DT-DEC01 and provide additional supportive evidence for this approach as we advance to the higher dose cohort into the next stage of clinical testing."
The Data and Safety Monitoring Board (DSMB) reviewed the low dose (2 million DEC cells per kg) clinical cohort data and gave a positive opinion on the safety of DT-DEC01 therapy. The DSMB recommend initiating the 4M/kg cohort.
Cohort 1 (low dose):
Patient 02B. (7-year-old ambulatory with deletion Exon 3-12) 3 months post treatment demonstrating improvement on a number of subjective and objective tests. Improved EMG (objective test) when compared to pretreatment baseline. Improved 6MWD, improved 10-meter walk/run time, grip strength and others. Increased step count via activity tracker.
Patient 03B. (15-year-old non-ambulatory with deletion Exon 48-50) 2 months post treatment, increased activity level when compared to baseline. Improved grip strength, improved spirometry, improved upper extremity strength.
Patient 04B. (6-year-old ambulatory with nonsense mutation), 1 month post treatment, increased step count via activity tracker when compared to baseline.
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 be delivered intraosseously and then systemically distributed to 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