Dystrogen Therapeutics’ Investigational DT-DEC01 Chimeric Cell Therapy for the Treatment of Duchene Muscular Dystrophy Shows Improvements in Safety and Functionality
Category: DNA RNA and Cells
Posted on Friday 08 April 2022 11:06
- Results continue to strengthen the safety and tolerability profile of DT-DEC01
- The first review of functional outcomes in the low-dose cohort revealed improvements 3 months after administration of DT-DEC01
- Therapy independent of 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 waiver protocol of DT-DEC01 (chimeric cells expressing the dystrophin (DEC)), the company’s experimental cell therapy for Duchenne muscular dystrophy (DMD). The results include functional and safety data over 1 to 3 months from three clinical trial participants in the low-dose cohort. During this period, no adverse events (AEs and SAEs) associated with DEC treatment were observed. DT-DEC01 is being developed for the treatment of DMD, a devastating neuromuscular disease associated with a lack of the protein dystrophin. DT-DEC01 is a modified chimeric cell that engrafts into skeletal and cardiac muscle, delivering a full-length dystrophin gene and related components of a healthy muscle cell, the absence of which is strongly associated with progressive degeneration and a shortened lifespan characteristic of the disease.
“There is currently no approved treatment for people with DMD that results in cure or significant reduction of the disease – a disease that causes significant disability in boys and young men and usually results in early mortality. It is very encouraging that we continue to see consistent results, positive data from our investigational cell therapy DT-DEC01 on multiple metrics, as we know the community needs more options,” said Maria Siemionow, MD Ph.D. ., Scientific Director of Dystrogen Therapeutics. “Improvements in functional measures at 1 and 3 months in participants of the low-dose cohorts who received DT-DEC01 are markedly different from what a matched natural history group according to age would predict with DMD. When combined with the strong and sustained expression of dystrophin results in preclinical studies and an encouraging safety profile seen to date, today’s results reinforce our confidence in DT-DEC01 and provide additional evidence supporting this approach as we move to the higher dose cohort in the next stage of clinical testing.
The Data and Safety Monitoring Board (DSMB) reviewed the data from the low dose clinical cohort (2 million DEC cells per kg) and issued a positive opinion on the safety of DT-DEC01 therapy. The DSMB recommends launching the 4M/kg cohort.
Cohort 1 (low dose):
Patient 02B. (7 year outpatient with Exon 3-12 deletion) 3 months post treatment showing improvement on a number of subjective and objective tests. Improvement in EMG (objective test) compared to baseline before treatment. Improved 6MWD, improved 10-meter walk/run time, grip strength and others. Increased number of steps via activity tracking.
Patient 03B. (Non-ambulatory age 15 with Exon 48-50 deletion) 2 months after treatment increased activity level from baseline. Improved grip strength, improved spirometry, improved upper limb strength.
Patient 04B. (outpatient 6-year-old child with nonsense mutation), 1 month after treatment, increase in number of steps via activity monitoring compared to baseline.
DT-DEC01 is a chimeric cell therapy. The Advanced Therapeutic Drug (ATMP) is made using Dystrogen’s proprietary cell engineering technology that creates a DEC cell. Clinically, DEC cells have been shown to express CD56 at significantly higher levels than Duchenne patient myoblasts. DEC cells express favorable HLA characteristics that have multiple advantages. In preclinical studies, DEC cells have also been shown to express clinically significant levels of dystrophin compared to controls. DEC cell therapy has demonstrated significant functional improvement in cardiac, diaphragm, and other skeletal muscle strength and related function in preclinical trials. Because DEC therapy is designed to prevent an immune system response from being triggered, a major advantage of DEC therapy is that it does not require immunosuppression. The therapy is not associated with any genetic manipulation and therefore does not involve any risk of off-target mutation, does not use viral vectors and its use does not depend on the genetic mutation of the DMD patient, thus making DEC a therapy Universal for all DMD patients.
About Dystrogen Therapeutics Corp.
Dystrogen Therapeutics is a clinical-stage life sciences company engaged in the development of therapies for rare genetic diseases. The company was founded on the pioneering work of Professor Maria Siemionow, a world-renowned scientist and surgeon who led the team that performed the first near-total face transplant in the United States. Professor Siemionow’s research initially focused on creating chimeric cells that play a role in modulating the immune system’s response to a transplant. This led to the development of dystrophin-expressing chimeric cell therapy (DEC) which is designed to prevent the immune system from attacking 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 defective cell from the Duchenne patient with a normal, functioning cell from a healthy donor. This new chimeric cell is composed of both donor and recipient cell structures, but resembles the patient’s immune system as its own cell and therefore does not trigger an immune response while it is functioning (i.e. that it produces dystrophin) like the patient’s normal cell. This offers a unique advantage and allows the patient’s body and immune system to accept the chimeric cell without rejection. In this way, Dystrogen has created dystrophin-producing cells that can be delivered intraosseously and then distributed systemically to be transplanted into the patient’s muscles (such as heart, diaphragm, skeletal muscles) and, as our research and related peer-reviewed publications demonstrate, increase their dystrophin levels. Increased dystrophin levels have been shown to correlate with improved functional outcomes, which has been confirmed in preclinical studies on DEC.
THE SOURCE: Therapeutic Dystrogen