Dystrogen Therapeutics announced positive results for patients with Duchenne muscular dystrophy (DMD) from an ongoing investigation in Poland as part of the dystrophin expressing chimeric (DEC) protocol known as DT-DEC01.
The trial assessed 1-3 month functional results and safety data in a low-dose cohort, in which no adverse events were observed. The treatment engrafts skeletal and cardiac muscle tissue and delivers a full-length dystrophin gene along with associated components of a healthy muscle cell.
“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,” said Maria Siemionow, MD, PhD, chief scientific officer of Dystrogen Therapeutics.
“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.”
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DT-DEC01 is based on Dystrogen’s engineered DEC cells, which express CD56 at significantly higher rates than do cells of patients with DMD. The therapy does not trigger an immune response, meaning it requires no immunosuppression.
The Data and Safety Monitoring Board has a positive opinion on the safety of the low dose DT-DEC01 therapy (2 million DEC cells per kg), recommending further study in a new cohort on 4 million DEC cells per kg. The DT-DEC01 treatment is not genetic-mutation dependent and involves no genetic manipulation, making it universally applicable to all patients with DMD.
Dystrogen Therapeutics investigational chimeric cell therapy DT-DEC01 for the treatment of Duchene muscular dystrophy shows safety and functional improvements. Dystrogen Therapeutics; News release. April 7, 2022.