Drug intelligence / Profile preview

DT-DEC01

Development stage
Unknown
Lead developer
Dystrogen Therapeutics
Modality
Cell Therapies
Administration
Intraosseous
01

Overview

DT-DEC01 is a novel dystrophin expressing chimeric (DEC) cell therapy developed for the treatment of Duchenne muscular dystrophy (DMD). It is created by ex-vivo fusion of allogeneic human myoblasts (from a healthy donor) and autologous myoblasts (from the DMD patient) to generate chimeric cells capable of expressing normal dystrophin. This therapy is administered via intraosseous infusion into the bone marrow compartment. Unlike other gene or cell therapies, DT-DEC01 does not require viral vectors or genetic modification, and it does not provoke an immune response, negating the need for immunosuppression. Preclinical and pilot human studies demonstrate DT-DEC01’s long-term safety (no adverse events or immune reactions for up to 24 months) and preliminary efficacy in improving muscle strength, motor function, cardiac and respiratory parameters, across DMD patients regardless of gene mutation or ambulatory status[1][3][6][8].

Other names
Dystrophin Expressing Chimeric cell therapyDEC cell therapy
02

Targets

DMD (Dystrophin)

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