DEC / Dystrogen - LARVOL DELTA

Long-Term Safety and Efficacy of Systemic DT-DEC01 Cell Therapy in Non-Ambulatory Duchenne Muscular Dystrophy Patients: a 24-Month Clinical Evaluation. (PubMed, Stem Cell Rev Rep) - "This study evaluated the long-term safety and efficacy of DT-DEC01, a Dystrophin Expressing Chimeric (DEC) cell therapy, in non-ambulatory DMD patients following systemic intraosseous administration...Notably, these improvements occurred in non-ambulatory patients - a disease stage typically associated with progressive cardiac and pulmonary decline, rather than functional recovery. These sustained functional benefits up to 24 months suggest that DT-DEC01 therapy may promote multisystem functional improvements in advanced DMD, independent of dystrophin mutation type or disease stage."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Developing Advanced Chimeric Cell Therapy for Duchenne Muscular Dystrophy. (PubMed, Int J Mol Sci) - "There was no correlation found between the myoblast passage number and fusion efficacy. This study successfully optimized the myoblast fusion protocol for creation of human DEC cells, introducing DEC as a new Advanced Therapy Medicinal Product (ATMP) for DMD patients."

Journal • Metastases • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Mechanisms of Chimeric Cell Therapy in Duchenne Muscular Dystrophy. (PubMed, Biomedicines) - "We have developed Dystrophin Expressing Chimeric (DEC) cell therapy, DT-DEC01, by fusing human myoblasts from healthy donors and from DMD patients...The Pappenheim assay confirmed myotube formation in the final product. This study highlights the unique properties of DT-DEC01 therapy and their relevance to DMD treatment mechanisms."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Chimeric Cell Therapy Transfers Healthy Donor Mitochondria in Duchenne Muscular Dystrophy. (PubMed, Stem Cell Rev Rep) - "The colocalization of the mitochondrial dyes MitoTracker Deep Red and MitoTracker Green confirmed the mitochondrial chimeric state and the creation of chimeric mitochondria, as well as the transfer of healthy donor mitochondria within the created DEC cells. These findings are unique and significant, introducing the potential of DT-DEC01 therapy to restore mitochondrial function in DMD patients and in other diseases where mitochondrial dysfunction plays a critical role."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Metabolic Disorders • Muscular Dystrophy

Assessment of Motor Unit Potentials Duration as the Biomarker of DT-DEC01 Cell Therapy Efficacy in Duchenne Muscular Dystrophy Patients up to 12 Months After Systemic-Intraosseous Administration. (PubMed, Arch Immunol Ther Exp (Warsz)) - "A novel, DT-DEC01 therapy, consisting of Dystrophin Expressing Chimeric (DEC) cells created by fusing allogeneic myoblasts from normal donors with autologous myoblasts from DMD-affected patients, was assessed for safety and preliminary efficacy in boys of age 6-15 years old (n = 3)...EMG of selected muscles of both, ambulatory and non-ambulatory patients confirmed preliminary efficacy of DT-DEC01 therapy by an increase in motor unit potentials (MUP) duration, amplitudes, and polyphasic MUPs at 12 months. This study confirmed EMG as a reliable and objective biomarker of functional assessment in DMD patients after intraosseous administration of the novel DT-DEC01 therapy."

Biomarker • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Safety and Efficacy of DT-DEC01 Therapy in Duchenne Muscular Dystrophy Patients: A 12 - Month Follow-Up Study After Systemic Intraosseous Administration. (PubMed, Stem Cell Rev Rep) - "We previously reported the preliminary safety and efficacy up to six months after the administration of DT-DEC01, a novel Dystrophin Expressing Chimeric (DEC) cell therapy created by fusion of myoblasts of DMD patient and the normal donor...DT-DEC01 introduces a novel concept of personalized myoblast-based cellular therapy that is irrespective of the mutation type, does not require immunosuppression or the use of viral vectors, and carries no risk of off target mutations. This establishes DT-DEC01 as a promising and universally effective treatment option for all DMD patients."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up. (PubMed, Stem Cell Rev Rep) - "Following systemic-intraosseous administration, DEC engraft and fuse with the myoblasts of DMD patients, deliver dystrophin and improve muscle strength and function. (Created with BioRender.com)."

Journal • P1 data • Duchenne Muscular Dystrophy • Fatigue • Genetic Disorders • Muscular Dystrophy