Cell therapy is an innovative medical approach involving the transfer of cells into a patient to treat or manage a disease. Many types of cells have the potential for therapeutic usei – including in the treatment of Duchenne muscular dystrophy.ii
While traditional medicines are often made of chemicals or proteins, cell therapy uses living cells as the “active ingredient.” The process often involves isolating a specific cell type, expanding it in a laboratory setting, and administering it to a patient.iii These cells can be sourced from the patient’s own body (autologous) or from a donor (allogeneic).i
An allogeneic cell therapy is being investigated as a treatment for Duchenne muscular dystrophy.ii This cell therapy is generated from donated heart cells. Once administered, the cell therapy releases exosomes (small delivery vesicles) and growth factors (signaling molecules), which can reduce inflammation and fibrosis (scarring)ii,iv – key drivers of muscle damage in Duchenne.v
This cell therapy is administered by intravenous infusion once every three months and can be given to Duchenne patients regardless of their genetic mutation.ii Results from a Phase 3 clinical trials have shown evidence that this cell therapy can slow the decline of upper limb function, with a safety profile similar to placebo.ii
i National Center for Advancing Translational Sciences. Cell therapy. In: NCATS Toolkit for Patient-Focused Therapy Development Glossary. U.S. Department of Health and Human Services; Accessed July 29, 2026. https://toolkit.ncats.nih.gov/glossary/cell-therapy/
ii McDonald CM, Villa C, Soslow JH, et al. Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. Lancet. Published online July 29, 2026. doi:10.1016/S0140-6736(26)01385-1
iii El-Kadiry AE, Rafei M, Shammaa R. Cell therapy: types, regulation, and clinical benefits. Front Med (Lausanne). 2021;8:756029. doi:10.3389/fmed.2021.756029
iv Li Y, Nice JB, Kozinova M, Adachi S, Marbán L, Elliott K, Sun M. A novel in vitro potency assay demonstrating the anti-fibrotic mechanism of action of CDCs in Deramiocel. Biomedicines. 2025;13(11):2652. doi:10.3390/biomedicines13112652
v Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13. doi:10.1038/s41572-021-00248-3
