ChatGene therapies for Duchenne muscular dystrophy focus on young ambulatory toddlers while leaving older teens in wheelchairs with declining heart function; deramiocel cell infusions halt heart and arm muscle deterioration in advanced non-ambulatory patients. Published in The Lancet, this Phase 3 trial delivers a therapeutic lifeline for teenage boys and young men battling late-stage Duchenne cardiomyopathy.
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In Duchenne muscular dystrophy, boys lose the ability to walk by their early teens. While modern gene therapy trials have focused heavily on early childhood walking, the leading cause of death for older boys in wheelchairs is the progressive scarring and failure of the heart muscle, for which no disease-modifying treatment existed.
Cardiologists developed deramiocel, an intravenous cell therapy derived from donor heart tissue. Instead of replacing missing genes, these specialized heart cells act like microscopic paramedics: they travel to damaged muscle tissues and release healing biological packages (exosomes) that halt inflammation, reduce fibrotic scarring, and stimulate existing muscle fibers.
The Phase 3 trial proved that quarterly infusions preserved vital heart pumping strength and upper-arm function, allowing patients to continue feeding themselves and using computers. By stabilizing declining heart muscles, by extending life expectancy for wheelchair-bound patients, and by validating cardiac regenerative medicine, deramiocel transforms rare disease care.
Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial
BACKGROUND Duchenne muscular dystrophy (DMD) is an X-linked genetic disease of skeletal and cardiac muscle that leads to loss of ambulation and premature death due to progressive myopathy and cardiomyopathy. Deramiocel, a heart-derived cellular therapy consisting of human allogeneic cardiosphere-derived cells, improved cardiac and skeletal muscle function in phase 1-2 studies of DMD. Our aim was to assess the efficacy and safety of deramiocel in advanced DMD and support the findings of HOPE-2. METHODS HOPE-3, a phase 3, multicentre, randomised (1:1), double-blind, placebo-controlled study, included participants aged 10 years or older with DMD. Investigational product was infused intravenously every 3 months in outpatient settings. Skeletal and cardiac function was evaluated at 12 months. The primary endpoint was total Performance of the Upper Limb 2.0 (PUL2.0) percentage change from baseline. The trial is registered with ClinicalTrials.gov (NCT05126758). FINDINGS Between June 22, 2022, and May 28, 2024, 139 participants were screened, of whom 106 were randomly assigned to deramiocel (n=54) or placebo (n=52), and included in the intention-to-treat population. The primary endpoint showed significant improvements in the deramiocel group versus placebo. For total PUL2.0, least-squares mean percentage change at 12 months favoured deramiocel by 4·55% (95% CI 0·47-8·63; p=0·029). The safety profile of deramiocel was similar to that of placebo. INTERPRETATION Deramiocel safely slows disease progression in advanced DMD, preserving skeletal muscle function. Administered quarterly in a simple outpatient regimen, deramiocel is a promising treatment for DMD, agnostic to the precise underlying genetic lesion. FUNDING Capricor Therapeutics.
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