MDA 2024: DMD cell therapy CAP-1002 continues to preserve limb, ...
CAP-1002, an investigational cell therapy, has shown to slow declines in upper limb and heart function in Duchenne muscular dystrophy (DMD) patients over two years in the HOPE-2 study. A Phase 3 trial is ongoing, with potential FDA approval pathways being explored. CAP-1002 aims to benefit late-stage DMD patients by slowing skeletal and cardiac muscle function decline, demonstrating disease-modifying potential and a favorable safety profile.
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CAP-1002, cardiosphere-derived cells, shows long-term safety and efficacy in DMD patients, attenuating disease progression by 64% over 24 months. It also benefits cardiac function, with 67% of patients showing improvement in EF% after ~3 years, indicating potential in modifying DMD's underlying disease.
CAP-1002, an investigational cell therapy, has shown to slow declines in upper limb and heart function in Duchenne muscular dystrophy (DMD) patients over two years in the HOPE-2 study. A Phase 3 trial is ongoing, with potential FDA approval pathways being explored. CAP-1002 aims to benefit late-stage DMD patients by slowing skeletal and cardiac muscle function decline, demonstrating disease-modifying potential and a favorable safety profile.