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Capricor's DMD Cell Therapy Has HOPE, But Market Is Unsure

Executive Summary

Capricor will start a Phase II trial in the first quarter of 2018 for its regenerative cell therapy CAP-1002 in Duchenne muscular dystrophy following positive results in an early-stage trial – the stem cell product was dropped by Capricor's big pharma partner Johnson & Johnson earlier this year.

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Italfarmaco’s HDAC inhibitor helped ambulatory Duchenne muscular dystrophy patients climb stairs faster than non-treated boys while Capricor’s cell therapy improved upper limb function in non-ambulatory patients.

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California biotech says it will move forward with knowledge and expertise gained through the failed partnership. Capricor hopes to re-partner CAP-1002 for cardiovascular indications, while continuing the candidate’s DMD development itself.

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