Tag: Rare Disease
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Can New Data Rescue Capricor’s Duchenne Cell Therapy at the FDA?
Capricor is submitting new 24-month upper-limb data for deramiocel after an FDA advisory committee voted against the existing efficacy evidence, reopening the regulatory debate around the DMD cell therapy.
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Epicrispr Raises $90 Million to Silence Disease Genes Without Cutting DNA
Epicrispr raised $90 million to advance EPI-321, a single-dose AAV therapy designed to silence DUX4 without permanently editing DNA.