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- FDA approves Ultragenyx gene therapy and Regeneron ultra-rare disease drugThe Food and Drug Administration gave the green light to a gene‑therapy product from Ultragenyx, marking a new treatment option in the company’s pipeline. In a separate decision, Regeneron secured…
- Capricor Therapeutics likely faces FDA rejection of its Duchenne cell therapyCapricor Therapeutics is working to avoid an imminent FDA rejection of its cell‑based treatment for Duchenne muscular dystrophy. The company says it remains confident in the drug, but the regulator…