Tag: gene therapy
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Ultragenyx’s Fayuvi Wins FDA Approval as the First Treatment for Sanfilippo Syndrome Type A
The FDA approved Ultragenyx’s Fayuvi as the first treatment for pediatric MPS IIIA, supported by a 23.5-point cognitive treatment effect.
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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.
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Eli Lilly Strikes $475 M Gene Therapy Deal with MeiraGTx for Rare Retinal Disease
Eli Lilly’s $475 million acquisition of MeiraGTx marks a bold step into next-generation gene therapies targeting rare retinal diseases. Despite challenges in the gene therapy market, the move reflects growing optimism about using advanced genetic tools to improve the lives of patients with rare inherited disorders.