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Sickle Cell Disease
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FDA decision on genetic sickle cell medicine looms as Vertex and CRISPR prep for approval
USA — The Food and Drug Administration is on the verge of deciding whether to approve a genetic medicine for sickle cell disease, which could…
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Novartis pulls sickle cell drug Adakveo from European market following regulatory reversal
EUROPE — A regulatory upheaval has led pharmaceutical giant Novartis to withdraw its sickle cell disease drug, Adakveo, from the European market. This decision comes…
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Game-changing gene therapies inch closer to FDA approval, but pricing looms large
USA —A transformative shift in medical treatment is on the horizon, as the U.S. Food and Drug Administration (FDA) weighs the potential approval of two…
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Sanofi partners with Scribe Therapeutics to advance in vivo gene editing for sickle cell disease
FRANCE — Sanofi has secured an exclusive license from Scribe Therapeutics to utilize its CRISPR X-Editing (XE) genome editing technologies in the pursuit of developing…