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Researchers at the meeting reported on long-term activity of commercialized gene therapies and preliminary data on investigational candidates.
In Brief This Week: UniQure, WHO, Vertex, Lonza, Elixirgen, Eli Lilly, ClearNote Health, Penn State
News items for the week of Sept. 23, 2024.
NICE Does Not Recommend Sickle Cell CRISPR Drug Casgevy in Draft Guidance, Calls for More Data
The agency said it wants to see additional data on effectiveness and duration of response and said Vertex should submit an updated managed access proposal.
Vertex CRISPR Therapy Casgevy Gets Conditional Marketing Authorization in EU
The EC's authorization for the sickle cell and transfusion-dependent beta thalassemia treatment follows marketing approvals in the US and UK.
FDA Approves Second Indication for Vertex Pharmaceuticals, CRISPR Therapeutics' Casgevy
The CRISPR gene-editing therapy is now approved in the US for transfusion-dependent beta thalassemia, in addition to sickle cell disease.
Nov 1, 2023