rare diseases

Businessman climbing steps to pluck a star from the sky
Biotech

Regeneron's ultrarare disease phase 3 hits, teeing up filings

The biotech plans to file for FDA approval of garetosmab in FOP by the end of 2025 on the strength of a pivotal trial that read out Wednesday.
Nick Paul Taylor Sep 17, 2025 9:31am
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Custom gene editor fixes mutation causing deadly disease in mice

Sep 11, 2025 1:48pm
patent infringement patent lawsuit

Arrowhead sues Ionis over patent dispute for Tryngolza rival

Sep 11, 2025 11:33am
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Capsida pauses phase 1 gene therapy trial after child dies

Sep 11, 2025 7:30am
Image of a neon sign with the word no lit up next to an unlit yes

FDA rejects another ultrarare disease prospect

Sep 8, 2025 10:59am
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Servier supercharges neuro pipeline with $450M deal

Sep 8, 2025 2:30am