The Phase 1/2 trial is a first-in-human, open-label, multi-national study designed to evaluate the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. Trial participants will receive a ...
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...
The need to change regulations around gene-editing treatments was endorsed in November by the head of the US Food and Drug ...
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Beam soars 22% as FDA signs off on its ambitious gene-editing plan
Beam stock launched Monday after the company reached a deal with the Food and Drug Administration for its gene-editing drug, ...
A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...
An international team including researchers from Nvidia and Microsoft has used AI on a biological trove of more than a ...
A new CRISPR breakthrough shows scientists can turn genes back on without cutting DNA, by removing chemical tags that act ...
Baby KJ was the first to receive a bespoke gene-editing treatment. Personalized drugs for others could be approved within the ...
Victoria Gray spent 34 years battling the debilitating pain of sickle cell disease. Then she volunteered to be the world's first "prototype" for a CRISPR therapy, based on technology invented at UC ...
"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...
A large genetic screen has revealed how stem cells transform into brain cells, exposing hundreds of genes that make this ...
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