Biotechnology company
Appears in 2 stories
Developer of CTX310, presenting Phase 1a data
A single infusion of a CRISPR gene-editing therapy kept LDL cholesterol about 52% lower and triglycerides about 48% lower for a full year. The results, from a 15-patient trial, are the longest durability data yet for in vivo CRISPR in heart disease. They were presented at the European Society of Cardiology meeting and published in the New England Journal of Medicine.
Updated Sep 3
Commercializing Casgevy globally
In August 2024, KJ Muldoon was born with a death sentence encoded in his DNA—a single broken letter among three billion that left his body unable to process protein. Six months later, he walked out of Children's Hospital of Philadelphia, cured by a gene-editing therapy designed, manufactured, approved, and delivered in 180 days.
Updated May 19
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