To the research team working to save him, KJ Muldoon was first known only as Patient Eta. But within months, KJ’s name — and ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

CRISPR Therapeutics recently drew attention as updates highlighted progress in its gene-editing pipeline, including early-stage studies of CTX310, an in vivo therapy designed to lower LDL cholesterol ...

Chicago-based CRISPR technology company Syntax Bio says it has developed tech which automates the slow, manual process of ...

Programmable CRISPR tools are turning stem cell biology into something closer to software engineering, shrinking ...

Both stocks are risky, but one is far less so. This juicy upside potential comes at a cost. Investors seeking stocks with ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. A group of scientists successfully made a bespoke gene ...

(NewsNation) — Doctors used personal gene therapy to treat an infant with a deadly genetic disease in a medical first. KJ Muldoon was born in August of 2024, and DNA sequencing revealed he had ...

Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond Street Hospital (GOSH), has shown promising results in ...

A single CRISPR infusion lowered harmful cholesterol and triglycerides by nearly half in early trial results. Although this was a small trial, early safety findings are reassuring. Participants ...