Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has been challenging. In the latest case, the scientists developed a CRISPR ...

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 ...

Scribe projected to enter the clinic in mid-2026 with STX-1150, a PCSK9-targeting CRISPR epigenetic silencing therapy for durable LDL-C reduction ...

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

For the first time, the tools to rewrite human biology are moving from speculative fiction into regulated clinics and ...

Intellia Therapeutics CEO John Leonard explained the circumstances of the patient death at the JP Morgan Healthcare ...

Zugo-cel, targeting CD19, is in an ongoing Phase 1 basket trial in autoimmune rheumatologic diseases. Preliminary clinical data from the Phase 1 study have been encouraging, and zugo-cel has been well ...

Co-founded by Jennifer Doudna and Fyodor Urnov, the company intends to simultaneously develop many gene editing treatments for rare conditions by using the agency’s “plausible mechanism” pathway.

Developing a gene therapy typically takes years, but when Baby KJ was diagnosed with a deadly genetic condition, scientists had only months. What followed was a successful collaboration among ...

In a major breakthrough, a patient has been treated with a personalized CRISPR therapeutic that aims to cure their rare genetic disease. This individual, who is known as KJ, was diagnosed with severe ...