Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial.

CRISPR therapy STX-1150 could replace daily cholesterol pills with a single dose, reducing LDL by 50% for 18+ months using ...

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

Intellia is instituting new safeguards following the death of a study participant that led regulators to pause two trials in ...

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 ...

What if a cup of coffee could help treat cancer? Researchers at the Texas A&M Health Institute of Biosciences and Technology ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct gene-editing experiments. The device, CRISPR-GPT, is an artificial intelligence lab ...

(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 ...

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 ...

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 ...

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 ...