Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic defect.

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

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

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.

Over the next two years, the planned funding will be allocated to projects that have received some FDA feedback as well as emerging efforts.

In November 2023, the UK became the first country in the world to authorise a Crispr gene-editing therapy.

CRISPR Therapeutics (NasdaqGM:CRSP) has set new 2026 milestones, including planned regulatory submissions for Casgevy in patients aged 5 to 11. The company outlined progress in several clinical ...

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 ...

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 ...

Intellia is instituting new safeguards following the death of a study participant that led regulators to pause two trials in people with transthyretin amyloidosis. One of those tests remains suspended ...

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 ...