CRISPR Therapeutics proved the strength of its technology a couple of years ago when it won approval for its first product.

Following its FDA-approved Casgevy therapy for sickle cell disease and beta thalassemia, CRISPR technology is now being tested in autoimmune disorders and early Down syndrome studies. CRISPR ...

Researchers have used modified CRISPR to silence the extra chromosome 21 in Down syndrome cells, showing promising ...

We'll know more about the company's medium-term prospects by the end of the year.

The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current ...

With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for ...

A new breakthrough could change how high cholesterol is treated, offering a powerful alternative to traditional drugs. Researchers have developed tiny DNA-based molecules that shut down PCSK9—a key ...

How do our genes determine our appearance and our susceptibility to disease? This question is central to biomedical research, ...

The company's treatment for hereditary angioedema reduced attacks by 87% vs. placebo in a Phase 3 trial, clearing the way for ...

The U.S. FDA has begun piloting real-time clinical trial tracking in two cancer studies to speed up safety and efficacy monitoring. The move comes as CRISPR Therapeutics attracts investor attention ...

A trio of common amino acids may hold the key to unlocking far more powerful gene therapies. Researchers found that adding ...

Genome‑wide CRISPR screens in primary human T cells reveal human genes that promote or block HIV infection, uncovering potent ...