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

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

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory approvals worldwide for 10 therapies — all small molecules and ...

David Liu and Richard Merkin of the Broad Institute of MIT and Harvard, along with Columbia professor Sam Sternberg, have developed a new, targeted means of inserting entire genes into human DNA ...

Beam Therapeutics Inc. pivots from platform story to execution-driven thesis, with regulatory clarity accelerating BEAM-302 ...

Doctors announced this week that they have treated a newborn baby with a rare genetic disease using the world’s first personalized gene editing therapy. Geoff Bennett discussed the treatment and its ...

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.

The CRISPR gene-editing tool can be used to silence an important hepatitis B virus gene, a proof-of-concept in vitro study suggests. "It's the first time we've seen CRISPR editing done in a hepatitis ...

Adeno-associated virus vector containing an adenine base editor provides an effective strategy to treat hereditary hearing loss caused by the R75W mutation. Moreover, this gene editing technology can ...