"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are common-sense reforms that will address the unique characteristics of cell and ...

At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

Lilly and German biotech Seamless Therapeutics will develop recombinase-based treatments for hearing loss.

A generation ago, writing a piece like this about using artificial intelligence to develop genome-editing medicines would have sounded like a Star Trek plotline. But both fields have matured to the ...

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, recently announced that the United States Food and Drug Administration (FDA) has ...

Verve's top drug, Verve-102, is designed to target a gene called PCSK9, which is associated with heart health and cholesterol levels. HealthDay News — Drugmaker Eli Lilly plans to buy Verve ...

WILMINGTON, Mass.--(BUSINESS WIRE)--Charles River Laboratories International, Inc. (NYSE: CRL) and Elly’s Team, a parent-led foundation striving to find a cure for Neurodevelopmental Disorder with ...

The FDA granted orphan drug designation to zavabresib, an investigational therapy for the treatment of myelofibrosis, a rare ...

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing candidate, BEAM-101, for the treatment of sickle cell disease (SCD), an ...

A new class of drugs is saving the lives of children who once had no hope. These high-tech medicines can replace defective genes. But there's a catch. Many cost millions of dollars for a single dose.