Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

Abu Dhabi achieved a medical milestone on January 5, 2026, with the UAE's first gene-therapy injection for inherited blood disorders. CASGEVY, utilizing CRISPR-Cas9, corrects faulty DNA in conditions ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...