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 ...

An ultra-rare enzyme deficiency that keeps infants from achieving proper muscle function among other developmental delays now has its first FDA-approved treatment. The new product, a gene therapy ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

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 ...

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 ...

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 ...

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 ...

According to DataM Intelligence, the global cell and gene therapy market reached a value of US$13.90 billion in 2024 and is ...