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

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

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

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

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 ...

As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. The series C was led by Arch ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

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 ...

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