Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...

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 ...

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 ...

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 ...

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 ...

Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...

The FDA granted accelerated approved to the first gene therapy directly administered into the brain on Nov. 14. The therapy, Kebilidi, was approved to treat adults and children with aromatic L-amino ...

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 ...

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 ...

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a ...

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 ...