JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and older with Wiskott-Aldrich syndrome, a rare primary immunodeficiency.

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

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 ...

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 ...

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...

Forbes contributors publish independent expert analyses and insights. Bruce Japsen writes about healthcare business and policy. More than 70% of employers and health plans expect affordability of gene ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...

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