Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...

In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory ...

India’s first indigenous CAR-T cell therapy, NexCAR19, developed by ImmunoACT, an IIT Bombay spin-off, marks a major leap in ...

The FDA’s Marty Makary and Vinay Prasad have laid out a path for CRISPR treatments on demand. An expert weighs in on what it ...

As a new €2 million gene therapy research project funded by APC and Research Ireland is announced, we speak with the project lead Prof Niall Barron about its potential and how Ireland is positioning ...

The agency is establishing a network of care centers to expand access to regenerative medicine beyond regions served by major ...

MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the first patient has been dosed in ...

Late last year, dozens of researchers spanning thousands of miles banded together in a race to save one baby boy’s life. The ...