The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year.

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

Developing new gene therapies for autosomal dominant polycystic kidney disease (ADPKD) is still a challenge to date. A group of researchers from the Johns Hopkins Medicine in Baltimore has presented ...

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

Strengthens Intellectual Property Portfolio and Provides Protection for Therapeutic Combination in Acclaim-3 Clinical Trial ...

A rare disease that has cut off children from the outside world now potentially has a cure. A clinical trial to treat severe ...

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 ...

Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available.

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 ...

More than 74,000 participants in clinical trials lost their access to care as a result of Trump administration cuts, ...

Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common ...