How cell and gene therapy developers can lessen the chances of receiving FDA complete response letters through the use of ...

In a preclinical study, UC Davis Comprehensive Cancer Center scientists have developed a highly targeted gene therapy that ...

A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin ...

Stunning success in halting Huntington's disease with gene therapy offers new hope to those with the disorder—and potential ...

The FDA has granted Fast Track designation to SAR446268 for the treatment of non-congenital myotonic dystrophy type 1.

By Daniella Parra NanoCell Therapeutics, Inc. has appointed three new members to its Scientific Advisory Board: Prof. Zoltán ...

From cancer biology to proactive health management, customized care is changing patient outcomes, but what are the costs and ...

After years of heartbreak, researchers have found an experimental treatment that can slow the progression of Huntington’s ...

With several disease-modifying therapies available for transthyretin amyloid cardiomyopathy (ATTR-CM) -- and more waiting in ...

Now what this therapy is, is that it's basically a gene knockout therapy, and you give this to the patient via IV route for ...

Opus Genetics today announced the positive 3-month pediatric data from its Phase 1/2 open-label, ascending-dose study of the ...

We caught up with the team behind the first ever mobile leukapheresis center to see what community-based care could look like ...