Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

IRSF investment positions organization at the forefront of early-stage Rett research as genomic medicine reshapes rare ...

The FDA released a much-anticipated draft guidance on how makers of rare disease genetic medicines can leverage a platform ...

At an event on Monday, the agency officially unveiled long-awaited draft guidance meant to help speed the development of bespoke treatments for extremely rare diseases.

Today, the FDA issued draft guidance outlining a new regulatory pathway intended to help sponsors gain approval for highly ...

According to an FDA press release, the draft guidance "focuses on therapies that target a specific genetic, cellular, or molecular abnormality and are designed to correct or modify the underlying ...

When a plant's immune system is triggered, its growth is stunted. Colorado State University researchers have discovered how to turn on a hormone that allows plants to keep growing as they defend ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...

The FDA expects a flood of applications for a new "plausible mechanism pathway" for approval of bespoke gene-editing treatments.

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...