The aim of the pathway is to remove red tape for bespoke therapies designed for patients with rare diseases.

The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

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 ...

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

Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would ...

Scientists generate world’s first ’extraordinary aromatic tomato plants’ by simultaneously altering two key genes ...

Scientists have long sought to understand why some plants are fragrant powerhouses while others remain subtle. Now, a ...

FDA Commissioner Marty Makary says the agency wants to remove barriers for patients suffering from rare genetic conditions.

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

Scientists are testing an entirely new way to fight heart disease: whether gene editing might offer a one-time fix for high cholesterol.