One year after receiving CRISPR gene editing therapy at CHOP, a Delaware County baby is thriving, and doctors want to expand ...

Evox Therapeutics Ltd ("Evox" or the "Company"), a biotechnology company developing innovative therapies for genetically driven neurodegenerative diseases using a next-generation gene editing modality ...

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teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.

"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...

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

To be the first person in the world cured of chronic granulomatous disease, a rare genetic condition, feels a little surreal ...

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

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 ...

Crispr’s ability to cut genetic code like scissors has just started to turn into medicines. Now, gene editing pioneer ...

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.

Scientists achieved the smell by editing genes in tomatoes responsible for their aroma to make them smell similar to popcorn.