In this clinical update, we look at recent advances in two clinical-stage gene-editing programmes. Editas Medicine's sickle cell disease candidate...

Spinal muscular atrophy is a severe neuromuscular disease caused by mutations in the SMN1 gene. While a few therapies have been approved for SMA, none...

Intellia Therapeutics recently announced that the FDA has cleared an Investigational New Drug application for NTLA-2002, a single-dose in vivo CRISPR...

Professor Stephanie Cherqui of University of California San Diego’s School of Medicine recently received a prestigious grant from the Californian...

This up-to-date roundup presents the 7 clinical-stage gene-editing approaches to sickle cell disease, which affects millions of people worldwide and...

Editas Medicine announced earlier this week that it had dosed the first child in the BRILLIANCE clinical trial of EDIT-101 for Leber congenital...

In this week's update, we provide news about Intellia Therapeutics' and Regeneron's ongoing Phase 1 trial for NTLA-2001 in transthyretin amyloidosis....

In this week's clinical trial update, we summarise the ongoing in vivo CRISPR clinical trials for three distinct rare genetic diseases: hereditary...