Rare Disease

In this article, we look at recent clinical updates from the gene-editing field. These updates include clinical data from Vor Bio's ongoing Phase 1/2a...

There are currently four clinical-stage gene-editing candidates in clinical trials for sickle cell disease, which affects millions of people worldwide...

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...

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....