Both CRSP and NTLA are advancing in vivo gene editing therapies targeting large markets, with financial stability supporting ...

The illustration shows how high trafficking of blood stem cells in the circulation early post-natally allows lentiviral-vector mediated in vivo gene transfer. “After birth, blood stem cells need to ...

Sickle cell disease (SCD) is a genetic condition that affects more than 8 million people worldwide by altering the shape of oxygen-carrying red blood cells. Normally, red blood cells are shaped like ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

Intellia Therapeutics NTLA has made meaningful progress with the development of its in vivo programs. The company is currently advancing two late-stage in vivo candidates — lonvo-z (or, NTLA-2002) for ...

Bristol Myers Squibb is securing an opportunity to compete in the next generation of cell therapies through the $1.5 billion acquisition of Orbital Therapeutics, a startup with a lead program nearing ...

First and only disease-modifying therapy for the treatment of eligible children with MLD also receives Orphan Drug ...

Robust efficacy data: ≥90% LDL-C reduction in non-human primates achieved within 48 hours of a single dose of EDIT-401; ≥90% LDL-C reduction in mice with high baseline LDL-C and reduced LDLR function ...