Biotech Bombshell: Omeros Stock Skyrockets on $2.1B Novo Nordisk Deal – What’s Next?

Intellia was an early pioneer of in-vivo CRISPR therapy. Based in Cambridge, MA, it was among the first to administer CRISPR-Cas9 therapies directly into patients ts2.tech. In 2021 Intellia reported the first clinical proof that in-body CRISPR editing could work – a single injection dramatically lowered the disease protein in ATTR amyloidosis patients ts2.tech. Today, the company’s focus is on curative gene edits in severe diseases. Its lead programs are NTLA-2001 for transthyretin amyloidosis and NTLA-2002 for hereditary angioedema ts2.tech ts2.tech. Both use CRISPR delivered via lipid nanoparticles to knock out genes with one dose. Intellia’s partnerships have fueled its pipeline: a 2016 deal with Regeneron co-developed NTLA-2001 ts2.tech, and Regeneron later expanded the alliance to other targets. Other collaborations include AvenCell, Kyverna, ONK Therapeutics and ReCode, which use Intellia’s platform to engineer cell therapies or improve delivery ts2.tech. These alliances help the small company leverage resources while concentrating on its late-stage in-vivo programs. Recent restructuring and discontinuation of a Phase 1 program reflect Intellia’s narrow focus on ATTR and HAE ts2.tech.