Intellia Therapeutics

Novo Nordisk announced plans to initiate Phase 3 trials for coramitug, a potential first-in-class amyloid depleter antibody for ATTR amyloidosis with cardiomyopathy, following successful completion of a Phase 2 study.

Regeneron Pharmaceuticals and Intellia Therapeutics are conducting a two-part clinical study evaluating REGV131-LNP1265, a CRISPR/Cas9-based gene insertion therapy for hemophilia B patients.

Intellia Therapeutics reported three-year follow-up data showing lonvoguran ziclumeran achieved a 98% mean reduction in monthly hereditary angioedema attack rates across all 10 Phase 1 patients.

CRISPR Therapeutics reported initial top-line results from an early-stage study on CTX310, showing peak reductions of up to 82% in triglycerides and 81% in LDL cholesterol with a single dose.

Singapore researchers are leading Asia's first clinical trial of Nexiguran Ziclumeran (nex-z), a groundbreaking gene-editing therapy targeting transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and potentially fatal heart disease.

CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas.

The global CRISPR-Cas9 therapy market is rapidly expanding with over 25 therapies currently in clinical trials, many of which are expected to receive regulatory approval and enter the market within the next 5-7 years.

Bayer is investing $300 million over five years in a new joint venture with CRISPR Therapeutics, plus $35 million for a minority stake in the gene-editing pioneer.

Orphan drug sales reached $168 billion globally in 2023, with growth expected to moderate to just under 10% annually through 2028, according to Evaluate's latest report.

Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.