Expert: In vivo CRISPR/Cas9 gene editing therapy. Lipid nanoparticle (LNP) delivers guide RNA + Cas9 mRNA to hepatocytes. Creates targeted double-strand break in TTR gene, permanently reducing transthyretin production by >90%.

Everyday: Uses CRISPR gene editing to permanently knock out the TTR gene in the liver — the gene that makes the misfolded protein causing amyloid deposits. A single infusion permanently stops the disease at its source, unlike drugs that need ongoing dosing.

Targets: ["TTR"]

Partnered with Intellia Therapeutics (NTLA). REGN co-develops and co-commercializes. One-time IV infusion for permanent TTR knockdown. Phase 1 showed >90% TTR reduction. Two Phase 3 studies: MAGNITUDE (ATTR-CM) and HASTEN (ATTRv-PN). Competes with vutrisiran (Alnylam siRNA, chronic dosing) and tafamidis (Pfizer, TTR stabilizer). Key differentiator: one-time treatment vs chronic therapy.