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Details

  • ●Modalities: in vivo CRISPR gene editing, ex vivo CRISPR cell therapy
  • ●Therapeutic areas: rare diseases, cardiology, hematology, immunology
  • ●Key targets: TTR (in vivo knockout), kallikrein B1 (KLKB1), ANGPTL3, HAO1, undisclosed
  • ●Indications: ATTR amyloidosis (cardiomyopathy & polyneuropathy), hereditary angioedema, refractory acute myeloid leukemia, hypercholesterolemia
  • ●Funding: $605M cash on hand (end 2025)

Partners & investors

  • Public (NASDAQ: NTLA)· Investor
  • Regeneron (collaboration partner)· Investor

Key considerations

  • ●AI tools in use: Proprietary guide RNA design algorithms, LNP formulation optimization platform, Computational off-target prediction
  • ●First company to demonstrate systemic in vivo CRISPR editing in humans
  • ●Lipid nanoparticle delivery platform enables non-viral gene editing
  • ●Guide RNA selection uses ML-based off-target scoring

Pipeline

  • Phase 32
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Source: Company website, SEC / Companies House filings, press releases, Crunchbase · biotech.today analysis (Apr 2026)

Key people

John Leonard

President & CEO

gene therapyclinical developmentrare diseases

Laura Sepp-Lorenzino

Chief Scientific Officer

CRISPR gene editingRNA therapeuticsLNP delivery
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