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Smart Score
Above averageOur mission is to create transformative gene-based medicines for serious human diseases. We are a leading biopharmaceutical company focused on the development of CRISPR-based therapeutics, including by using CRISPR/Cas9 technology. CRISPR/Cas9 is a revolutionary technology for gene editing, the process of precisely altering specific sequences of genomic DNA. We have advanced this technology from discovery to an approved medicine with unparalleled speed, culminating in the landmark first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023 with our collaborators at Vertex Pharmaceuticals Incorporated, or Vertex.
In the company’s own words · 10-K filed Feb 12, 2026 · SEC EDGAR
Business segments
Q1 2026 · $1.5MThe company’s own SEC segment disclosure.
- Collaboration$1.0M69%
- Grant$458.0K31%
Its largest product in 2026-Q1 was Collaboration at $1.0M, 69% of the disclosed total, just ahead of Grant at 31%.