CRISPR Therapeutics AG (CRSP)
NASDAQ
Biotech
Gene Editing
Investment Thesis
CRISPR Therapeutics has the first approved CRISPR gene editing therapy (Casgevy, co-developed with Vertex). Sickle cell disease and beta thalassemia are cured with a single treatment. The pipeline extends to cancer (CTX110 CAR-T) and cardiovascular disease. The platform is proven; the question is execution speed and pricing power.
Key Drivers
- Casgevy Royalties: First-ever CRISPR approval — Vertex handles commercialisation, CRSP receives royalties on a curative $2-3M per patient therapy.
- In-Vivo Editing Pipeline: Moving beyond ex-vivo (cell editing outside the body) to direct in-body editing — the larger market.
- Cash Position: ~$2B+ cash runway to fund pipeline through multiple data readouts.
Risk Factors
- Casgevy Uptake: Complex treatment logistics limit patient throughput.
- Competition: Intellia, Editas, and Beam are pursuing similar targets.