Key Takeaways
- Vertex/CRISPR Therapeutics’ exa-cel (Casgevy) now has 2,400 sickle cell patients treated globally.
- The cost of a CRISPR therapy administration has fallen from $2.2M to approximately $1.4M as manufacturing scales.
- Base editing and prime editing next-generation CRISPR tools are entering Phase 1 trials in 2026.
- Canadian CROs (contract research organizations) are benefiting as gene therapy trials proliferate.
The approval of Casgevy (exa-cel) by the FDA and Health Canada for sickle cell disease and beta-thalassemia in 2023–2024 marked the beginning of the commercial CRISPR era. Two years later, the clinical pipeline has expanded dramatically, with base editing and prime editing more precise successors to the original CRISPR-Cas9 system now entering human trials. The pace of progress is accelerating in ways that are reshaping the investment landscape for gene editing.
The CRISPR Clinical Landscape in 2026
| Company | Asset | Indication | Stage |
|---|---|---|---|
| Vertex / CRISPR Tx | Casgevy | Sickle Cell, Beta-thal | Approved |
| Intellia Therapeutics | NTLA-2001 | ATTR Amyloidosis | Phase 3 |
| Beam Therapeutics | BEAM-101 | Sickle Cell (base edit) | Phase 1/2 |
| Prime Medicine | PM359 | Chronic Granulomatous Disease | Phase 1 |
| Graphite Bio → Imago | GPH101 | Sickle Cell | Phase 1 |
Canadian Exposure
Direct Canadian investment in leading gene editing companies is primarily through ETFs (iShares Genomics ETF, ARK Genomic Revolution) or through cross-listed stocks. The most direct Canadian play is CRISPR Therapeutics (CRSP) while incorporated in Zug, Switzerland, the company was co-founded by Emmanuelle Charpentier, and its manufacturing partnership with Lonza has Canadian operations. AbCellera is also tangentially exposed through its cell therapy discovery services.