CRISPR gene-editing therapies (Casgevy)
Ex-vivo editing of a patient's stem cells with CRISPR-Cas9, as in Casgevy for sickle cell disease and beta-thalassemia — approved by the FDA, MHRA, EMA and Health Canada in 2023–2024 and replacing regular blood transfusions.
1 · What it gives
CRISPR gene editing modifies genetic material precisely and in a targeted way. Casgevy (exagamglogene autotemcel) is the commercial example: patient stem cells are harvested, edited ex vivo with CRISPR-Cas9, the patient receives chemotherapy and the edited cells are reintroduced. It replaces the need for regular blood transfusions in severe beta-thalassemia and sickle cell disease. It received regulatory approvals in 2023 and 2024.
2 · What it replaces 6
3 · How to implement 4 steps
- 1Harvest cellsHarvest the patient’s stem cells.
- 2Edit ex vivoEdit them ex vivo with CRISPR-Cas9.
- 3Condition the patientGive the patient chemotherapy (myeloablative conditioning).
- 4ReinfuseReintroduce the edited cells.
4 · Who delivers
Matched suppliers (matched automatically)
| Huazhi Rice Gene Technology | China | Not classified | active |
| Shandong Shunfeng Biotechnology | China | Producer | active |
| Kheiron Biotech | Argentina | Not classified | active |
| AGseed | Argentina | Research / RTO | active |
| ALBORS SRL | Animal Health & Welfare | Italy | Research / RTO | active |
| Absolute | India | Not classified | active |
| Acceligen | United States | Not classified | active |
| Agricultural Research Center (ARC Egypt) | Egypt | Research / RTO | active |
| Agricultural Research Council (ARC) | South Africa | Research / RTO | active |
| Alliance of Bioversity International and CIAT | Colombia | Research / RTO | active |
| Arauco (Bioforest) | Chile | Research / RTO | active |
| BNARI-GAEC | Ghana | Research / RTO | active |
Leading companies and institutes (from the brief)
Vertex Pharmaceuticals and CRISPR Therapeutics are named as the companies that developed and deliver Casgevy. Others are matched by the portal under Advanced therapies.
5 · Rules & route to market
155-235 working days under EAEU rules (plus dossier); national 61-FZ route open until 31.12.2027
Information, not legal advice — confirm the procedure for your product. (2026-10-06)
United States
Casgevy is approved by the US FDA.
European Union
Casgevy is approved by the European Medicines Agency, the UK MHRA and Health Canada.
China
The sources reviewed on 2026-10-16 identified no China regulator or standard specific to this technology. Treat the route as case-by-case and ask for a check.
6 · Money: tenders & grants
7 · Tech stack & innovations
Main risks from the sources: off-target gene modifications, uncertain long-term safety, and the potential need for myeloablative conditioning.
Sources 4
- Cell.com — www.cell.com 2026-10-16
- Nice.org.uk — www.nice.org.uk 2026-10-16
- Ir.crisprtx.com — ir.crisprtx.com 2026-10-16
- Ncbi.nlm.nih.gov — www.ncbi.nlm.nih.gov 2026-10-16