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Company intelligence / Profile preview
Kangxian
Hangzhou, Zhejiang Sheng, Chinakanglinbio.com ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
KL003, a lentiviral vector-based gene therapy delivering a functional β-globin gene for one-time treatment of beta thalassemia and sickle cell disease, has shown 100% efficacy in a study of 17 patients with rapid transfusion independence and engraftment. The product has received Pediatric Rare Disease Designation and Orphan Drug Designation from the US FDA. Clinical trial application approved by China CDE for transfusion-dependent beta thalassemia; pivotal Phase 2 to begin soon
- Therapeutic areas
- HematologyNeurologyInfectious Diseases
- Modalities
- Gene therapyLentiviral vector gene therapyAAV based gene therapyHematopoietic stem cell therapyFusion protein
- Industry
- Biotech
02
Partnerships
None publicly disclosed as of June 2025.
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