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Kangxian

Hangzhou, Zhejiang Sheng, Chinaseizure-journal.com ↗

Ownership: Private
Development stage: Other

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

Partnerships

None publicly disclosed as of June 2025.

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