Company intelligence / Profile preview
Kanglin Biotechnology
- Ownership
- Private
- Development stage
- Other
Overview
Kanglin Biotechnology’s lead asset, KL003, is a lentiviral vector gene therapy delivering a functional β-globin gene for one-time treatment of beta thalassemia and sickle cell disease. In a Phase 1/2 study and investigator initiated trial, KL003 demonstrated encouraging efficacy and safety—100% efficacy in 17 patients with rapid transfusion independence. The product has received Pediatric Rare Disease Designation and Orphan Drug Designation from the US FDA. Manufacturing costs are reported to be significantly lower than other approved gene therapies. A pivotal Phase 2 trial is expected to start in 2025.
- Therapeutic areas
- HematologyNeurologyInfectious Diseases
- Modalities
- Gene therapyLentiviral vector gene therapyAAV-based gene therapyHematopoietic stem cell therapy
- Industry
- Biotech
Drug pipeline
5 assets4 more assets in the full pipeline.
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