Kanglin Biotechnology (Hangzhou) Co., Ltd. has announced the completion of a $20 million Series A financing round to propel the clinical development of its lead gene therapy asset, KL003, for beta thalassemia and sickle cell disease. The funding will also support the evaluation of additional therapeutic indications within the company's pipeline.
KL003: A Promising Gene Therapy Candidate
KL003 is a lentiviral vector gene therapy designed to deliver a functional β-globin gene, offering a potential one-time treatment for both beta thalassemia and sickle cell disease. The therapy has shown encouraging results in early clinical trials.
Dr. Haoquan Wu, CEO and Founder of Kanglin Biotechnology, stated, "Our best-in-class gene therapy has the potential to cure patients living with beta thalassemia and sickle cell disease through a one-time intervention... In our study, KL003 demonstrated 100% efficacy in 17 patients and resulted in a dramatically shorter time to transfusion independence and engraftment."
Clinical Trial Data and Regulatory Designations
Data from a Phase 1 study and an Investigator Initiated Trial indicated an encouraging efficacy signal and a favorable safety profile for KL003 in beta thalassemia patients. The company also highlights that the manufacturing costs for KL003 are significantly lower than those of currently approved gene therapies, potentially increasing patient access.
KL003 cell infusion has been granted Pediatric Rare Disease Designation (RPDD) and Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). Furthermore, in January 2024, the clinical trial application for KL003 cell injection was approved by the Center for Drug Evaluation (CDE) in China for the treatment of transfusion-dependent beta thalassemia.
Anticipated Phase 2 Trial
Kanglin Biotechnology plans to commence a Phase 2 pivotal study for KL003 in beta thalassemia in 2025. This trial will further evaluate the safety and efficacy of the gene therapy in a larger patient population.
About Beta Thalassemia and Sickle Cell Disease
Beta thalassemia and sickle cell disease are inherited blood disorders characterized by defects in the beta-globin gene, leading to reduced or absent production of functional hemoglobin. These conditions often result in chronic anemia, requiring regular blood transfusions and leading to significant morbidity and reduced quality of life. Current treatments, including hematopoietic stem cell transplantation, have limitations, highlighting the need for innovative therapies like gene therapy.
