Kanglin raises $20M to advance sickle cell gene therapy KL003
Phase 2 trial planned for beta thalassemia patients in 2025
Kanglin Biotechnology has raised $20 million in financing to advance the development of KL003, an experimental gene therapy for sickle cell disease (SCD) and beta thalassemia.
The China-based company plans to launch a pivotal Phase 2 trial next year to test KL003 in people with beta thalassemia, a genetic condition similar to SCD marked by anomalies in the production of hemoglobin, the protein that red blood cells use to carry oxygen through the bloodstream.
“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,” Haoquan Wu, CEO and founder of Kanglin, said in a company press release. “We believe our treatment may fundamentally change the lives of patients living with these burdensome diseases.”
SCD is caused by mutations in a gene that’s needed to make a part of hemoglobin. These mutations lead to the production of an abnormal version of hemoglobin that tends to form clumps inside red blood cells, deforming them into the sickle-like shape that gives the disease its name. Sickled red blood cells are prone to getting trapped inside blood vessels and to die prematurely, ultimately leading to SCD symptoms like anemia.
Beta thalassemia is caused by mutations in the same gene that lead to abnormally low production of hemoglobin, rather than resulting in the production of an abnormal form of the protein.
Therapy provides healthy version of mutated gene
The idea behind gene therapy for SCD and beta thalassemia is to deliver a healthy version of the mutated gene to blood stem cells in the bone marrow, restoring the body’s ability to produce red blood cells with functional hemoglobin.
Similar to Lyfgenia (lovotibeglogene autotemcel), one of the first SCD gene therapies to win approval in the U.S., KL003 is designed to deliver a healthy version of the mutated gene to blood stem cells. According to Kanglin, KL003 is cheaper to manufacture than other gene therapies, which tend to carry high price tags. Lyfgenia’s list price has been reported to be more than $3 million.
“The significant reduction in manufacturing costs may provide a solution to the currently available high-cost gene therapies and ultimately increase access for this important therapy to those patients who need it most,” Wu said.
In a preliminary study of people with beta thalassemia, KL003 treatment showed favorable safety and promising hints of efficacy, according to Kanglin, which didn’t disclose details about the study results.
“In our study, KL003 demonstrated 100% efficacy in 17 patients and resulted in a dramatically shorter time to transfusion independence and engraftment,” Wu said. “We believe that KL003’s clinical profile sets it apart from others in the class and look forward to beginning our Phase 2 pivotal study next year.”
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