Proprietary gene therapy for sickle cell disease licensed to Calimmune
Good news for patients with sickle cell disease. Cincinnati Children’s and Calimmune, Inc., have announced a license agreement to develop and commercialize a gene therapy discovered by Punam Malik, MD, the Marjory J. Johnson chair of gene and cell therapy and director of the Cincinnati Comprehensive Sickle Cell Program. The therapy has the potential to make treatments more effective, less toxic and more accessible.
Sickle cell disease and beta thalassemia are among the most common genetic defects worldwide. In the United States alone, it is estimated that at least 2 million people carry one gene encoding for sickle cell anemia.
Calimmune, a clinical-stage gene therapy company, will combine our proprietary gene therapy construct gamma-globin (known to have anti-sickling properties) with their Select+™ technology. The technology aims to improve engraftment of stem cells in the bone marrow, one of the historical challenges for gene therapy. Once the genetically modified stem cells are given back to patients, Select+™ is used to positively select the modified cells, thus increasing the population of modified versus unmodified cells in the patient’s system.
“Our current clinical vector has been optimized to efficiently correct hematopoietic stem cells in patients suffering from sickle cell disease, so that the cells are able to produce the hemoglobin our circulatory system requires to function properly,” said Malik. “This collaboration with Calimmune allows us to create long-term or permanent solutions for patients with these diseases who are facing shortened life expectancy and reduced quality of life – even with the benefit of current therapies.”
