Calimmune Expands Lentiviral Gene Therapy Pipeline Through License of Sickle Cell Disease Therapeutic Candidate

License of Gamma-Globin from Cincinnati Children’s Hospital Medical Center to be used in combination with Calimmune’s Select+™ Technology

TUCSON, Ariz.-- December 5, 2016

-- (BUSINESS WIRE) -- Calimmune, Inc., a clinical-stage gene therapy company, today announced a license agreement with Cincinnati Children’s Hospital Medical Center  (Cincinnati Children’s) to develop and commercialize gene therapies combining Calimmune’s Select+™ technology with Cincinnati Children’s proprietary gene therapy construct for the treatment of patients with sickle cell disease and beta thalassemia. Calimmune’s Select+™ platform aims to improve engraftment of stem cell gene therapies through a post-cellular infusion selection process. 

“The combination of Cincinnati Children’s proprietary lentiviral construct with Calimmune’s Select+ technology is a powerful innovative step in the fight against debilitating hematologic conditions such as sickle cell disease and beta thalassemia,” said Salim Yazji, M.D., chief medical officer of Calimmune. “We are applying our unique experience in ex vivo lentiviral vector gene therapy to make treatments for hemoglobinopathy more effective, less toxic, and ultimately more accessible to the patients that need them.” 

“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 Punam Malik, M.D., the Marjory J. Johnson chair of gene and cell therapy and director of the Cincinnati Comprehensive Sickle Cell Program at Cincinnati Children’s. “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.” 

Monogenic diseases such as hemoglobinopathies are prime targets for gene therapy due to their high prevalence, significant morbidity and mortality, and the resulting high cost of medical care that indicate that gene therapy can greatly improve patient outcomes and significantly reduce associated medical costs. Calimmune’s approach for the treatment of sickle cell disease involves introduction of the gamma-globin gene (known to have anti-sickling properties) into the patient’s own hematopoietic stem cells (HSCs) via a self-inactivating lentiviral vector. Following introduction of the gamma-globin gene, Calimmune’s Select+™ technology will be used to positively select for the modified HSCs, thus increasing the population of modified versus unmodified cells in the patient’s system. 

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. Lentiviral vector delivery of a gamma-globin gene therapy to autologous HSCs holds great promise in creating healthy, long-lived HSCs in patients suffering from these currently incurable genetic diseases.  

About Select+™
Calimmune’s proprietary Select+TM technology is a safe tool aimed at driving selection of the genetically modified stem cells once they are given back to patients, to improve potency and lower toxicity.  One of the historical challenges for gene therapy is achieving a high enough engraftment of stem cells in the bone marrow to reach the relevant therapeutic window.  Toxic conditioning regimens used to drive engraftment of gene modified cells cause a list of adverse events that often require hospitalization and have additional long-term risks.  Calimmune has focused on and made significant investments to solve this issue with Select+TM.  By coupling Select+TM with lentiviral therapeutic applications, we aim to reduce the conditioning regimens, increase efficacy, and improve the patient experience, ultimately making stem cell gene therapy an out-patient modality.

About Calimmune
Calimmune is accelerating the promise of gene therapy to liberate patients from chronic and currently incurable diseases. To achieve our ambitious goal, we have built a suite of technologies to advance the delivery, manufacturing, and overall efficiency of these life-changing medicines. Calimmune’s lead development programs are novel ex vivo gene therapies for hematologic diseases.

Cammy Duong
MacDougall Biomedical Communications

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