Calimmune

Calimmune was a clinical-stage biotechnology company focused on developing gene therapies for chronic diseases, with an initial focus on HIV/AIDS and later on hematologic diseases like sickle cell disease and β-thalassemia. The company was founded in 2006, based on gene modification and cell delivery technology from the laboratories of Nobel Laureate David Baltimore, Ph.D. (California Institute of Technology), Irvin Chen, Ph.D. (University of California, Los Angeles), and Inder M. Verma, Ph.D. (Salk Institute for Biological Studies).

The company's core approach was centered on ex-vivo hematopoietic stem cell (HSC) gene therapy, which involves modifying a patient's own stem cells outside the body and then re-infusing them to create a disease-resistant blood and immune system. Initially, Calimmune's lead program, Cal-1, was a gene medicine developed to protect individuals from HIV by blocking the CCR5 receptor that the virus uses to enter cells. The company conducted Phase I/II clinical trials for this therapy.

Calimmune developed two key proprietary platform technologies to overcome challenges in the commercialization of stem cell therapies. The 'Select+' technology was designed to improve the engraftment of genetically modified stem cells after infusion, potentially reducing the need for toxic pre-conditioning regimens and enhancing efficacy. The 'Cytegrity' technology was a scalable manufacturing platform for producing lentiviral vectors, the delivery mechanism used in their gene therapies.

In August 2017, Calimmune was acquired by the global biotherapeutics leader CSL Behring for an upfront payment of $91 million, with potential for up to $325 million in additional performance-based milestone payments. The acquisition provided CSL Behring with Calimmune's technologies and its preclinical asset, CAL-H, an HSC gene therapy for sickle cell disease and β-thalassemia. At the time of the acquisition, Calimmune had facilities in Pasadena, California, and Sydney, Australia.

Keywords: ex-vivo gene therapy, hematopoietic stem cell therapy, sickle cell disease, β-thalassemia, lentiviral vectors, cell delivery technology, CSL Behring, David Baltimore, CCR5, CAL-H, Select+, Cytegrity, gene modification, clinical-stage biotechnology, hematologic diseases, HIV/AIDS therapy, stem cell engraftment, biotherapeutics, lentiviral manufacturing