Takara Bio is allying with the University of Pennsylvania to support an HIV gene therapy clinical trial in the U.S. The company and Carl H. June, M.D., professor of pathology and laboratory medicine, will jointly conduct preclinical studies and regulatory steps required for completion of an IND.
Takara has been developing a technology for HIV gene therapy in which MazF, an RNA cleavage enzyme from E. coli, is stably delivered to CD4 T cells by retroviral vector. Human T cells transduced with MazF can significantly inhibit replication of HIV in vitro while not affecting cell growth, the firm reports. MazF has also been shown to be effective against multidrug-resistant HIV strains, the company continues. Animal experiments on the MazF HIV gene therapy using macaques have been undertaken in collaboration with Tsukuba Primate Research Center of National Institute of Biomedical Innovation.
Takara aims to launch its first clinical trial of MazF HIV gene therapy in the U.S., considering the market potential for HIV treatment, successful achievements in ex vivo gene therapy in the country to date, and possible expansion of the business worldwide after completion of the clinical trial. Takara Bio is based in Shiga, Japan.