March 11, 2010 — Takara Bio Inc. (President and CEO; Koichi Nakao) announced today that it has executed a collaborative research agreement with University of Pennsylvania on March 11, 2010 to support an HIV gene therapy clinical trial in the United States. Under this agreement, Takara and Carl H. June, MD, professor of Pathology and Laboratory Medicine at the University of Pennsylvania School of Medicine, will jointly conduct pre-clinical translational studies and regulatory steps required for completion of an Investigational New Drug (IND) application to the Food and Drug Administration (FDA).
Takara has been developing a novel 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. We have demonstrated that human T cells transduced with MazF can significantly inhibit replication of HIV in vitro, while not affecting cell growth. We have also demonstrated a promising result that MazF is similarly effective against multi-drug resistant HIV strains. 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 (Tsukuba, Japan).
Takara aims to launch its first clinical trial of MazF HIV gene therapy in the United States, considering market potential for treatment of HIV, 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. In the past decade, Professor June has conducted multiple clinical trials for HIV gene therapy in the United States, and therefore he and his team have extensive IND and clinical trial experience.
Under the agreement, Takara and Penn will jointly pursue an IND application, in particular pre-clinical studies including animal tests and GMP manufacturing scale-up, regulatory submission to the Recombinant DNA Advisory Committee of the National Institutes of Health (NIH RAC), pre IND meetings with FDA, and preparation and submission of documents for the IND application. We expect that the IND application will be filed in two years, and a clinical trial in the United States will be launched shortly afterwards.
[ About Takara Bio Inc. ]
Takara Bio Inc. is an innovative biotechnology company based in Shiga, Japan. As a world leader in biotechnology research and development, Takara Bio was the first company to market PCR technology in Japan and is also the developer of the RetroNectin® reagent, which is a world-standard in gene therapy protocols. In addition to providing research reagents and equipment to the life science research market, Takara Bio has active research and product development activities in the fields of gene and cell-based therapy, and agricultural biotechnology; and is committed preventing disease and improving the quality of life for all people through the use of biotechnology. Through strategic alliances with other industry leaders, the Company aims to extend its reach around the world. More information is available at http://www.takara-bio.com.