Takara Bio submits application for HIV-1 gene therapy to FDA

ntttttttttttt

Takara Bio and collaborator University of Pennsylvania have jointly submitted an Investigational New Drug (IND) application to the Food and Drug Administration (FDA) in order to conduct a ribonuclease MazF based gene therapy phase I clinical trial for HIV-1 infection disease in the United States.

Takara Bio has been developing a novel retroviral technology for HIV-1 gene therapy, in which MazF, an endoribonuclease from Escherichia coli, confers resistance to a broad-spectrum of HIV-1 strains on CD4+ T cells.

The company has demonstrated that human T cells transduced with MazF can significantly inhibit the replication of HIV-1 in vitro without affecting cellular functions.

It has also demonstrated a promising result that MazF is similarly effective against multi-drug resistant HIV-1 clinical isolates in collaboration with Kagoshima University.

Furthermore, it has confirmed the safety of the MazF-transduced T cells in vivo animal experiments using macaques in collaboration with Tsukuba Primate Research Center of National Institute of Biomedical Innovation (Tsukuba).

Takara Bio entered into a collaboration research agreement with University of Pennsylvania on March 11, 2010 and has been pursuing an IND application with a group headed by Carl H. June, MD, Professor of Pathology and Laboratory Medicine at the University of Pennsylvania School of Medicine, and has now completed a series of preparations such as; preclinical translational studies, GMP manufacturing of MazF retroviral vector, GMP manufacturing of MazF-transduced CD4+ T cells, and has finally submitted the documents for the IND application.

After the IND review by the FDA, followed by protocol reviews by academic/additional regulatory committees including IRB, Takara Bio will initiate a phase I clinical trial in the U.S. The study will be conducted by Dr Jeffrey Jacobson, Professor of Medicine, Microbiology and Immunology, and Chief of the Division of Infectious Diseases and HIV Medicine at Drexel University College of Medicine.