Takara Bio Submitted an Investigational New Drug (IND) Application to fda

[ichigo_kun]

March 19, 2012---- Takara Bio Inc. announced today that Takara Bio and its collaborator, University of Pennsylvania have jointly submitted an Investigational New Drug (IND) application to the Food and Drug Administration (FDA) dated March 17, 2012 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. We have demonstrated that human T cells transduced with MazF can significantly inhibit the replication of HIV-1 in vitro without affecting cellular functions. We have also demonstrated a promising result that MazF is similarly effective against multi-drug resistant HIV-1 clinical isolates in collaboration with Kagoshima University. Furthermore, we have 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, Japan).

Takara Bio has 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 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.


[Outline of planned clinical trial]
Sponsors   :   Takara Bio Inc./University of Pennsylvania
Trial subjects   :   HIV-1 positive adult men and women
Objectives   :   To evaluate the safety, tolerability and immunogenicity of autologous CD4+ T cells transduced with a retroviral vector expressing the MazF endoribonuclease in patients with HIV-1
Number of subjects   :   12 subjects
Trial period   :   3 years
Location of trial   :   Drexel University College of Medicine,Pennsylvania, U.S.A.
Principal Investigator   :   Jeffrey Jacobson, MD


[ 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

[freewillie99]

Some great science behind this one.

Update:

http://www.takara-bio.com/news_e/2013/01/07.htm

Takara Bio Announces Initiation of Phase 1 HIV Gene Therapy Clinical Trial
January 7, 2013---- Takara Bio Inc. announced today that Takara Bio and its collaborators, University of Pennsylvania and Drexel University College of Medicine have initiated a Phase 1 clinical trial in its program for HIV-1 infections.

Takara Bio has developed an investigational 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 Regulatory Sponsor of this study is Carl H. June, M.D., Professor of Pathology and Laboratory Medicine at the University of Pennsylvania. The Principal Investigator of the study is Jeffrey M. Jacobson, M.D., Chief of the Division of Infectious Diseases and HIV Medicine and Professor of Medicine, Microbiology and Immunology Division at Drexel University College of Medicine. The MazF retroviral vector for this study is manufactured at the GMP compliant Center for Cell and Gene Therapy Facility of Takara Bio (Kusatsu, Shiga, Japan). The patients' CD4+ T cells will be modified with MazF retroviral vector at the GMP compliant Cell and Vaccine Production Facility at the University of Pennsylvania.

In this clinical trial, the patients will be monitored for safety, tolerability and immunogenicity of the autologous CD4+ T cells modified with the MazF endoribonuclease for six months. The estimated total enrollment is twelve subjects, six in each of the Cohorts 1 and 2.