Start of the HSV-TK Gene Therapy Clinical Trial for Relapsed Leukemia

Oct 7, 2008 --- Takara Bio Inc. (President & CEO, Ikunoshin Kato, Ph.D.) announces that it has entered into an agreement with National Cancer Center in Japan as of October 1, 2008 to start a Phase I clinical trial of the HSV-TK gene therapy for treatment of patients with hematological malignancies such as leukemia (Development code: TBI-0301) at the National Cancer Center Hospital, as the company went through required procedures after submission of a clinical trial notification of the investigational new drug to Pharmaceuticals and Medical Devices Agency on June 30, 2008. This is the first case of clinical trials of ex vivo gene therapy to be conducted in Japan.

The HSV-TK therapy is being developed worldwide under an alliance with the Italian biotech company MolMed S.p.A. (MTA: MLM), from which Takara Bio Inc. has licensed Japanese and selected Asian rights to HSV-TK for hematological malignancies, while MolMed has licensed European and US rights to RetroNectin^®, a crucial reagent used in clinical-grade, retrovirus-mediated gene therapy techniques: the partners are sharing data on the clinical development of HSV-TK. Last Spring, MolMed has started a Phase III trial of HSV-TK in high-risk leukemia in Italy.

Recently, donor lymphocyte infusion (DLI) therapy is performed for relapsed leukemia patients after the allogeneic hematopoietic stem cell transplantation. However, a serious problem accompanying this therapy is frequent occurrence of graft-versus-host disease (GVHD) as a side effect. The HSV-TK gene therapy performed by this clinical trial aims to treat leukemia by DLI therapy using genetically modified donor lymphocytes, which are prepared by transduction of the HSV-TK gene into donor lymphocytes with the retroviral vector (Investigational new drug, TBI-0301). When GVHD occurs, ganciclovir is administered to trigger specific elimination of the donor lymphocytes, that enables control of GVHD.

The objective of this clinical trial is to assess the safety of the DLI therapy using the genetically modified lymphocytes, the circulation in peripheral blood of the lymphocytes, and the feasibility of controlling GVHD by administration of ganciclovir in serious GVHD cases. This clinical trial is non-blinded and nine patients will be enrolled. This clinical trial is expected to be an important toehold for development of cell and gene therapy in Japan.

[ 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.

This article is translated from press release in Japanese for your convenience.

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