MolMed S.p.A., a strategic partner of Takara Bio in gene therapy, obtains a regulatory approval to start a Phase III trial of HSV-TK gene therapy for high risk acute leukemia in Italy

Jan 22, 2008 --- Takara Bio Inc. (President and CEO: Ikunoshin Kato, Ph.D.) announces that its partner in development of gene therapy, MolMed S.p.A. (Milan, Italy) obtained a regulatory approval from AIFA (the Italian National Health Authority ) to start in Italy a Phase III clinical trial of HSV-TK gene therapy.

Under the mutual license agreements between the parties, Takara Bio acquired an exclusive right to commercialize HSV-TK gene therapy for hematological malignancies in Asian countries, while MolMed is granted a right to use Takara Bio's proprietary RetroNectin technology non-exclusively in the United States and Europe. The companies have been conducting clinical development of HSV-TK gene therapy in their respective territories.

Hematopoietic stem cell transplantation (HSCT) is thought to be an effective treatment for hematological malignancies such as leukemia, lymphoma and myeloma. However, the majority of patients cannot benefit from HSCT due to the lack of fully compatible donors. The HSV-TK gene therapy allows patients lacking fully compatible donors to safely receive HSCT from partially compatible (haplo-identical) family donors, such as their parents or children (haplo-HSCT) by infusing HSV-TK transduced donor lymphocytes (TK-DLI) to the patient after the haplo-HSCT, since the therapy enables to control the major complication of haplo-HSCT, i.e. Graft versus Host Disease (GVHD), an immune attack of the donor's T cells against the patient's organs and tissues. If GVHD occurs, the patient receives a drug (ganciclovir) that leads to the selective elimination of the donor's T cells carrying the HSV-TK gene, thus abrogating GVHD while preserving the underlying bone marrow and native immune system unaffected.

MolMed's Phase I/II trial of the HSV-TK gene therapy conducted in Europe for high risk hematological malignancies showed promising results, showing that the TK-DLI enables a prompt and sustained reconstitution of the patient's immune system after haplo-HSCT, as well as its safety and efficacy on haplo-HSCT. Especially, the results show an unprecedented improvement in survival of patients when compared to conventional treatments. In the approved multicentric, randomised Phase III trial, MolMed will assess these very positive outcome obtained from the Phase I/II trial. Authorization of the trial by the AIFA requires the completion of analytical characterization of TK components within the treatment of 20% of patients involved. In 2008, MolMed also plans to initiate a Phase I/II trial of HSV-TK gene therapy in MD Anderson Cancer Center in the United States.

Takara Bio is currently planning to start a clinical trial of TK-DLI for relapsed leukemia patients after allogenic HSCT in Japan within 2008.

[ 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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