Gene Medicine | Business Outline | About Us | TAKARA BIO INC.

Gene Therapies

Takara Bio advances clinical development of the following gene therapies:

Takara Bio acquired the HF10 business from M's Science Corporation in November 2010 and is currently conducting a Phase I clinical trial in the United States for the treatment of head and neck cancer and other solid tumors, which is sched-uled for completion in fiscal 2014. HF10 is a spontaneously occurring attenuated mutant of Herpes Simplex virus Type 1 that displays strong antitumor activity (oncolytic activity) when locally injected into tumors. Moreover, preclinical data suggests that HF10 also contributed to the acquisition of immunity against the tumors.

In Japan, three clinical studies were conducted in patients with breast cancer, head and neck cancer, and pancreatic cancer at Nagoya University School of Medicine. The results of these studies showed oncolytic activity and tolerability of HF10.

In December 2011, Mie University Hospital began a clinical study on solid tumors. Further, in April 2013, Nagoya University School of Medicine commenced a clinical study with patients suffering from non-resectable pancreatic cancer for the treatment in combination of HF10 with existing anticancer drugs.

MolMed S.p.A, of Italy, which has in-licensed the RetroNectin method from Takara Bio, is now conducting a Phase III clinical trial of HSV-TK gene therapy for high-risk, acute hematological malignancies in Europe. Takara Bio has exclusive rights to this treatment technology in most Asian countries.

In 2008, Takara Bio began conducting a Phase I clinical trial of HSV-TK gene therapy (project code: TBI-0301) at the National Cancer Center Hospital for treatment of patients with relapsed leukemia after hematopoietic stem cell transplantation. However, the number of existing patients with this condition remained low. In order to accelerate this gene therapy project, the TBI-0301 trial was discontinued and a new joint clinical trial in Japan and South Korea (phase I / II clinical trial, project code: TBI-1101) is in preparation to commence in fiscal 2016. The new trial will focus on patients with hematological malignancy after having undergone HLA mismatched hematopoietic stem cell transplantation.

Takara Bio, in a joint effort with both the University of Pennsylvania and Drexel University, commenced an endoribonuclease MazF based gene therapy Phase I clinical trial in the United States for patients that have been infected the human immunodeficiency virus (HIV, otherwise known as the AIDS virus). This clinical trial is scheduled for completion in fiscal 2016.

In the mechanism of AIDS, replication of the virus in infected HIV immune cells causes However, the MazF-modified T-cells (a type of immune cells) are expected to remain functional even if infected by HIV, by preventing replication of the virus. MazF genes are transduced into patient-derived T-cells ex vivo using retroviral vectors that express MazF conditionally upon HIV infection. The MazF-modified T-cells that are infused back into the patients will cleave the RNA strand of HIV and thereby block the replication of the virus when it infects the transduced T-cells. As a result, this method has the potential to become a gene therapy treatment for HIV infections.

Takara Bio, in collaboration with Mie University Hospital, is preparing a Phase I clinical trial in fiscal 2014 on MAGE-A4 antigen-specific T-cell receptor (TCR) gene therapy utilizing next-generation retroviral vectors developed by the Company. The main mechanism of TCR gene therapy is that the gene-transduced lymphocytes, which acquire the capability to specifically recognize cancer cells, attack and eliminate the cancer. TCR genes that are capable of recognizing cancer antigens are tranceduced into the patient's own lymphocytes, which are then re-infused into the patient.

Follow this link:
Gene Medicine | Business Outline | About Us | TAKARA BIO INC.