Cancer-Selective Immunotherapy

Why Cancer-Selective Immunotherapy Is Promising

Cancer is one of the leading causes of death in the United States. New forms of therapy continue to be needed, and we believe a major avenue of next-generation therapeutics will be based on cancer immunotherapeutics, particularly those based on gene transfer technology (“cancer-selective immunotherapy”).

Unlike current conventional cancer treatments such as cytotoxic chemotherapy and radiation therapy, cancer-selective immunotherapy has the potential to enable specific delivery of therapeutic agents to tumors, either locally or to many sites throughout the body, depending on how the gene therapy agent is administered. Further therapeutic potential is provided by the flexibility to deliver different types of therapeutic proteins or therapeutic interference RNA (RNAi) aimed at specific target genes, that are designed to kill the cancer cells directly, inhibit tumor activated metabolic pathways, and/or induce anticancer immunity. We believe cancer immunotherapy based on gene therapy technology has the potential to be highly effective, with fewer side effects compared to conventional cancer treatment.

Significant professional experience with viral vector products has accumulated for all stakeholders in this area over the past two decades. This experience provides an infrastructure and knowledge-base that make the drug development process for such agents more reliable and predictable. Improvement, experience and standardization has accumulated in viral vector (gene delivery vehicle) design, vector production and scale-up, implementation of Good Manufacturing Practice (“GMP”), preclinical models for target diseases, clinical trial protocols, efficacy endpoint definitions, overall regulatory agency requirements and the drug approval processes. Thus, many aspects of gene transfer technology have now matured to the point of potential commercial feasibility.

Nonetheless, the Company believes that an important remaining barrier to commercialization of gene transfer cancer products is the lack of efficiency and selectivity of gene transfer (bio-distribution) to a sufficient portion of cancer cells in a solid tumor while not harming healthy tissue. We also believe gene therapy for the treatment of many cancers may require the delivery of genes to selectively activate the immune system against the cancer cells.

Tocagen™ believes its proprietary retroviral immunotherapy technologies represent an important scientific advance over challenges discovered with prior gene therapy approaches and may be able to fulfill the promise of gene therapy as a safe and effective treatment for cancer.

Additional information about Tocagen’s technology:

Brain Cancer (High Grade Glioma)

Product Candidates
Toca 511 & Toca FC