On January 10, 2017 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new immunotherapies, and Intrexon Corporation (NYSE:XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported the signing of a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) for the development of adoptive cell transfer (ACT)-based immunotherapies genetically modified using the Sleeping Beauty (SB) transposon/transposase system to express T-cell receptors (TCRs) for the treatment of solid tumors (Press release, Intrexon, JAN 10, 2017, View Source [SID1234517330]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The principal goal of the CRADA is to develop and evaluate ACT for patients with advanced cancers using autologous peripheral blood lymphocytes (PBL) genetically modified using the non-viral SB system to express TCRs that recognize specific immunogenic mutations, or neoantigens, expressed within a patient’s cancer. Clinical evaluations of the ability of these SB-engineered PBL to express TCRs reactive against cancer mutations to mediate cancer regression in patients with metastatic disease will be performed.

Research conducted under the CRADA will be at the direction of Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI’s Center for Cancer Research, in collaboration with researchers at ZIOPHARM and Intrexon.

"Treating liquid tumors with chimeric antigen receptors has yielded extraordinary results with genetically engineered T cells and the next stage in the evolution of this immunotherapy is the expression of T-cell receptors to target solid tumors," said Laurence Cooper, MD, PhD, Chief Executive Officer of ZIOPHARM. "Through use of the scalable non-viral Sleeping Beauty platform to express an array of TCRs that recognize neoantigens within each patient’s tumor, we can customize T-cell therapies and enhance their function through cytokines and switches."

The SB transposon-transposase is a unique system for introducing genes encoding chimeric antigen receptors (CARs) and TCRs into lymphocytes. This non-viral platform may play an important role in immunotherapy and has several potential advantages over viral-based delivery systems including lowering the cost of genetic modification and the generation of T cells with minimal ex vivo processing supporting the personalization of T-cell therapy.

Dr. Cooper added, "The Sleeping Beauty system is the most advanced non-viral cell engineering platform in clinical development and we look forward to working with Dr. Rosenberg and the NCI to explore its potential to express neoantigen-specific TCRs to develop individualized immunotherapies for patients with cancer."