Chiara Bonini, M.D., head of the experimental hematology unit at San Raffaele Hospital in Milan, performed the studies in her laboratory in collaboration with Luigi Naldini, head of TIGET at San Raffaele Hospital, and Sangamo scientists. Bonini presented the data at the opening session of the 2012 Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT) in Geneva, Switzerland. The work’s importance has been indicated by the receipt of the Van Bekkum Award, which is the most prestigious EBMT award for the best abstract submitted to the physician’s program.
The study’s data represent a notable advance in maximizing the potential safety and efficacy of adoptive T-cell therapies for cancer. While it is presently possible to reprogram immune system cells to preferentially recognize and kill tumors, several hurdles still exist that limit the potential of this approach. ZFN-based genome editing allows specific modification of these redirected cells, disrupting genes that interfere with their potency and specificity and thereby generating a safer, more effective therapeutic product.
Sangamo BioSciences specializes in the research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. The company has ongoing phase 2 and phase 1/2 clinical trials to evaluate a novel ZFP Therapeutic for the treatment of HIV/AIDS. The company’s other therapeutic programs are aimed at monogenic diseases such as hemophilia and hemoglobinopathies like sickle cell anemia. The company has agreed to a strategic collaboration with Shire for developing therapeutics for hemophilia and other monogenic diseases and has forged strategic partnerships with companies in non-therapeutic applications of its technology, including Dow AgroSciences and Sigma-Aldrich Corp.
For more information, visit the company’s Web site at www.sangamo.com
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