RICHMOND, Calif., Jan. 19 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. announced today that preliminary data from the University of Pennsylvania investigator sponsored Phase 1 safety study of Sangamo’s zinc finger nuclease (ZFN) based product, SB-728-T, for HIV/AIDS were presented on Friday, January 15, 2010 at the Keystone Symposium Session “HIV Biology and Pathogenesis.” Sangamo’s collaborator, Carl June, M.D., Director of Translational Research at the Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine, presented the data as an invited speaker in an NIAID Workshop entitled “The Next Challenge: Elimination of HIV Reservoirs.”
Dr. June described data from a single HIV- positive subject treated with SB-728-T who, as part of the study, began a structured treatment interruption (STI) from his antiviral drug therapy four weeks after SB-728-T treatment. Twelve weeks later, the STI ended and the subject resumed antiviral therapy. During the study, the subject’s CD4+ T-cell count, the number of circulating ZFN-modified cells and viral loads were measured periodically. In addition, rectal biopsies were taken prior to treatment and at the end of the STI period to monitor levels of CD4+ and ZFN-modified T-cells in the GALT.
“These are the first human data from a ZFN-based therapeutic and, although preliminary, are very encouraging and recapitulate observations that we have made in preclinical studies,” stated Dale Ando, Sangamo’s vice president of therapeutic development and chief medical officer. “Importantly, ZFN-modified cells expanded over the period that we monitored the subject and were well tolerated. As expected, the subject’s viral load increased during the STI. However, the kinetics of this subject’s viral rebound was delayed. Presence of ZFN-modified cells in the GALT, an important HIV reservoir, demonstrates that we are achieving our pharmacologic biodistribution target. GALT HIV persistence in CD4+ T-cells is the reason that HIV is not eradicated in patients who are fully suppressed on highly active anti-retroviral treatment, or HAART. Ultimately, having a protected CD4+ T-cell population in the GALT may be extremely important in combating this disease.
About SB-728-T
SB-728-T is a cell product based on Sangamo’s ZFN technology. CD4+ T-cells are removed from the subject’s blood and treated with Sangamo’s ZFNs designed to modify the DNA sequence encoding the CCR5 gene. This modification can occur directly in T-cells with only a brief exposure to the ZFNs. Once the CCR5 gene is modified, the gene is permanently disrupted in these cells.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy and a Phase 2 trial in ALS. Sangamo also has two Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS and another Phase 1 trial to evaluate safety and clinical effect of a treatment for recurrent glioblastoma multiforme. Other therapeutic development programs are focused on neuropathic pain, nerve regeneration, Parkinson’s disease and monogenic diseases. Sangamo’s core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TF) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company’s web site at http://www.sangamo.com/.
CONTACT: Elizabeth Wolffe, Ph.D. of Sangamo BioSciences, Inc.,
+1-510-970-6000, ext. 271, ewolffe@sangamo.com
Web site: http://www.sangamo.com/
