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After HIV-related advances with zinc fingers, Sangamo adds diabetic neuropathy and gliobastoma to mix
by Jeffrey Bouley  |  Email the author


RICHMOND, Calif.—Sangamo BioSciences Inc. recently announced the initiation of two new clinical trials related to its ZFP Therapeutics program, one of them a Phase 2b study in diabetic neuropathy  and the other a Phase 1 trial in glioblastoma—as well as announcing the renewal of $3 million in funding for the Phase 2b trial by the Juvenile Diabetes Research Foundation International (JDRF).  
"We are developing our lead therapeutic, SB-509, for neurological indications including diabetic neuropathy and ALS. The data generated to date in diabetic neuropathy, our most advanced program, provide direct histologic evidence of nerve regrowth with SB-509 treatment and a mechanistic proof of concept for its neuroregenerative effects," says Edward Lanphier, Sangamo's president and CEO. "Our enthusiasm for the potential and importance of this drug is shared by JDRF who, after extensive review of the clinical data, has committed to fund a further $3 million of development costs for this new Phase 2b trial. While we continue active discussions with potential corporate partners, timely initiation of this trial enables us to maximize the future value of this program."  
The glioblastoma trial itself is notable for being Sangamo's third ZFP Therapeutic and the second zinc finger nuclease (ZFN)-based program to enter the clinic, Lanphier notes.  
This follows news late last year that the California Institute for Regenerative Medicine had granted a $14.5 million Disease Team Research Award to develop an AIDS-related lymphoma therapy based on the application of Sangamo's ZFN gene-editing technology in stem cells. The four-year grant supports an innovative research project conducted by a multidisciplinary team of investigators led by Dr. John Zaia, the Aaron D. and Edith Miller Chair in Gene Therapy and chair of virology at City of Hope.
"Sangamo scientists have developed a ZFN-mediated gene-editing technology that has been demonstrated to make hematopoietic stem cells and mature immune system cells resistant to HIV infection," Dr. Zaia says. "This will be the first test of whether hematopoietic stem cells made HIV resistant using Sangamo's technology can correct the disease. If successful, our work could open the door to ZFN-based cell therapies for other important diseases."  
The support of the HIV-related ZFN- based therapy supports earlier research that also moved to the clinic just under a year ago, thanks to work by Dr. Carl June and colleagues at the University of Pennsylvania, with whom Sangamo opened a Phase 1 clinical trial to evaluate SB-728-T for the treatment of HIV/AIDS.  
The idea for the HIV-based applications for ZFN technologies was inspired in part from a case in which a man with leukemia received a bone marrow transplant from a donor who was naturally immune to HIV, and seems to have subsequently been cured of AIDS. With that case in mind, the plan was to mimic that natural mutation in human blood cells to endow patients with immunity to HIV.  
The challenge has been the inability to make precise alterations to human DNA, though, June and his team have noted, which seems to be overcome by the ZFN technology which, in principle, should work on almost any site on any chromosome.  
"Our ZFP technology functions at the DNA level and, as this application demonstrates, enables us to address highly validated therapeutic targets that have proven difficult to drug at the protein and RNA levels," said Lanphier when the SB-728-T trial was announced. "ZFPs can be engineered to regulate or modify any gene, in any cell type, which provides numerous opportunities for its therapeutic applications. This trial is another important step in establishing our ZFP technology as a major new therapeutic product development platform."

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