Update July 4, 2014: Edward Lanphier, President and CEO, Sangamo Biosciences, sent me the following statement:
We read with interest your article on data presented at the meeting of the International AIDS Society earlier this week (“Hint to a Cure?: Two Men Remain HIV Free After Bone Marrow Transplants,” July 3, 2013). The story notes the difference between the Brigham and Women’s approach used in these two patients. with the approach developed by Sangamo. The latter technique utilizes our zinc-finger nuclease (ZFN)-mediated genome editing technology to create the CCR5 mutation that essentially blocks HIV from infecting the immune system.
Many clinicians would agree, however, that the three possible treatment options described in the article—including that used in the two highlighted cases– are not practical in a treatment setting, or may present too many safety and tolerability risks for the vast majority of HIV-infected patients. Sangamo’s approach, now in a Phase Two clinical study, seeks to replicate the dramatic and enduring benefit seen both in the case of Timothy Brown (“The Berlin Patient”), and in many patients naturally resistant to HIV infection. Additional Sangamo studies have found that the ZFN-based treatment creates a reservoir within the immune system of T-cells resistant to HIV infection. The ZFN-based therapy appears to be well- tolerated in patients studied to date, and may provide HIV-infected patients with a means of controlling the virus without further treatment. We await completion of our ongoing clinical studies later this year.