Sangamo is attempting to revolutionize care for tough-to-treat diseases by developing therapies that promote or suppress gene activity.
The company's most advanced program is SB-728-T, a therapy that seeks to modify the gene encoding CCR5, a receptor used by HIV to infect healthy immune system cells.
Because people who are naturally immune to HIV are found to produce non-functional CCR5 proteins, Sangamo SB-728-T attempts mimic that genetic mutation, thereby removing the need to rely on daily HIV medication. Research into SB-728-T is currently in phase 2 studies.
Sangamo is also conducting pre-clinical research with Biogen to create therapies that address beta thalassemia and is working with Shire on therapies addressing hemophilia. Phase 1 studies resulting from its collaboration with Biogen are expected to begin in 2016.
Although Sangamo's approach is endlessly intriguing, investors are right to be cautious. Tough to treat diseases have a particularly high failure rate during clinical trials and Sangamo's operating expenses totaled $72.7 million in 2014 and $19.7 million in Q1, so losses are likely to continue into the foreseeable future.
Clearly, SB-728-T has game-changing potential that shouldn't be ignored, but overall, most investors will find Sangamo to be too risky to buy -- at least at this stage of SB-728-T's development. Instead, investors may want to keep checking in with Sangamo to monitor its progress, an update into which may be provided when the company reports second quarter results on August 5.