Shares of Sangamo Therapeutics Inc. (NASDAQ:SGMO), a clinical-stage biotech with gene editing technology, rose 14.9% in February, according to data from S&P Global Market Intelligence. Adding Gilead Sciences, Inc. (NASDAQ:GILD) to its list of collaboration partners provided most of the lift last month.
Sangamo's zinc finger nuclease (ZFN) gene-editing technology lost its luster amid the rapid ascent of CRISPR-Cas9 techniques, but it's one of today's hottest biotechs. The stock has soared 506% since Pfizer (NYSE:PFE) agreed to develop one of Sangamo's hemophilia candidates last year, so there wasn't a lot of room to run last month when it announced a relatively minor discovery pact with Gilead Sciences.
Gilead Sciences, through its cancer-focused subsidiary Kite Pharma, wants Sangamo to discover 10 or more new cell-based therapies. Headlines highlighted $3.01 billion in potential payments, but Sangamo will need a great deal of luck to realize a fraction of those biobucks. Gilead's offer includes $150 million up front, and the partners haven't designated any pre-clinical stage candidates to advance into human studies yet. Every new drug candidate that makes its way to an FDA review leaves about 100 preclinical candidates on the scrap heap.
While we wait for the partnership to announce its first clinical-stage candidate, Sangamo should have important data for three in vivo gene-editing therapies. A few hemophilia A patients have been dosed with SB-525 so far, and the company plans on sharing data from the Pfizer-partnered study about halfway through the year.
Trials with Sangamo's wholly owned mucopolysaccharidosis candidates should give us something to chew on later this year as well. Investigators have already dosed at least two patients with SB-913, a virus that "infects" liver cells with the DNA they need to combat MPS. Look for hints of efficacy when the company releases top-line data from the study later this year.