Sangamo is the elder citizen of the gene editing world, but now finds itself an in-demand partner for larger drugmakers eager to cure disease by changing genetic expression.
A day after signing a $350 million neurology-focused deal with Biogen — one of the biggest ever for preclinical assets in this space — Sangamo CEO Sandy Macrae said his company, founded in 1995, has brought big biopharma on board by delivering experimental drugs that can be advanced quickly.
"What I found was a company that had published a fantastic array of academic papers. They weren't thinking what a partner wants to see," Macrae, who joined Sangamo in 2016, said in an interview with BioPharma Dive.
"A partner wants to see efficacy, off-target [effects], what the development plan is," he said.
What Sangmo's new focus has yielded is collaborations with three companies worth more than $600 million in cash, covering diseases spanning neurology, hematology and oncology, adding to two collaborations that pre-date Macrae's tenure.
Sangamo's flagship technology uses zinc finger proteins to alter genetic expression. The technology has been around for at least as long as the company, but what has changed is the understanding of how it can be used, as well as ever-faster genomic sequencing and increased computing power.
"When Sangamo was started we hadn't even sequenced the human genome," Macrae said. "There are many things that make the editing world easier now that are adjacencies."
The deal names two known projects in Sangamo's pipeline, ST-501 and ST-502. These target two of the more treatment-resistant central nervous system disorders, Alzheimer's and Parkinson's, by seeking to alter production of aberrant proteins called tau and alpha-synuclein.
Neither drug has been tested in humans, however, but the deal terms suggest Biogen likes what it has seen so far.
A third, undisclosed project in neuromuscular disease will also be part of the collaboration. Sangamo will conduct early research, which will be jointly funded by the two parties, before turning the experimental drugs over to Biogen to prepare them for Investigational New Drug applications that permit their testing in humans.
Biogen also has the right to choose up to nine other experimental drugs over the next five years.
Sangamo shopped the projects to several companies working in neurological disease, arriving at a deal with Biogen, said Melita Sun Jung, the company's vice president of business development.
"There's so many bits of DNA that we can target that we can't take them all forward ourselves," Macrae said. "The most logical, sensible thing for us to do is find blue-chip partners."
In Biogen, Sangamo signed a partner whose chief focus is in neurological disease and is staking its future in these tricky areas of Alzheimer's and Parkinson's disease, among other disorders.
Biogen is preparing the experimental Alzheimer's drug aducanumab for submission to the Food and Drug Administration. That agent targets a protein called amyloid beta thought to lead to the cognitive degeneration characteristic of the disease.
ST-501 aims to disrupt production of tau, another protein that has been implicated in Alzheimer's. Biogen's own tau-targeting experimental drug BIIB076, meanwhile, is in Phase 1 trials in Alzheimer's disease.