Adaptimmune ($ADAP) is tapping a small, Seattle-based biotech for the gene-editing skills needed to develop off-the-shelf T-cell immunotherapies to fight cancer.
In a deal that starts with a small $5.5 million ante and winds up to $41 million in milestones, Universal Cells will use a nuclease-free approach to reengineer stem cells into customized therapeutics that can be created with donor cells. Universal Cells says it uses a recombinant adeno-associated virus (rAAV)-mediated technology to "edit" cells, avoiding the double-strand breaks in DNA that can create off-target alterations, making it safer for patients.
Adaptimmune has been pursuing a TCR strategy that many believe will have broader uses than the initial CAR-T strategy--reengineering patient cells into cancer attack weapons--that helped sweep Juno Therapeutics ($JUNO), Kite ($KITE) and others to the forefront of the T-cell immunotherapy development wave. Moving past customizing cells extracted from patients and moving to a universal, off-the-shelf approach would radically simplify manufacturing, and that has captured broad interest in the field.
Adaptimmune's allogeneic approach puts the company on a collision course with Cellectis ($CLLS), a Paris/New York hybrid which boasts of its nuclease technology for gene editing. Cellectis CEO Andre Choulika has championed the company's TALENs-based gene editing tech for off-the-shelf remedies, openly ridiculing the competition in the field. But there are several editing tools to choose from. Juno recently tied up with Editas, one of a handful of upstarts now championing CRISPR/Cas9 gene editing tools, the same approach Johnson & Johnson's ($JNJ) Janssen plans to use. And Sangamo uses zinc finger nucleases to do its gene editing work.
Universal Cells' work depends heavily on the research of CSO and co-founder David Russell, who's been using rAAV-mediated gene editing technology to engineer HLA genes in human stem cells. Adaptimmune, a 2014 Fierce 15 honoree, is based in Oxford, U.K., with facilities in Philadelphia.
"We believe that Universal Cells' platform for generating universal donor cells is also best in class and provides us with a great opportunity to test the feasibility of a longer term allogeneic product, thus allowing large numbers of patients to be treated from a single cell line," said Helen Tayton-Martin, Adaptimmune's chief operating officer.
- here's the release
Special Report: FierceBiotech's 2014 Fierce 15 - Adaptimmune