Aiming to conduct the largest sequencing project for primary sclerosing cholangitis (PSC) to date, the nonprofit research accelerator Curable has partnered with the Regeneron Genetics Center (RGC) and the Mayo Clinic to launch the International PSC Genome Project.
The RGC—a wholly owned subsidiary of Regeneron Pharmaceuticals—is bringing its sequencing and analytical capabilities to the Project with plans to sequence 5000 or more patients with the rare liver disease, as well as samples from healthy volunteers.
“Through sequencing and genetic analysis, we will be able to learn more about the genetic basis of PSC and hope to uncover actionable findings for drug discovery and genomic medicine,” Aris Baras, M.D., vp and co-head of the RGC, said in a statement.
The RGC and all collaborators in the coalition will be able to analyze the genetic data and publish their findings, Curable said.
Mayo Clinic plans to contribute more than 3000 DNA samples from volunteers, including over 1200 samples from PSC patients. In addition, Curable is establishing a coalition of nonprofit institutions that will participate in the Project.
“It is this type of large-scale collaborative effort that will dramatically accelerate the R&D process,” added Curable CEO Lisa Boyette, M.D., Ph.D. “That progress will enable us to deliver the new therapeutics and early diagnostics in Phase II clinical trials that we have promised our customers, who are PSC patients, in 5 years.”
Dr. Boyette co-founded Curable in 2014 with younger brother Jon, who has PSC, and Dietrich Stephan, Ph.D., chairman of the Department of Human Genetics at the University of Pittsburgh Graduate School of Public Health and former executive director of the Gene Partnership at Children’s Hospital Boston–Harvard Medical School.
The accelerator was founded under the name SAVE JON, with the goal of working toward a cure for PSC, and took its current name in July. Curable says its organizational blueprint can be successfully applied to other diseases affecting underserved patient populations.