Jacob Cain, PhD

I am currently at the Center for Breakthrough Medicines where I work as a Scientific Program Leader. My previous was as a Principal Investigator at Amicus Therapeutics, where I led a team of researchers developing AAV potency assays for gene therapies to treat rare disease. I began my research into rare diseases as a Post Doctoral Fellow at Sanford Research in 2014. While at Sanford, I studied the basic biology underlying a family of rare pediatric neurodegenerative diseases known as Batten Disease. As part of my studies I worked to develop in vivo and in vitro assays to understand both the biology behind the disease, as well as develop methods to screen potential therapies, including small molecules and AAV-based gene therapies.