376: James Geraghty | Biotechnology For Rare Diseases “Inside The Orphan Drug Revolution”

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as “rare” because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these “orphaned” diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families.

Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry.

Jim Geraghty is an industry leader with over 30 years of strategic experience, including more than 25 years as a senior executive at biotechnology companies developing and commercializing innovative therapies. Mr. Geraghty is chairman of the boards of Idera and Pieris Pharmaceuticals and of Orchard Therapeutics, and a member of the board of Voyager and Fulcrum Therapeutics.

He was from 2013-16 an entrepreneur in residence at Third Rock Ventures, and before that served as senior vice president, North America strategy and business development at Sanofi. He spent over 20 years at Genzyme, one of the world’s leading biotech companies, as senior vice president of international development, president of Genzyme Europe, and founding president and CEO of Genzyme Transgenics. He started his career in healthcare strategy consulting at Bain.

A graduate of the Yale Law School, Mr. Geraghty also holds a Master’s degree from the University of Pennsylvania and a Bachelor’s degree from Georgetown.