Amicus Therapeutics: Prioritizing the Gene Therapy Portfolio

Student: Grace Edgarton, 2019-2020

Sponsor: Amicus Therapeutics, Cranbury Township, NJ

This Capstone project with Amicus Therapeutics to Prioritize the Gene Therapy Portfolio is aimed at trying to provide guidance for future investment into research and development of rare disease gene therapies. Specifically, this project will look at lysosomal storage disorders which vary greatly but in general involve build-ups of different toxic materials in the body that can lead to catastrophic effects and often death. Gene therapy is a promising treatment route but involves a great deal of investment, resources and time. Amicus is looking at five different lysosomal storage disorders that need to be systematically evaluated for their commercial opportunity, risk and uncertainty. By doing a deep dive into these diseases and developing business valuations for them, the diseases can be ranked by priority and the top indication can be identified for a full commercial analysis for future investment.