Opportunities in Rare Diseases

Student: Laura Hession, 2022-2023

Amicus Therapeutics is a global biotechnology company at the forefront of advancing therapies for a range of rare and devastating diseases. All programs headed by Amicus have three common characteristics – the disease must be rare and lead to a devastating disorder, the technology developed by Amicus must have the potential to be the best possible solution in the field, and clinical data from the program must display real, meaningful benefits for patients. Amicus currently has one FDA-approved product, which is a small molecule pill to treat Fabry disease.

This capstone project will include an investigation of the current competitive and technological landscapes for a specific class of rare genetic diseases – the majority of which have no treatment. The purpose of this project is to assess Amicus’ technological, developmental and commercialization capabilities, to determine if they should bring advances to treatments in this area. The project will consist of key stakeholder interviews, a literature review, and market and financial analyses to drive the recommendation to Amicus on the potential opportunities within this space.