Opportunity Assessment – Gene Therapy for a Rare Disease "X" (confidential)

Student: Ashley Guduza, 2021-2022

Sponsor: Amicus Therapeutics, Philadelphia, PA

Amicus Therapeutics is a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare diseases. I will be partnering with them to conduct an opportunity assessment for the development of a gene therapy for a rare disease "X"(confidential). There is a lot of unmet need around this disease. with no cure currently in existence. I am going to be assisting Amicus with exploring the possibility of a gene therapy in that area. My main responsibilities will include performing a technology review. Here, I take a deep dive into the disease literature, as well as explore and research on the current therapies available, as well as do a comparison of them with our proposed technology. I will also conduct interviews for problem/solution validation, and carry out market analysis. The market analysis will include market research, SWOT analysis as well as determination of Amicus’ gene therapy's competitive position in the marketplace. I will use this and other research to come up with a business model and ultimately, a Launch Strategy.