Gene Therapy: Developing a market model for rare genetic disease.

Student: Graeme White, 2018-2019

Sponsor: Amicus Therapeutics, Cranbury, NJ

Gene therapy and gene editing technologies are an emerging but highly promising new therapeutic modality which present a potentially transformative long-term solution for treating a wide variety of genetic disease. The potential for these treatments to be administered one time and result in life-altering “cures” for the patient is expected to disrupt entire treatment paradigms for many different types of monogenic diseases. The goal of gene therapy, to replace a diseased gene with a functional gene, is accomplished through use of one of two methods: 1) gene transfer or 2) gene editing. As a patient-centric company, Amicus Therapeutics is continuously evaluating new technologies which have the potential to benefit patients living with rare and orphan conditions, including lysosomal storage diseases (LSDs). This project aims to assess the markets of several LSDs and adjacent conditions, in order to identify those that may be best suited for treatment using a gene transfer approach and those best suited for a gene editing approach.