Phoenix Nest (http://www.phoenixnestbiotech.com/) was co-founded with a rare disease parent and is working on treatments for Sanfilippo syndrome (MPS III), a devastating neurodegenerative lysosomal storage disorder of childhood. In the space of four years, we have built academic-industry collaborations and submitted multiple NIH grant proposals. While our primary focus has been to develop chaperones for MPS IIIC we have been able to obtain NIH funding for other MPS III types. We have now completed our first phase I NIH NINDS STTR grant to fund development of an enzyme replacement therapy, with collaborator Patricia Dickson, at LA BioMed, for MPS IIID. This has led us to file a provisional patent and enabled us to submit a phase II grant proposal to further evaluate this product. Challenges that we have started to address include 1) identifying patients for clinical studies with this disease and 2) demonstrating to investors a return on investment (ROI). We will describe the importance of the rare pediatric disease priority review voucher as a means to demonstrate ROI. In addition, we have recently obtained a second phase I grant to develop a treatment for MPS IIIB. We will highlight our evolving, lean company strategy, which enables a small virtual team to coordinate and leverage the academic research resources that have been accumulated. We will summarize our collaborative strategy and efforts to partner with global groups to pursue additional treatments for MPS III. We suggest that other rare diseases could leverage our approach. To do this requires at least one scientist who can submit grant proposals and refereed publications, and ultimately, licensing so that the work will aid in funding of further development. We would encourage other scientists to meet with rare disease parents, patients, or advocates and to work with them to further the research on their diseases.

Virtual collaborations for developing Sanfilippo syndrome treatments on a shoestring

251st National Meeting (2016)

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