Catalyst Director Candidate Seminar

Sean Ekins, Ph.D., D.Sc.

Candidate: Director, UNC Catalyst for Rare Diseases

Co-founder & CEO of Phoenix Nest, Inc., and Founder & CEO of Collaborations Pharmaceuticals, Inc.

“No Disease is Too Small: Leveraging NIH Funding to Start Rare- and Neglected-Disease Companies”

Monday, December 18

4004 Marsico

11 a.m. – 12 p.m.

No Disease is Too Small: Leveraging NIH Funding to Start Rare and Neglected Disease Companies

In the 6 years since we formed Phoenix Nest, Inc. (http://www.phoenixnestbiotech.com/) with Jill Wood, the parent of a child with Sanfilippo syndrome (MPS III) Type C, we have defined an approach which could be used to develop and translate treatments for other rare and neglected diseases. Our approach involved: 1. Set up a collaborator network with leading academics and industry, 2. Submitted and won multiple NIH STTR grants to fund research in collaborator labs, 3. Placed company employees in collaborator labs, and 4. Optioned licenses to technologies developed with collaborators (e.g. gene therapy for MPS IIIC and enzyme replacement therapy for MPS IIID). To date this company has obtained 4 Grants and approximately $2.2M to fund essential preclinical research. By partnering with academics small rare disease focused companies can help them fund their work and ultimately translate it to the clinic.

In an effort to address other rare and neglected diseases Collaborations Pharmaceuticals, Inc. (www.collaborationspharma.com) was formed and we have obtained 5 NIH grants (> $2.1M) in the past year with projects for Chagas disease, HIV, Ebola, tuberculosis and machine learning software development. In addition, we have optioned treatments for different rare diseases (HNPP) or collaborated with groups (Pitt Hopkins, Multiple Sulfatase Deficiency, Batten Disease) to submit grants and access NIH resources. Our approach leverages a wide array of computational approaches to organize our efforts efficiently so we can work on many proposals and projects simultaneously with a small staff. Ultimately, if we are to address more of the 7000 rare diseases we need to build the scale that could handle orders of magnitude more diseases. These two companies represent prototypes of a new approach to develop treatments that may be outside the current realm of big pharma and biotechs. The stories behind founding these companies the resulting science as well as the challenges and opportunities will be highlighted.