Pharmaceutical Research

, Volume 33, Issue 4, pp 809–815 | Cite as

Incentives for Starting Small Companies Focused on Rare and Neglected Diseases

Perspective

Abstract

Starting biotech or pharmaceutical companies is traditionally thought to be based around a scientist, their technology platform or a clinical candidate spun out from another company. Between us we have taken a different approach and formed two small early stage companies after initially leveraging the perspective of a parent with a child with a life-threatening rare disease. Phoenix Nest (http://www.phoenixnestbiotech.com/) was co-founded to work on treatments for Sanfilippo syndrome a devastating neurodegenerative lysosomal storage disorder. In the space of just over 3 years we have built up collaborations with leading scientists in academia and industry and been awarded multiple NIH small business grants. The second company, Collaborations Pharmaceuticals Inc. (http://www.collaborationspharma.com/) was founded to address some of the other 7000 or so rare diseases as well as neglected infectious diseases. The Rare Pediatric Disease Priority Review Voucher is likely the most important incentive for companies working on rare diseases with very small populations. This may also be partially responsible for the recent acquisitions of rare disease companies with late stage candidates. Lessons learned in the process of starting our companies are that rare disease parents or patients can readily partner with a scientist and fund research through NIH grants rather than venture capital or angel investors initially. This process may be slow so patience and perseverance is key. We would encourage other pharmaceutical scientists to meet rare disease parents, patients or advocates and work with them to further the science on their diseases and create a source of future drugs.

KEY WORDS

entrepreneurship neglected diseases rare diseases Sanfilippo syndrome Small companies 

Abbreviations

eRA

Electronic research administration

FDASIA

Food and drug administration safety and innovation act

IND

Investigational new drug application

MPS

Mucopolysaccharidosis

NDA

New drug application

NIH

National institutes of health

NME

New molecular entities

SBIR

Small business innovation research

STTR

Small business technology transfer

VC

Venture capital

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Copyright information

© Springer Science+Business Media New York 2015

Authors and Affiliations

  1. 1.Phoenix Nest, Inc.BrooklynUSA
  2. 2.Collaborations Pharmaceuticals Inc.Fuquay VarinaUSA
  3. 3.Jonah’s Just BegunBrooklynUSA

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