Uncommon Cures, LLC, a privately held rare disease clinical research organization (CRO), begins operations after completing its Series A financing led by PilotRock Investments, LLC. Uncommon Cures brings together a team of rare disease physicians with over 75 years of combined experience and a deeply experienced clinical trials team. Uncommon Cures’ mission is to reduce the cost and time to completion of rare disease clinical trials using centralized facilities, patient-centric low-impact study design, and remote monitoring. Given the increasing number and cost-burden of orphan and rare disease therapeutics, lowering the cost of getting them to market and increasing time on patent can ease pricing pressure, benefiting all parties.
The company was founded by four principals with deep ties to the rare disease community:
-Marshall Summar, MD, Founding Director of the Rare Disease Institute at Children’s National Hospital, past president of the Society for Inherited Metabolic Disorders, served as Chair of the Board of Directors of the National Organization for Rare Disorders, member of NIH’s National Center for Advancing Translational Sciences Advisory Council, member of the Rare Disease Diversity Coalition’s Steering Committee, and awardee of NORD’s Lifetime Achievement Award in Rare Diseases.
-Rob Freishtat, MD, MPH, Physician-executive with a combined 20+ years of experience in emergency management of rare disease patients, scientific research, and biotechnology business development.
-Andrew W. Lo, PhD, Charles E. and Susan T. Harris Professor at MIT, director of the MIT Laboratory for Financial Engineering, co-founder and director of BridgeBio Pharma, and also a member of NIH’s National Center for Advancing Translational Sciences Advisory Council.
-Simon Frost, Founder and CEO of Tiber Capital Group, and active in the field of rare disease research. Board member of RARE-X/Global Genes and the National Center for Advancing Translational Sciences’ Cures Acceleration Network Review Board, and President of both Cure AHC and Hope for Annabel.
Collectively, the team brings expertise in the methodologies, economics, and logistics of rare disease clinical trials. Uncommon Cures has formed alliances with leading organizations experienced in rare disease human subject protection, biostatistical analysis and trial design, clinical trial informatics, and diversity. Under the Uncommon Cures model, patients will come to a central facility for critical portions of a trial, while all other aspects will be organized around the patients’ homes and local care. With Uncommon Cures, pharmaceutical companies will only need one contract and one IRB for studies with a wide geographic distribution. Future growth will include international sites to increase global access for patients to studies and increase the pool of potential participants.