Research Partners
Check out our research partners to learn more about their programs and how you can get involved in helping to advance treatments for patients and families.
As the leading research platform dedicated to rare diseases, AllStripes makes it easy for the rare disease community to contribute to new treatment research from home.
COA Therapeutics
Founded in 2018 with the aim of developing novel therapies for difficult to treat and rare genetic disorders by harnessing advances in understanding of the CoA pathway.
Coughlin Laboratory
We are a translational research lab located at the University of Colorado Anshcutz Medical Campus focusing on inborn errors of metabolism such as pyridoxine-dependent epilepsy (PDE), glutaric aciduria type I (GA I), and vitamin B6 metabolism.
Hemoshear Therapeutics
Driven to apply our scientific insights to develop medicines that improve the lives of patients with rare metabolic disorders.
LogicBio
LogicBio® Therapeutics, based in Lexington, Massachusetts, is a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood.
Moderna
Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed for the development of therapeutics and vaccines for infectious diseases, immunooncology, rare diseases, cardiovascular diseases and auto-immune diseases.
National Institutes of Health
The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency — making important discoveries that improve health and save lives.
Selecta Biosciences
At Selecta, we strive toward a common goal: to use precision immune tolerance platforms to improve the lives of patients who suffer from serious and debilitating diseases.