Clinical Research Sponsors

Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering high-quality medicines for people living with rare metabolic diseases.



BioMarin is a world leader in developing and commercializing innovative biopharmaceuticals for rare diseases driven by genetic causes



As the specialty care global business unit of Sanofi, Genzyme’s work is focused in the areas of rare diseases, rare blood disorders, multiple sclerosis, oncology, and immunology.


Horizon Therapeutics

For Horizon, true impact is about the culture we’ve created that always puts patients at the center. The outcomes we achieve reflect our fierce determination to make a difference for the patients and communities we serve.


Italfarmaco S.p.A.

Italfarmaco SpA is a private Italian multinational company located in Milan, operating in Italy and abroad in both the pharmaceutical and fine chemical industries through its controlled and/or participated companies.


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.


Prevail Therapeutics

At Prevail Therapeutics, we are developing a pipeline of gene therapies to slow or stop the underlying disease process for patients with Parkinson’s disease and other neurodegenerative disorders.


Reata Pharmaceuticals

At Reata Pharmaceuticals, we develop small molecule therapeutics with novel mechanisms of action for the treatment of severe life-threatening diseases with no approved or effective therapy.



We, at Sanofi, are there beside people in need, as a health journey partner. We are committed to high standards of manufacturing excellence.


Sarepta Therapeutics

Sarepta is a global biotechnology company on an urgent mission: to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short.


Solid Biosciences

Solid has been purpose-built to advance the best science and accelerate the discovery and development of treatments that may benefit all patients with Duchenne muscular dystrophy


Vtesse, Inc.

Vtesse, Inc. was Cydan’s first spin company and was dedicated to developing drugs for patients suffering from diseases that are underserved. Vtesse worked to advance clinical study of VTS-270 for Niemann-Pick Type C1 Disease.