12 Grants Awarded to Fund New Treatments of Rare Diseases


The U.S. Food and Drug Administration announced this week that it has awarded 12 new clinical trial research to enhance the development of medical products for patients with rare diseases. The grants, which total more than $18 million over the next four years, were awarded to principal investigators from academia and industry across the United States through the Orphan Products Clinical Trials Grants Program.

The awarded grants include:

  • Alkeus Pharmaceuticals, Inc. (Cambridge, Massachusetts), Leonide Saad, phase 2 study of ALK-001 for the treatment of Stargardt disease – $1.75 million over four years
  • Arizona State University-Tempe Campus (Tempe, Arizona), Keith Lindor, phase 2 study of oral vancomycin for the treatment of primary sclerosing cholangitis – $2 million over four years
  • Cedars-Sinai Medical Center (Los Angeles), Shlomo Melmed, phase 2 study of seliciclib for the treatment of Cushing disease – $2 million over four years
  • Columbia University of New York (New York), Yvonne Saenger, phase 1 study of talimogene laherparepvec for the treatment for advanced pancreatic cancer – $750,000 over three years
  • Emory University (Atlanta), Eric Sorscher, phase 1/2 study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma – $1.5 million over three years
  • Fibrocell Technologies, Inc. (Exton, Pennsylvania), John Maslowski, phase 1/2 study of gene-modified ex-vivo autologous fibroblasts for the treatment of dystrophic epidermolysis bullosa – $1.5 million over four years
  • Johns Hopkins University (Baltimore), Amy Dezern, phase 1/2 study of CD8-reduced T cells for the treatment of myelodysplastic syndrome or acute myeloid leukemia – $750,000 over three years
  • Oncolmmune, Inc. (Rockville, Maryland) Yang Liu, phase 2b study of CD24Fc for the prevention of graft versus host disease – $2 million over four years
  • Patagonia Pharmaceuticals, LLC (Woodcliff Lake, New Jersey), Zachary Rome, phase 2 study of PAT-001 (isotretinoin) for the treatment of congenital ichthyosis – $1.5 million over three years
  • The General Hospital Corporation (Boston), Stephanie Seminara, phase 2 study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia – $1.4 million over four years
  • University of Minnesota (Minneapolis), Kyriakie Sarafoglou, phase 2a study of subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia – $1.4 million over three years
  • University of North Carolina at Chapel Hill (Chapel Hill, North Carolina), Matthew Laughon, phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia – $2 million over four years

Disclaimer: The viewpoint expressed in this article is the opinion of the author and is not necessarily the viewpoint of the owners or employees at Healthcare Staffing Innovations, LLC.

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