National Institute of Neurological Disorders and Stroke (NINDS) at NIH has awarded $229,560 to Collaborations Pharmaceuticals Inc of Raleigh, NC to initiate a project at Washington University (St. Louis, MO) aimed at developing an enzyme replacement therapy to treat infantile Batten disease, also known as CLN1. The company will partner with Batten disease researcher Jonathan Cooper, PhD, Professor of Pediatrics at Washington University School of Medicine.
This treatment approach, originally funded by Taylor’s Tale (an LDN-affiliated patient advocacy group) and NINDS, began in the lab of Sandra Hofmann, MD, PhD, at the University of Texas Southwestern (Dallas, TX). Supported by these funds, Dr. Hofmann and her research team performed extensive development of the protein as well as pre-clinical assessment. Taylor’s Tale, a 501(c)3 public charity based in Charlotte, NC, funded Dr. Hofmann’s work beginning in 2007.
Moving forward, Collaborations Pharmaceuticals Inc will further develop the PPT1 enzyme, setting the stage for future clinical studies of this potential treatment for CLN1. In 2017 Collaborations Pharmaceuticals Inc received Orphan Drug Designation from the FDA for this PPT1 enzyme.
Collaborations Pharmaceuticals Inc is led by CEO Sean Ekins, PhD, who is also the President, CEO, and co-founder of Phoenix Nest, Inc.