Study aims to research genetic therapies for Alport syndrome
A collaborative therapeutic development project between researchers at The University of Manchester and Duke University in the United States, has received funding from Four Points Innovation, a private company wholly-owned by affiliates of Deerfield Management. This investment aims to accelerate research to develop a novel therapy to treat a genetic condition called Alport syndrome.
"Alport syndrome affects around 1 in 5,000 individuals and is caused by alterations in collagen genes leading to kidney failure, hearing loss, and eye problems,” said Professor Rachel Lennon, Pediatric Nephrologist and Director of the Wellcome Centre for Cell-Matrix Research at the University of Manchester.
Though there are treatments that can slow the progression of the kidney disease, there are currently no therapies that address the root cause of Alport syndrome, genetic variants that disrupt the genes encoding collagen IV, which is found throughout the body in a protein meshwork called the basement membrane.
"Fixing collagen in the kidney basement membrane is exciting, as this should also open new therapies for basement membrane-associated pathologies in other organ systems, such as in blood vessels, muscles, eyes, and ears.” David R. Sherwood, Jerry G. and Patricia Crawford Hubbard Professor of Biology at Duke University.
Bryan Baines, Director of Scientific Collaborations, Duke University Liaison to Four Points Innovation said: "This project has great potential and illustrates the unique opportunity presented by this funding mechanism. It’s seizing on the strengths of the research coming from both Duke and The University of Manchester and catalyzing a milestone-driven research plan. In the absence of this arrangement with Deerfield, it is difficult to envision that this project would have gotten off the ground through more customary funding vehicles."
Through Four Points Innovation, Deerfield has committed up to $130 million of funding for 10 years for Duke-affiliated preclinical development of new drugs for improved quality of life and cures for disease.
"We are excited to launch our first project with Four Points and begin our collaborative work to translate this novel therapy into a potential treatment" said Michael Foley, PhD, CEO, Deerfield Discovery and Development at Deerfield.