Drug Discovery

Southern Research Receives $7.5 Million Award to Discover New Therapies for Cystic Fibrosis Patients with Rare Mutations

October 21, 2015

BIRMINGHAM, Ala. – October 21, 2015 – Southern Research has received an award from the Cystic Fibrosis Foundation’s nonprofit drug discovery and development affiliate, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT). The funding will support research focused on the development of novel drugs for cystic fibrosis (CF) patients with rare genetic mutations, furthering the ultimate goal of treating all people with CF. The milestone-based research phase is for $7.5 million over five years and can be increased to $9 million.

Cystic fibrosis is a life-threatening, genetic disease that affects approximately 30,000 people in the U.S., with almost 1,000 new cases each year, and nearly 70,000 people worldwide. In the U.S., CF is the second most common inherited disease. More than 10 million Americans unknowingly carry the CF gene and there are more than 1,800 mutations of the gene.

Southern Research will be joined in this effort by the University of Alabama at Birmingham (UAB). Southern Research’s High Throughput Screening Center, UAB, and CFFT have been working together for several years investigating the repurposing of known drugs for the treatment of CF.

In addition, through the Alabama Drug Discovery Alliance (ADDA), Southern Research and UAB established a program to look for potential new treatments for several rare genetic diseases such as Hurler’s syndrome, with mutations similar to those found in some CF patients. Initial funding through the ADDA provided the groundwork for this drug discovery project, which originated with UAB’s David Bedwell, Ph.D., and Steven M. Rowe, M.D., working with Southern Research’s HTS group, headed by Bob Bostwick, Ph.D.

“We are excited about the opportunity to expand our relationship with CFFT on the discovery of new treatments for cystic fibrosis,” said Mark J. Suto, Ph.D., vice president of Drug Discovery at Southern Research. “This project highlights the strengths and capabilities of the ADDA, which combines UAB’s basic and clinical research with the drug discovery and development expertise of Southern Research.”

“We are extremely pleased to be working with CFFT and Southern Research in this effort to find therapies for cystic fibrosis patients with a nonsense mutation,” said Steven Rowe, M.D., professor of Medicine at UAB and director of the UAB Gregory Fleming James Cystic Fibrosis Research Center. “The groundbreaking studies of nonsense mutations for this project were done by Dr. David Bedwell, Professor of Microbiology, Genetics and Cell Biology, UAB. With this award, we are uniquely positioned to build on those successes. Our understanding of the genetic causes of cystic fibrosis continues to grow, and we expect meaningful discoveries will result from this work.”

“This is a great achievement for Southern Research and a further testament to the value of the Alabama Drug Discovery Alliance partnership with UAB,” said Southern Research President and CEO, Art Tipton, Ph.D. “We appreciate the confidence that CFFT has placed in our team to identify treatments and therapies that could make a real difference.”

About Southern Research

Southern Research is a not-for-profit 501(c)(3) organization with nearly 500 scientists and engineers working across four divisions: drug discovery, drug development, engineering, and energy and environment.

  • We’re developing 18 drugs to combat various forms of cancer, ALS, Alzheimer’s, diabetes, kidney disease, and Parkinson’s, among others.
  • We’ve developed seven FDA-approved cancer drugs.
  • We’re developing new medical devices.
  • We’re helping to launch manned missions to Mars.
  • We’re making the air and water cleaner here on Earth.

We work on behalf of the National Cancer Institute, National Institutes of Health, the U.S. Department of Defense, the U.S. Department of Energy, NASA, major aerospace firms, utility companies, and other private and government organizations as we solve the world’s hardest problems. Founded in 1941, Southern Research is headquartered in Birmingham with additional laboratories and offices in Wilsonville and Huntsville, Alabama, Frederick, Maryland, Durham, North Carolina, Cartersville, Georgia, and Houston.

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