ANN ARBOR, MICH - Genomenon, Inc. recently announced a partnership with Don’t Forget Morgan, a nonprofit support organization focused on rare disease patients living with Beta-Propeller Protein-Associated Neurodegeneration (BPAN), a progressive disease causing abnormal iron deposits in the brain leading to profound developmental delays. The goal of this collaboration is to provide resources to assist researchers and encourage pharmaceutical companies to invest in finding effective treatments for BPAN, as well as empower patient care teams with actionable genetic information.
Based in Michigan, Don’t Forget Morgan was established in 2020 by Kelly Kozole, a Detroit Lions SVP and Christina Ftikas, a Senior Director at Xerox Corporation, mothers of children with BPAN. Bound by a common struggle, Kelly, Christina, and other “BPAN Moms” have worked tirelessly to present decision makers at leading hospitals and research institutions with a case for more aggressive BPAN research. As a result of these efforts, Don’t Forget Morgan has funded research programs at major facilities, including the University of Michigan and Sanford Research, and co-funded with Harper’s Hope Foundation for a gene therapy research grant at Massachusetts General Hospital. Additional grants are currently under review. Also, a BPAN Clinic focused on custom medical care was created at Children’s Hospital of Philadelphia (CHOP). These activities all serve a common goal: to support families dealing with this ultra-rare disease by facilitating access to scientific research and community resources.
Contributing to this vision, Genomenon was able to use artificial intelligence (AI) to quickly identify, assemble, and deliver a curated “Genomic Landscape” with 129 unique genetic variants found within WDR45, a gene linked to BPAN. This annotated genomic data provides an extensive overview—as well as profound insights—for all researchers working on the disease.
“The goal of our partnership is to accelerate the discovery of a treatment for BPAN. The comprehensive genomic landscape of this genetically-driven disease enables researchers to more quickly identify the molecular drivers of the disease and validate drug targets,” said Mike Klein, CEO of Genomenon. “We hope to take years off the front end of the drug discovery process with the use of this genetic knowledge and shorten the time to develop a treatment that can save these children’s lives.”
“We are thankful for the partnership with Genomenon and the support they will provide as we search for a treatment and cure for all children suffering with BPAN,” said Kelly Kozole, Executive Director of Don’t Forget Morgan.
The Don’t Forget Morgan team consists of volunteer professionals with diverse experiences and perspectives in rare disease research, drug development, patient advocacy, and clinical care. Their ability to access the Genomic Landscape for BPAN will play an important role in expediting the global BPAN research process. Together, Genomenon and Don’t Forget Morgan are not only working to support families during those essential first years of diagnosis, but also providing a solid foundation for pharmaceutical companies to begin the search for a cure—with a resource that would otherwise take years to assemble.
For a firsthand perspective on how this collaboration is creating hope for patients and their families, read our guest blog featuring a Don’t Forget Morgan advisor and BPAN Mom.