CureDuchenne, a global leader in research, patient care, and innovation in improving and extending the lives of those living with Duchenne muscular dystrophy (Duchenne), has initiated the development of a biobank for Duchenne in partnership with University of California, Irvine (UCI) and RUCDR Infinite Biologics. The CureDuchenne Biobank is dedicated to empowering Duchenne research and the search for a cure for Duchenne that is independent and accessible to all researchers.
Founded in 2003, located in Newport Beach, California, CureDuchenne is the nation’s leading nonprofit organization dedicated to finding a cure for Duchenne, the most common and lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys living today. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy.
Established in 1998, along with a Department of Genetics at Rutgers, as Rutgers University Cell and DNA Repository, RUCDR Infinite Biologics is located on the university’s Busch Campus in Piscataway, New Jersey. It offers a complete and integrated selection of biological sample processing, analysis and biorepository services to government agencies, academic institutions, foundations and biotechnology and pharmaceutical companies within the global scientific community. RUCDR Infinite Biologics provides DNA, RNA and cell lines with clinical data to hundreds of research laboratories for studies on mental health and developmental disorders, drug and alcohol abuse, diabetes and digestive, liver and kidney diseases. RUCDR Infinite Biologics completed an $11.8 million expansion and renovation of its facilities in May 2013.
In partnership with Dr. Tahseen Mozaffar of the University of California, Irvine (UCI) for strategic development and implementation, and with RUCDR Infinite Biologics, the world’s largest university-based biorepository, for biobanking services, the CureDuchenne Biobank plans to begin sample collecting immediately at CureDuchenne Cares events around the country.
Minimally invasive blood and skin tissue samples will be requested on a voluntary basis from patients and, if possible, from their unaffected immediate blood relatives (parents, siblings or adult children) who may be interested in supporting scientific studies. To help ensure that Duchenne patients from all population groups can potentially benefit from the research, Duchenne patients from diverse age ranges, ambulatory status, genetic mutations and racial, cultural and socioeconomic groups are strongly encouraged to participate in this initiative.
By donating specimen to the dedicated CureDuchenne Biobank, families will: enable the development of patient cell lines specialized to their child’s gene mutation that can be used by any researcher to develop customized treatments; contribute to genetic studies to understand how any of the mutations in the body affect disease progression and response to therapies; and contribute to research investigating how to harness the immune response to allow for more effective treatment.
The proposed CureDuchenne Biobank board will consist of experts in biorepositories and registries, technology transfer, genetics, neuromuscular disease, immunology and ethical, legal and social issues. Dr. Tahseen Mozaffar and Dr. Armando Villalta, assistant professor, Department of Physiology & Biophysics, UCI School of Medicine are leading the CureDuchenne Biobank board. Together with CureDuchenne executives, the governing board will determine access to the CureDuchenne Biobank collection. Deidentified data and samples will be released to approved researchers in Duchenne.
“CureDuchenne is committed to supporting projects that could lead to a cure for Duchenne. But with limited resources to conduct research, scientists at academic institutions, biotechnology and pharmaceutical companies are not able to conduct the work necessary to find a cure for every child with Duchenne.” … “To fill that gap, it’s important to provide researchers with free and unrestricted access to the cell lines they need for Duchenne research.” – Debra Miller, founder and CEO, CureDuchenne
“Advances in precision medicine rely on open access biobanks with human biological specimens for analysis with the latest technologies long after the time of their collection.” … “We are thrilled to add Duchenne to the growing list of biobanks to help cure genetic diseases like Duchenne.” – Dr. Andrew Brooks, chief operating officer, RUCDR Infinite Biologics
“Only 5% of rare diseases have a treatment approved by the FDA, mostly due to the lack of high quality biospecimens for research.” … “Human specimens have long been used for translational purposes in cancer research, to investigate disease pathogenesis, to test scientific hypotheses, and to assess biomarkers identified in experimental studies.” – Dr. Tahseen Mozaffar, professor and interim chair, Department of Neurology, UCI School of Medicine
“Having access to patient cells is the first step in pursuing precision medicine initiatives that will enable us to facilitate families getting involved and engaged in research, and ensure all stakeholders are able to conduct research for all individuals living with Duchenne.” – Romina Foster-Bonds, director of programs, CureDuchenne and lead for the CureDuchenne Biobank