CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mitobridge, Inc., a biopharmaceutical company pioneering the discovery and development of products that improve mitochondrial function, announces the signing of a sponsored research agreement to assess Mitobridge’s selective Peroxisome Proliferator-Activated Receptor Delta (PPARδ) modulators in mitochondrial disease patient cell lines. Mitobridge will support research in the laboratory of Dr. Marni J. Falk, Assistant Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, and Director of The Children’s Hospital of Philadelphia Mitochondrial-Genetic Disease Clinic. The aim of the collaboration is to evaluate the therapeutic effects of PPARδ modulators on mitochondrial function in fibroblasts derived from patients with genetically-defined mitochondrial disorders.
“We have a shared commitment to progressing innovative treatments for mitochondrial diseases, a group of disorders with tremendous unmet medical need,” commented Effie Tozzo, PhD, Vice President of Translational Biology at Mitobridge. “We are delighted to be collaborating with a leading mitochondrial disease clinician-scientist who has demonstrated expertise in translating her experience with patients into important scientific advancements.”
The research plan is designed to evaluate the ability of Mitobridge’s proprietary compounds to improve mitochondrial function in patient cells. The cells harbor specific disease-causing mutations in mitochondrial DNA and in nuclear genes that are essential for mitochondrial energy production. Positive results could support the clinical development of the PPARδ modulators for certain mitochondrial diseases such as MELAS and Leigh’s Syndrome. Earlier this year, Mitobridge convened a panel of key opinion leaders in the field including, Patrick Chinnery, MD, PhD, Richard Haas, MD, Gregory Enns, MD, Michio Hirano, MD, and Tanja Taivassalo, PhD, and they recommended that Mitobridge study the activity of PPARδ modulators on mitochondrial function in patient-derived cell lines.
Mitobridge believes that testing therapeutic candidates in Dr. Falk’s collection of fibroblast cell lines, which are derived directly from mitochondrial disease patients’ skin biopsies, should lead to a better understanding of the defects in these cells and the potential benefit for patients.
Members of Mitobridge Mitochondrial Myopathy Advisory Panel
Patrick Chinnery, MD, PhD, Professor of Neurology and Head of the Department of Clinical Neurosciences at the University of Cambridge, Richard Haas, MD, Professor of Neuroscience at UC San Diego (UCSD) School of Medicine and Director of the UCSD Mitochondrial Disease Laboratory, Gregory Enns, MD, Professor of Pediatrics (Medical Genetics) at Stanford University, Michio Hirano, MD, Professor of Neurology at Columbia University and Co-director of the North American Mitochondrial Disease Consortium (NAMDC) and Tanja Taivassalo, PhD, Associate Professor, Department of Kinesiology at McGill University.
Mitobridge is a leader in the emerging field of mitochondria-related drug discovery and development. The Company is utilizing its innovative screening platform to identify and develop novel therapeutics that improve mitochondria function and provide treatments for mitochondrial genetic diseases, musculoskeletal, kidney and neurodegenerative disorders as well as diseases and conditions of aging. Mitobridge’s scientific founders and advisors include world-renowned leaders in biology and mitochondrial function. For more information about Mitobridge, please visit the company's website at www.mitobridge.com