Carbon Biosciences
Dr. Engelhardt is internationally recognized as a leader in cystic fibrosis (CF) research and gene therapy for this disorder. His pioneering work on genetic engineering in ferrets to study CF pathogenesis and stem cell biology in the lung and pancreas led to the establishment of a federally-funded National Resource Center in this area, serving the greater scientific community and biotech sectors. Over the past three decades, his laboratory has made seminal discoveries on the biology of AAV and HBoV1 transduction and intracellular trafficking that led to improved vector systems to treat CF lung disease.
Dr. Engelhardt currently holds 53 issued patents and has 23 active patent applications. Patents and applications in his portfolio have been licensed to 6 companies, including 2 start-ups and a Fortune 100 company. He was recently elected to the National Academy of Inventors (NAI) in 2019. Dr. Engelhardt’s research has been highly funded by the National Institutes of Health, the Cystic Fibrosis Foundation, and corporate sponsored research agreements, with a career total of more than $100 million in funding.
Current research in the Engelhardt laboratory focuses on the molecular basis of cystic fibrosis disease pathologies, and on the development of gene therapies for this disorder. Included are four major research areas of study: 1) lung molecular and cellular biology as it relates to the pathogenesis and treatment of cystic fibrosis (CF) lung disease, 2) viral vector development and gene therapy for cystic fibrosis, 3) pathogenesis of cystic fibrosis-related diabetes, and 4) the study of airway stem cell niches, the regulatory mechanisms that control stem cell proliferation and repair in the airway, and the development of cell-based therapies for CF using stem cells. Dr. Engelhardt's scholarly work is supported by an h-index of 94, derived from 274 peer-reviewed papers with over 30,721 citations (Google Scholars). Findings from his lab led to the co-founding of the gene therapy company Talee Bio, which was sold and is now Spirovant Sciences.
Dr. Engelhardt received his undergraduate degree in biochemistry from Iowa State University in 1985, his doctorate in human genetics from Johns Hopkins University in 1990, and conducted postdoctoral research at the University of Michigan with Dr. James Wilson.
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Carbon Biosciences
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Carbon Biosciences is expanding the therapeutic potential of gene therapy through its proprietary platform which leverages novel parvoviruses that have been pressure tested by nature to target specific tissues, carry a larger cargo, with minimal neutralizing immunity and the potential to re-dose. Founded by Longwood Fund and gene therapy pioneers John F. Engelhardt, Ph.D., and Robert M. Kotin, Ph.D., Carbon is expanding the gene therapy toolbox for the treatment of the world’s most devastating and difficult to treat diseases.