Our beachhead indication is autosomal dominant polycystic kidney disease (ADPKD), which affects 600,000 Americans and has no cure or effective treatment. Tolvaptan (JYNARQUE®) is the only FDA approved drug for ADPKD, costs $13K for a 28-day supply, and must be taken twice daily for decades for maximum benefit. Our solution aims to halt ADPKD from the onset and provide an outright cure rather than a temporary Band-Aid®.
We're boldly committed to reimagining therapeutics for life threatening kidney disease. Our broad patent portfolio will allow us to rapidly translate our technologies into foundational partnerships and transformative treatments.
Nephrogen is harnessing recent advances in genome editing (CRISPR-Cas9) and nucleic acid delivery (AAV, nanoparticles) to develop outright cures for genetic kidney disease. We are advancing several different therapeutic approaches including ex vivo perfusion and re-transplantation, in vivo local delivery, and in vivo systemic delivery.
We take the kidney out of a patient, perfuse it with our editing solution, and then put it back in.
We inject our editing solution directly into the kidney.
We inject our editing solution into the bloodstream (or even orally) and it finds its way to the kidney.
Nephrogen's drug discovery platform leverages innovations in computer vision and high-throughput screening (HTS) to find new small molecule drugs for other cystic kidney diseases, including and nephronophthisis.
Demetri's family has suffered from kidney disease for four generations, so he founded Nephrogen in August 2015 to immediately begin working towards a cure. Demetri earned his BS in Computational Biology from Stanford University where he developed a novel 3-D cell culture system for modeling polycystic kidney disease as a part of his honors thesis. His thesis was awarded the Firestone Medal of Excellence in Undergraduate Research, which is awarded to the top 10% of all theses submitted across the Stanford campus in a given year. Previously, Demetri completed research training at Tufts University, Harvard Medical School, and the Jackson Laboratory. Overall he has invented several biomedical technologies in the kidney field, which include an early detection test for chronic kidney transplant rejection and a new method to grow human kidney tissue from stem cells. He has presented his work at several scientific conferences including the American Society of Nephrology's Kidney Week and the Biomedical Engineering Society's Annual Meeting, and published his work in several scientific journals including the Journal of the American Society of Nephrology (JASN) and the Journal of Cell Science. He was also a finalist in the 2016 Intel Science Talent Search, the nation's most prestigious pre-college science competition, and a Two-Time First Place Winner at the Intel International Science and Engineering Fair, the world's largest pre-college science competition.
Antonio Varone finished his Master’s degree in the Biomedical Engineering department at Tufts University in February 2014 where he worked on optical characterization of pre-cancerous lesions induced by human papillomavirus (HPV) in engineered epithelial tissues using two-photon excited fluorescence microscopy. He joined the Wyss Institute for Biologically Inspired Engineering at Harvard where he worked on the Skin-on-Chip and Small airway-on-a-Chip projects and is now a Scientist II at Emulate Inc. Antonio is now focused on a project based on Organs-on-Chips technology, which has the ultimate goal to develop an integrated system that recapitulates the functions of basic organ units and help more accurately predict human organ response to drugs, chemicals and infective agents.
George Church is a Professor of Genetics at Harvard Medical School and the Director of PersonalGenomes.org, which provides the world's only open-access information on human Genomic, Environmental & Trait data (GET). His 1984 Harvard PhD included the first methods for direct genome sequencing, molecular multiplexing & barcoding. These led to the first genome sequence (pathogen, Helicobacter pylori) in 1994 . His innovations have contributed to nearly all "next generation" DNA sequencing methods and companies (CGI-BGI, Life, Illumina, Nanopore). This plus his lab's work on chip-DNA-synthesis, gene editing and stem cell engineering resulted in founding additional application-based companies spanning fields of medical diagnostics (Knome, Alacris, AbVitro, Pathogenica, Veritas Genetics) & synthetic biology / therapeutics (Joule, Gen9, Editas, Egenesis, enEvolv, WarpDrive). He has also pioneered new privacy, biosafety, ELSI, environmental & biosecurity policies. He is director of an IARPA BRAIN Project and NIH Center for Excellence in Genomic Science. His honors include election to NAS & NAE & Franklin Bower Laureate for Achievement in Science. He has coauthored 430 papers, 90 patent publications & one book (Regenesis).
Dr. Watnick is a graduate of the Yale School of medicine and trained in Internal Medicine at Yale-New Haven Hospital. She completed both clinical and research fellowships in nephrology at the Johns Hopkins School of Medicine. During her research training, she became interested in inherited kidney diseases and specifically in autosomal dominant polycystic kidney disease (ADPKD). She has been involved in both basic and clinical research related to polycystic kidney disease. Her laboratory has been studying the vascular phenotype that is associated with PKD1/2 mutations in humans and in mice. She directs an inherited renal disease clinic and has served as the site prinicipal investigator for several clinical trials testing therapies in patients with ADPKD. She is also the Principal Investigator for a PKD Center of excellence that is one of 4 P30 Core centers funded by the NIH/NIDDK.
Dr. Perrone has had a long-standing and wide-ranging interest in autosomal dominant polycystic kidney disease (ADPKD), including basic investigations addressing mechanisms of ion transport by cystic epithelia, clinical investigations describing causes of mortality in the ADPKD ESRD population, and participation and direction of clinical trials in ADPKD. He has had extensive experience in clinical trials including serving as the Boston site principal investigator of the HALT PKD study, as a member of the HALT PKD Steering committee, member of the Endpoints and Publications/Ancillary studies subcommittees, and Chairman of the Quality Control committee. Dr. Perrone is Boston site principal investigator and member of the global steering committee for the TEMPO 3/4 and REPRISE trials of tolvaptan in ADPKD. He has been a member of the Scientific Advisory Committee of the Polycystic Kidney Disease Foundation since 1999 (Chairman from 2006-2010). Over the past 8 years, Dr. Perrone initiated and co-led the PKD Outcomes Consortium and has been the principal clinician involved in bringing together the contributors from academia, the pharmaceutical industry, NIH, FDA, EMA, CDISC, and C-Path. Total kidney volume has been approved as a prognostic biomarker by both the FDA and the EMA. His familiarity and experience with performing clinical trials, biomarker development, background in basic science, and his expertise and experience working with multicenter clinical trials and large consortia will enable Dr. Perrone to contribute extensively to the development of new biomarkers. Dr. Perrone earned his bachelors of science in Zoology from Pennsylvania State University and his medical degree from Hahnemann Medical College (now Drexel University College of Medicine).
Dr. Chandraker graduated from Glasgow University in 1987 and trained in clinical medicine in the United Kingdom before coming to the Brigham and Women’s Hospital/Harvard Medical School to complete his Fellowship in Nephrology/Transplant Immunology. Dr. Chandraker joined the faculty at the Brigham and Women’s Hospital in 1998 and currently serves as the Medical Director of Kidney and Pancreas Transplantation. He is also the Director of the Transplantation Research Center, at Brigham and Women’s Hospital/Harvard Medical School. He is an Associate Professor of Medicine at Harvard Medical School.Dr. Chandraker has academic interests in basic, translational and clinical areas of transplantation research. He has served as a PI or Protocol Chair on several NIH sponsored Clinical Trials in Organ Transplantation. In addition he has clinical/translational research interests in BK nephropathy, anemia and genetics of transplantation. His basic research interest is focused primarily on T cell costimulatory pathways such as the B7-CD28/CTLA4 and PDL1-PD1 pathways and their role in tolerance and chronic allograft injury. He has mentored eighteen fellows in basic and clinical research and received research funding from the NIH, Juvenile Diabetes Research Foundation and the National Kidney Foundation. He has co-authored over 100 original scientific articles, review articles and book chapters including contributing chapters to Harrisons, Brenner and Rectors The Kidney, AST Primer on Transplantation and ACP Medicine Textbook, amongst others. He has served as a co-chair for the International Society of Nephrology's (ISN) program committee for the world congress in nephrology, as well as serving on various committees for the Massachusetts Medical Society, National Kidney Foundation, and The Transplantation Society. He has served on various grant review bodies for the NIH, Medical Research Council (UK), National Kidney Research Fund (UK) Canadian Institute of Health Research and the Harvard Catalyst, and is currently serving a one-year term as the President of the American Society of Transplantation.