Our initial 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 for the disease.
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.
Our groundbreaking platform combines the power of CRISPR-Cas genome editing with the latest advances in nucleic delivery, such as adeno-associated viruses (AAVs), to create a solution that targets kidney disease at the source.
Our novel technology has three primary components:
We have developed a non-invasive method for delivering gene therapy locally to the kidney using breakthrough filed IP.
We have engineered novel AAV viruses via directed evolution that target the kidney with 100x better efficiency than existing AAVs
We are pursuing several unique gene targets in PKD1, the gene that causes, autosomal dominant polycystic kidney disease (ADPKD).
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 is the Founder and CEO of Nephrogen and has been actively immersed in kidney disease research since he was 14 years old. He completed his undergraduate and graduate research training at Stanford University in the Division of Nephrology under the mentorship of Vivek Bhalla, Avnesh Thakor, and world renowned virologist, Mark Kay. Demetri also formerly worked in Dr. George Church’s lab at Harvard Medical School and with Dr. Terry Watnick at the National NIH center for Polycystic Kidney Disease (PKD) Research. Demetri’s family has PKD, and he founded Nephrogen based on his graduate research to realize his vision of curing all genetic kidney diseases by 2030.
Dr. Vivek Bhalla is the Chief Medical Officer of Nephrogen and an Associate Professor of Medicine (Nephrology) at Stanford University. Using in vivo and in vitro approaches, Dr. Bhalla’s laboratory focuses on molecular mechanisms of diseases that involve the kidney, including diabetes and hypertension . As a physician-scientist and a practicing nephrologist, Dr. Bhalla has complementary clinical and translational research interests related to mechanisms of kidney disease and hypertension. He also has leadership roles both at Stanford and nationally. Dr. Bhalla founded and directs the nationally-accredited Stanford Hypertension Center and is Past Chair of the Council on Kidney and Cardiovascular Disease for the American Heart Association.
Maggie Thompson is a fourth-year student at New York University studying Biomolecular Science with a minor in Public Health. She first performed research focused on using protein engineering to develop theranostics for glioblastoma. More recently Maggie was involved in a US Department of Energy research project where she focused on developing and testing CRISPR interference systems in bacteria. Maggie has also been involved in epidemiological research focusing on the impacts of COVID-19 on marginalized populations in New York City, presenting her findings as the American Public Health Association National Conference. Maggie has interests in exploring drug design and genetic therapies and has a strong passion for making disease treatment accessible.
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. Thakor is an Attending Interventional Radiologist who runs his own translational laboratory at Stanford University investigating the use of mesenchymal stem cell (MSC) based therapies and novel in vivo injection methods for delivering to traditionally difficult to reach organs such as the kidney and pancreas. Dr. Thakor leads the multidisciplinary Interventional Radiology Innovation at Stanford (IRIS) program and is a leader in the new emerging field of Interventional Regenerative Medicine.
Dr. Kay is a leading researcher in the fields of gene therapy and adeno-associated virus (AAV) biology, including the identification of new AAV capsids. Dr. Kay is the head of the Division of Human Gene Therapy and professor in the Department of Pediatrics and Genetics at Stanford University School of Medicine. He is one of the founders of the American Society of Gene and Cell Therapy, served as its president from 2005-2006, and was the recipient of the society’s Outstanding Investigator Award in 2013. Dr. Kay received the E. Mead Johnson Award for Research in Pediatrics in 2000. He was elected to the American Society for Clinical Investigation in 1997 and the Association for American Physicians in 2010. He has organized many national and international conferences, including the first Gordon Conference related to gene therapy. Dr. Kay is respected worldwide for his work in vector development, gene therapy and non-coding RNA biology. He has published over 250 papers. He is currently the deputy editor of Human Gene Therapy and serves on the editorial boards of other peer-reviewed publications.
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. Liphardt is an Associate Professor of Bioengineering at Stanford University and a Professor in Residence at StartX. For his Ph.D. work at Cambridge University, he used computers, information theory, and statistics, such as stochastic context free grammars, to find patterns in genomes. He was a physics professor at UC Berkeley until 2013, when he moved to Stanford. Jan is a Sloan Research Fellow, a Searle Scholar, and has served on the board of numerous biotechnology companies.
