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 Advisor 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.
Fernando graduated from the University of Almería (UAL) in 2020 with a Bachelor's degree in Biotechnology. The following year, he obtained a Master's degree in Biochemistry, Molecular Biology and Biomedicine at the Complutense University of Madrid (UCM). Fernando feels great interest in everything related to genomic editing, which is why he did his Master's Thesis on genetic editing (CRISPR / Cas9) at the Gene Therapy Laboratory of the Carlos III Health Institute (ISCIII). Currently, he is doing a Doctorate in Molecular Medicine at the Laboratory of Genetics and Developmental Biology of Kidney Diseases (NefroCHUS) from the Health Research Institute of Santiago de Compostela (IDIS) and at the Genomics and Bioinformatics laboratory of the Center for Research in Molecular Medicine and Chronic Disease (CiMUS), where he has been awarded a predoctoral contract. The main theme of his thesis is the application of new genetic editing technologies (base editing and prime editing) in cell models and animals carrying genetic mutations that cause polycystic kidney disease (PKD). Among his objectives is to lay the foundations for, in the long term, develop a gene therapy that allows improving the quality of life of patients with this disease. Fernando is a Senior Research Associate at Nephrogen.
Miriam comes to Nephrogen from her previous role as a Lab Operations intern at Biolabs@NYULangone, where she has developed a keen interest in innovation and the startup ecosystem. Miriam is a biotechnologist who recently graduated from the European University of Madrid. Simultaneously, sh earned her Postgraduate Diploma in Bioengineering from the University of California, Riverside. She feels passionate about scientific research and its potential to deliver impactful solutions to society. During her undergraduate training, she participated in several research projects, the most notable being CoralCure, where her team worked toward solving coral bleaching by genetically engineering the microbiota of the coral. This project significantly deepened her interest in synthetic biology and gene therapy. Miriam is an enthusiastic advocate of therapeutic solutions leveraging the latest advancements in biotechnology and she is now excited to contribute her expertise and passion as a biomedical research intern at Nephrogen.
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. Patel is a Professor (with Tenure) in the Dept. of Internal Medicine at UT Southwestern Medical Center, where he holds the Yin Quan-Yuen Distinguished Professorship in Nephrology. He obtained his M.D. from India (2000) and completed his Internal Medicine residency at Northwestern University (2004) and clinical Nephrology fellowship at UT Southwestern (2005). Following his clinical training, he was an NIH-funded Physician-Scientist trainee at UT Southwestern (2005 to 2009), studying kidney genetics and primary cilia biology. In 2010, as an independent investigator, Dr. Patel charted a new course in the then little- studied non-coding RNA field. They investigate basic aspects of RNA biology (mRNA translation regulation, RNA chemical modifications, non-coding RNAs, etc.) in the context of polycystic kidney disease (PKD) and kidney development. Their seminal scientific contribution is devising a framework for an endogenous, mutation-agnostic, PKD1/2 mRNA therapy for autosomal dominant PKD, which has already progressed to human clinical trials. Dr. Patel’s lab has been continuously funded by the NIH since 2009 and has received additional funding from the Department of Defense. They have published their work in highly-regarded journals such as PNAS, Nature Communications, and Cell Metabolism. In 2017, Dr. Patel initiated the Comprehensive PKD and Kidney Genetics clinic at UT Southwestern, which is now among the largest nationally and was designated as a Center of Excellence by the PKD Foundation in 2023. Dr. Patel is a successful and sought-after mentor. He has mentored ten postdocs; six now have academic faculty appointments, including a K08-funded physician-scientist, a K01-funded basic scientist, and a 2023 K01 applicant. He has also mentored eight undergraduate students, including two who are now MD-PhDs, six women scientists, four URM trainees, and two first- generation college graduates. Dr. Patel is the founding Director of the Training Core of a proposed new multi-institutional (UTSW, UT Dallas, UT Arlington) predoc and postdoc NIH U2C/TL1 training program. Dr. Patel is active in several important national leadership positions. He serves NIH and DOD grant study sections, including as a study section chair. He is an Editor for Scientific Reports and Frontiers of Physiology. He is also the Chair of the scientific advisory committee for the PKD Foundation. He serves as a consultant for many pharmaceutical companies, and is an elected member of the ASCI.
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.
