“In 10 years, we’ve gone from being entirely unrecognized in the field of human sequencing and human genetics to being recognized as the world leaders in this space.”
George Yancopoulos, Co-founder, President and Chief Scientific Officer
We use proprietary technology to invent new medicines. By combining deep scientific expertise with our exclusive VelociSuite® technologies, we accelerate the way antibody medicines are traditionally discovered and developed. As we look to the future, we continue to improve and expand our antibody approach while also pursuing new methods of addressing human disease, such as through novel genetics medicine technologies.
Prissette et al. harness CRISPR screening to identify genetic modifiers of tau aggregation, implicate disruption of nuclear envelope integrity as a possible initiating event in tauopathies and reveal targets for therapeutic intervention.
Regeneron was founded and named with the goal of “regenerating neurons.” Our neurologist founder, Dr. Len Schleifer, developed a particular interest in Alzheimer’s disease after growing frustrated with the lack of effective treatments for these serious neurodegenerative diseases. Although, unfortunately, little progress has been made across the industry against this devastating disease, we remain committed to pursuing and, we hope, finding a viable, life-changing solution.
This year, we published intriguing preclinical research revealing a potential root cause of the cellular process leading to Alzheimer’s and other neurodegenerative diseases. The findings point to nuclear envelope integrity as a promising area of investigation in understanding the origins of tau-related diseases. Further studies could offer new genetic-based targets for therapeutic intervention.
Tau proteins, which help stabilize the internal skeleton of neurons in the brain, are essential to healthy brain function. In neurodegenerative diseases such as Alzheimer’s, tau proteins in the form of “tangles” build up in the brain. Over time, this leads to cognitive decline. The study evaluated CRISPR screens to illuminate the early cellular process that initiates tau pathology, as well as to identify the genes that either positively or negatively modify tau tangles. Our findings revealed that tau tangles begin with a leak in the nuclear envelope, which is the protective barrier surrounding the cell nucleus and its genetic materials.
Since the impact of nuclear envelope integrity on tau aggregation had not previously been appreciated, this study has revealed potential targets for therapeutic innovation, with confirmatory preclinical in vivo studies already underway.
Timelapse showing images of live cells revealing nuclear leakage
Our Regeneron Genetics Center (RGC®) has a singular mission: genetics to therapeutics, designed for all. Our geneticists and colleagues in bioinformatics, clinical research and other disciplines complement our groundbreaking and ambitious work by identifying genetic mutations linked to human diseases that Regeneron colleagues can then target for therapeutic discoveries. As we celebrate the RGC’s 10th anniversary, we continue to advance large-scale sequencing programs across the globe by partnering with leading human genetics researchers – now with more than 120 collaborations in 23 countries. View our collaboration model.
Since its inception, RGC has created one of the world’s largest and most diverse genomic databases, with around 2 million exomes sequenced as of the end of 2022. Paired with proprietary data analytics, human ingenuity, machine learning and artificial intelligence, we can quickly and effectively analyze data to make meaningful associations among genes and diseases.
In 2022, RGC scientists discovered a rare genetic loss-of-function mutation in the CIDEB gene associated with substantial protection from liver disease, including serious diseases such as nonalcoholic steatohepatitis (NASH) and cirrhosis. To make this discovery, RGC sequenced the exomes of more than 540,000 individuals across five ancestry groups and cohorts including the UK Biobank and the Geisinger Health System MyCode cohort.
By analyzing this genetic data in conjunction with deidentified health records, RGC researchers found that individuals who carry loss-of-function mutations in one of two copies of the CIDEB gene had an approximately 53 percent reduction in the risk of nonalcoholic liver disease and approximately 54 percent reduction in the risk of nonalcoholic cirrhosis. The study also found that CIDEB mutations had greater protective associations in individuals with obesity or type 2 diabetes, who are traditionally at higher risk for NASH, compared to individuals without these conditions. Thanks to these genetic discoveries, Regeneron is researching therapeutic approaches to mimic the genetic variations, including RNA interference, in collaboration with Alnylam Pharmaceuticals, Inc.
“The unprecedented protective effect that these CIDEB genetic variants have against liver disease provides us with one of our most exciting targets and potential therapeutic approaches for a notoriously hard-to-treat disease for which there are currently no approved treatments.” - Aris Baras, M.D., Senior Vice President and Head of the Regeneron Genetics Center
Our Regeneron Genetics Center team is working to build the world’s largest genomic database, create novel tools to mine endless possibilities in genetics research and spark the potential in genetics medicine.
Regeneron’s cutting-edge technical toolkit is powered by human genetics and biology, including VelociSuite and unique cell production technologies to scale new medicines for impact.
Our work inventing new medicines is built on a strong foundation of technology. We combine the deep scientific expertise of our team with proprietary technologies, such as VelociSuite. This enables us to accelerate the discovery and development of medicines beyond traditional methods.
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