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ABOUT THE REGENERON GENETICS CENTER (RGC)

Date post: 04-Feb-2022
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The goal of the RGC is to improve patient care by using genomic approaches that speed drug discovery and development through identifying new drug targets and validating existing programs. WHAT THE RGC DOES As one of the world’s most comprehensive genetics databases, the RGC uses state- of-the-art automation and cloud-based informatics to sequence and analyze exomes Pairs sequenced data with de-identified real-world electronic health records and other clinical datasets to make genetic and disease associations to: Allows Regeneron to potentially speed and optimize drug development by applying findings to initiate and validate clinical explorations and inform ongoing translational medicine approaches across the company OUTCOMES TO DATE Identify novel drug targets Validate indications and screen for safety considerations in existing development programs Pursue pharmacogenetics (the effect of genetics on drug response) and how to apply precision and personalized medicine approaches to treating diseases ABOUT THE REGENERON GENETICS CENTER (RGC) “Scientifically and medically, it’s pretty exciting. As far as I’m aware, it’s the largest clinical sequencing undertaking in this country so far by a long shot.” DR. LESLIE G. BIESECKER, chief of the Genetic Research Branch at the National Human Genome Research Institute Aiming to Push Genomics Forward in New Study “There are sequencing efforts underway elsewhere, of course, but none matches Regeneron’s for its combination of size, speed, diversity of samples, and detail of their accompanying health records.” Regeneron: The Best Bet in Biotech Stocks Sequencing at rate of 200,000 exomes per year, with DNA from more than 200,000 people sequenced as of August 2017 Identified hundreds of novel candidate disease genes across range of diseases and traits Predicted loss of function variants identified for virtually all of Regeneron’s target genes Published actionable discoveries in peer-reviewed publications including New England Journal of Medicine and Science The RGC is a wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc. REAL WORLD APPLICATION Genetic and therapeutic antagonism of ANGPTL3 in humans and of ANGPTL3 in mice was associated with decreased levels of all three major lipid fractions and decreased odds of atherosclerotic cardiovascular disease. FE Dewey. Genetic and Pharmacologic Inactivation of ANGPTL3 and Cardiovascular Disease. N Engl J Med 2017; 377:211-221. JACK HOUGH, Senior Editor, Barron’s
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The goal of the RGC is to improve patient care by using genomic approaches that speed drug discovery and development through identifying new drug targets and validating existing programs.

W H AT T H E R G C D O E SAs one of the world’s most comprehensive genetics databases, the RGC uses state-of-the-art automation and cloud-based informatics to sequence and analyze exomesPairs sequenced data with de-identified real-world electronic health records and other clinical datasets to make genetic and disease associations to:

Allows Regeneron to potentially speed and optimize drug development by applying findings to initiate and validate clinical explorations and inform ongoing translational medicine approaches across the company

O U T C O M E S T O D AT E

Identify novel drug targets

Validate indications and screen for safety considerations in existing development programs

Pursue pharmacogenetics (the effect of genetics on drug response) and how to apply precision and personalized medicine approaches to treating diseases

A B O U T T H E R E G E N E R O N G E N E T I C S C E N T E R ( R G C )

“Scientifically and medically, it’s pretty exciting. As far as I’m aware, it’s the largest clinical sequencing

undertaking in this country so far by a long shot.” DR. LESLIE G. BIESECKER, chief of the Genetic Research Branch

at the National Human Genome Research Institute

Aiming to Push Genomics Forward in New Study“There are sequencing efforts underway elsewhere, of

course, but none matches Regeneron’s for its combination of size, speed, diversity of samples, and detail of their accompanying health records.”

Regeneron: The Best Bet in Biotech Stocks

Sequencing at rate of 200,000 exomes per year, with DNA from more than 200,000 people sequenced as of August 2017

Identified hundreds of novel candidate disease genes across range of diseases and traits

Predicted loss of function variants identified for virtually all of Regeneron’s target genes

Published actionable discoveries in peer-reviewed publications including New England Journal of Medicine and Science

The RGC is a wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc.

REAL WORLD APPLICATION

Genetic and therapeutic antagonism of ANGPTL3 in humans and of ANGPTL3 in mice was associated with decreased levels of all three major lipid fractions and decreased odds of atherosclerotic cardiovascular disease.

FE Dewey. Genetic and Pharmacologic Inactivation of ANGPTL3 and Cardiovascular Disease. N Engl J Med 2017; 377:211-221.

JACK HOUGH, Senior Editor, Barron’s

O U R C O L L A B O R AT I O N M O D E LThe RGC is collaborating with leading academic institutions, government organizations and integrated medical systems. Our high-touch collaborative model is focused on working closely together to gather and analyze data, exchange expert perspectives and ultimately make discoveries that will lead to improved patient care.

R G C C O L L A B O R AT O R S

“For Geisinger, this relationship is about the potential to improve individualized patient care...we expect that

many of our patients and their family members will directly benefit from their participation in this research.”

DAVID H. LEDBETTER, PH.D.,

Executive Scientific Officer and Chief Scientific Officer, Geisinger Health System

“The relationship with Geisinger is a cornerstone of the effort the Regeneron Genetics Center is building,

which we believe can advance the goals of human genetics research and personalized medicine.”

GEORGE D. YANCOPOLOUS, M.D., PH.D.,

President and Chief Scientific Officer, Regeneron Pharmaceuticals

Data Analysis & Call Back

Studies

Actionable Scientific Discoveries

Peer reviewed publications (e.g. NEJM)Translational medicine (e.g. new targets, functional models, biomarkers, potential new therapies)Return of results to collaborators

Sequencing & Informatics

DNA Samples

De-identified Phenotypic

Data

C O L L A B O R AT O R R G C W O R K I N G T O G E T H E R

Shared Genomic Dataset

GeneralPopulation

Family-based Studies

Founder Populations

Disease Area Focus

500,000 participants to be sequenced

250,000 planned; nearly 100,000 sequenced to date

45+ Collaborators


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