Genes have gone mainstream, and genetic medicines are beginning to flip the drug industry inside out.
The human genome is driving healthcare innovation, and the biopharmaceutical industry is increasingly focusing research and development centered on understanding the role of genetics in health and disease. The explosion in our understanding of human genetics and disease biology—combined with advanced technology—may hold the key to addressing some of the most intractable diseases at their very source.
The Path from Proteins to Genes
At Regeneron, we have spent over three decades honing a deep expertise in disease biology and genetics. By rigorously pursuing an understanding of pathobiology—the mechanisms by which diseases happen—our portfolio of antibody medicines has treated countless patients with difficult-to-treat diseases.
This same drive to gain a true understanding of disease origins has naturally led us to advance our expertise into genetic medicines.
“An antibody works by binding to something on the surface of a cell or something that is circulating in the human body,” says Christos Kyratsous, Ph.D., Senior Vice President and Co-Head of Regeneron Genetic Medicines. “With genetic medicines, we can actually send a therapy inside a cell to change how disease-causing genes are being expressed to potentially treat or prevent disease. These can include introducing genes that may be missing, removing genes that may be mutated or extra, or changing how genes are being expressed.”
Taking a Versatile Approach
Research and development in genetic medicines begins with a thorough understanding of disease biology, which involves identifying and characterizing both disease-causing and -preventing (protective) genetic changes.
Regeneron Genetics Center (RGC) was founded in 2013 to harness the power of human genetics. “In the decade since its inception, the work done at RGC has provided us with one of the world’s largest and most diverse human genomic databases,” notes Aris Baras, M.D., Head, Regeneron Genetics Center and Senior Vice President, Co-Head of Regeneron Genetic Medicines, “which we and our collaborators can use to identify and validate novel gene targets for genetic medicines in the future.”
“While the use of antibodies remains our cornerstone,” Aris continued, “our productivity in genetic discovery coupled with the availability of advanced biomedical technologies necessitated that we also become a genetic medicines company.”
And today, we are expanding our toolbox and expertise, leveraging technologies that enable us to modify genes – silencing, adding or editing – to support a growing portfolio of genetic medicines:
- siRNA Technology: leveraging technologies like small interfering RNA (siRNA) to switch off expression of genes that could trigger disease inside cells in a process called RNA interference (RNAi).
- CRISPR Technology: using CRISPR-based gene editing to deliver molecular machinery to specific cells for precise gene modification.
- Delivery and payloads: uses genetically engineered viruses called adeno-associated viruses (AAVs) and other delivery mechanisms – for example, lipid nanoparticles or our antibody expertise to target tissues with greater specificity – to get inside target cells and deliver genetic material
Access to new genetic target insights and multiple technologies, all in one place, means we can apply the best approaches for each disease area: more targets combined with more technologies means more meaningful new therapies.
Collaborating the Regeneron Way
Taking the very best approach also means seeking out the best expertise, even if that falls outside of our walls. With an emphasis on the open exchange of ideas and technologies to drive innovative solutions, our versatile approach carries over to our external collaborations, as well.
Since 2016, we have accelerated CRISPR-based gene editing therapies with Intellia Therapeutics and are now advancing our programs in hematology as well as cardiovascular, neurological and muscular diseases. In 2019, we began a collaboration with Alnylam Pharmaceuticals to develop RNAi therapeutics for a broad range of disease areas, with medicines targeting the liver and the brain already in clinical trials. Most recently, in 2023 we acquired Decibel Therapeutics, cementing a long-standing collaboration and expanding our auditory program as we continue development of genetic medicines for profound, congenital hearing loss.
Regeneron is rooted in the power of genetics, technology and collaboration—the blueprint for our antibody portfolio and now, genetic medicines. Unlocking innovation to deliver life-altering therapies to people in need remains our singular vision, with more avenues than ever that will shape the future of medicine.
