The Power of Genetics

What we asked: “Looking ahead to the next 5–10 years, what will be the key disruptors and/or what can be improved upon in the pharma industry?”

Response from: Gary Herman, Senior Vice President and co-head, Regeneron Genetic Medicines Clinical Development Unit

Drug discovery and development is incredibly difficult under any circumstances, but more so when the biology underpinning disease is unknown and the approaches taken are largely empirical. Having the ability to understand the human genome is one of the greatest forces that changes the odds of making breakthrough discoveries. As such, the biopharma industry is increasingly focusing R&D 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 and truly gives us a fighting chance.

R&D in genetic medicines creates a real opportunity to develop a thorough understanding of disease biology, which involves identifying and characterizing both disease-causing and -preventing (protective) genetic changes. Integrating genetic data into drug development continues to accelerate the discovery process, starting with sequencing genomes at scale, which allows for the identification of novel targets that are key drivers of disease. Alongside this, new technologies are now enabling us to modify these genes – silencing or deleting, adding, or editing – that have the potential to not only better address diseases with high unmet needs, but also yield potentially curative treatments.

While genetic medicines have come a long way in transforming our approach, challenges remain, including ensuring therapeutic agents reach their intended targets within the body without affecting other tissues. Many current approaches involve systemic administration of viral vectors carrying a gene of interest. With the liver filtering and intercepting therapeutic agents before they reach their targets, however, this often necessitates higher doses of viral vectors, which could cause toxicity and adverse events. 

It will be critical to leverage multiple technologies to tackle persistent delivery challenges. As one example, at Regeneron, we're leveraging our legacy in protein therapeutics and the development of fully human, highly specific antibodies to develop targeting strategies to guide genetic medicines directly to the specific cells or tissues that are underpinning the disease. This is accomplished by attaching antibodies against cell- or tissue-specific receptors (e.g. a muscle specific receptor for a disease driven by an abnormal protein expressed in muscle) to the viral delivery vectors, allowing therapeutic payloads to be introduced into desired cells with high precision – thereby bypassing the liver and allowing for lower therapeutic doses, potentially reducing unwanted toxicity and side effects.

Over the next ten years, the refinement of delivery systems will be crucial in propelling gene editing technologies into the mainstream, addressing a broader range of diseases, with increased selectivity and reduced off-target effects. This is especially critical to those affecting complex systems, such as the central nervous system, where precise targeting is paramount. Ultimately, the marriage of multiple tools and technologies within the platform of genetic medicines, combined with careful and rigorous clinical investigation, holds the potential to transform the healthcare landscape, offering more personalized, targeted treatments once thought to be out of reach.

Read over 100 other views on the future of the pharma industry on our special web page.