The Multifaceted Future of Pharma – Chapter 2: Pushing the Boundaries of Research & Drug Discovery
Next-generation sequencing, protein-prediction tools, CRISPR, and increased knowledge of molecular mechanisms are yielding breakthroughs in drug discovery. Read on for thoughts on current trends, exciting technologies, and what might be possible in the future.
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?”
Looking to Next-Generation Sequencing – with Josh Mandel-Brehm, President & CEO, CAMP4 Therapeutics
“The Human Genome Project has had an incalculable impact on our understanding of disease drivers and potential therapeutic strategies. Our first read of the genome focused on characterizing the sequence of genes encoding proteins, which provided a roadmap to drug the genome. Armed with this information, our industry has developed an arsenal of therapeutics using multiple modalities that are primarily designed to either inhibit protein function or downregulate the expression of specific genes, including silencing target genes. Despite this progress, we lack deeper insights into the regulation of gene expression to develop a broadly applicable approach to upregulate gene expression and address the more than 1,200 diseases where even a modest increase in gene expression could potentially be transformative for patients.
“In the last decade, technological advancements in next-generation sequencing techniques have provided us with a growing understanding of the mechanisms underlying gene regulation, shining light on a completely new target class for drug development. Through the development of novel approaches and creative applications of proven therapeutic strategies, we have an incredibly vast opportunity to not only to directly address diseases caused by an under expressed gene, but also leverage this newfound capability to advance regenerative medicine as well as disease prevention and health optimization.”
“Digitalization has also revolutionized how the industry operates. Over the past decade, we have seen AI become a cornerstone in drug discovery, helping to identify promising therapeutic candidates much faster. Similarly, modeling and simulation have improved our ability to predict drug performance in humans, while automation is beginning to transform manufacturing processes, ensuring consistent quality and manufacturing efficiency. Remote and virtual disease management has also surged, driven by advancements in mobile technology and accelerated by the pandemic with greater demand for virtual consultations. In the next ten years, these digital innovations will continue to evolve, with AI, automation, and virtual healthcare becoming even more integral to the industry, and making personalized, data-driven care the norm.”
Getting Away From the Herd Mentality – with Hamish Ryder, CEO, Therapeutic Innovation, Cancer Research Horizons
“To truly revolutionize healthcare, the industry needs to move away from the herd mentality that favours the discovery and development of drugs in established areas. We need to rebalance our resources towards novel targets.
“Investing predominantly in known mechanisms – exemplified by the many anti-PD1s in development and the current clustering around GLP1 – creates head-to-head competition, drives down margins, and minimizes the potential for breakthroughs. The number of drug approvals in recent years may have increased, but that is not matched by improved performance for the pharmaceutical sector.
“Industry must be bolder about engaging with breaking science, and act on it sooner, either through direct interaction with academia or by collaborating with partners that operate in novel spaces. Regulatory agencies must go further to smooth the path for new approaches and first-in-class therapies. Breakthrough therapies and those that target smaller patient populations need additional incentives, such as patent term extensions, to encourage true innovation.
“Technological advances offer so much potential that we will not meet if the sector maintains this herd mentality. In an era of the coming of age of AI, novel therapeutic sub-modalities, and a deeper characterization of disease, incremental improvements in patient outcomes should no longer be considered a satisfactory result from the biopharma industry. We need to be aiming for step-change breakthroughs in patient benefit. For that, we need to leave the herd behind.”
