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The seeds of this transformation are already visible in the FDA’s latest drug approvals. In late 2024, precision medicine scored significant wins with targeted therapies such as Bizengri (zenocutuzumab-zbco) for specific lung and pancreatic cancers, while the approval of Attruby (acoramidis) for transthyretin-mediated amyloidosis highlighted the growing prominence of genetic considerations in cardiovascular disease.
On integrating genetic validation
Sandeep Kulkarni, MD
“In 2025, the cardiovascular field will continue to see a profound shift toward integrating genetic validation as a cornerstone of R&D,” says Sandeep Kulkarni, MD, co-founder and CEO of Tourmaline Bio. “The success of treatments like PCSK9 inhibitors and the anticipated outcomes from Lp(a) studies underscore how robust genetic evidence is reshaping drug development priorities.” He explains that as researchers refine our understanding of validated pathways—from IL-6 inhibition to next-generation lipid mechanisms—genetic data will increasingly serve as a prerequisite for advancing therapies into large outcomes studies. By tying approvals directly to known genetic drivers, developers can evaluate novel mechanisms and stand on firmer ground when asking investors for support. “This shift will ideally serve to de-risk novel mechanisms and increase R&D success rates,” Kulkarni adds. “As precision-driven approaches become the norm, we’ll see a new era of innovation in tackling chronic illnesses like cardiovascular disease, delivering more effective solutions to patients worldwide.”
This precision-medicine paradigm is not confined to cardiovascular diseases alone. The ripple effects of integrating genetic validation are being felt across a spectrum of chronic conditions. As the EY Biotechnology Report 2024 echoed, “Life sciences companies are now generating new treatments for chronic disease areas that had fallen out of fashion in favor of rare disease; conditions that impact large patient populations, like diabetes, obesity, mental health, and Metabolic Dysfunction-associated Steatohepatitis (MASH) are all benefitting from an R&D renaissance.”
Data as the new gold standard in biotech investments
The growing amount of data available—thanks to advances in genomics, electronic health records, and digital health technologies—has another significant aspect: it is leading to potentially better-informed bets from investors. Simultaneously, biotech investors have become more selective thanks to a variety of challenges including elevated interest rates, market volatility, and sector-level operational hurdles. “Investors have truly tightened their belts, leaving behind the pre-Covid era of investment frenzies in unvalidated approaches. In 2025, investors will continue to be discriminating and thoughtful,” says Caroline Loew, PhD, CEO of Mural Oncology.
EY, in the aforementioned report, reached similar conclusions, noting that savvy biotechs were still able to find capital. “Companies that have demonstrated maturity in these areas—strong scientific rationale, experienced management teams, and assets in innovative disease areas—have still been able to obtain venture investments,” it observed.
“With financing constrained, priority will go to proven approaches. Investments will be focused on more mature science,” notes Loew.
Meanwhile, oncology remains a central draw as it has been for years—but even this high-profile category is evolving.
“Early-stage programs will need to prove efficacy earlier in the program, and the bar will be higher to compete. While on one hand this could tighten the landscape, it also means only the most innovative will progress and the quality of programs under development could improve overall.
Oncology will remain competitive, and an area of strong investor interest. While ADCs and bispecifics are still in the spotlight for early-stage investors, don’t discount other players like those in immuno-oncology where we expect to see resurgent investor interest in 2025,” Loew adds.
RWD as a catalyst for clinical trial evolution
Seema Verma
“The future of clinical research lies in integrating real-world data (RWD) with clinical trial data to accelerate innovation. The line between clinical research and clinical care will blur, creating a virtuous cycle as data flows seamlessly from bench to bedside and back. By bridging this gap, continuous discovery and delivery can help to transform the way research and care come together—until it’s all just care. We will see more networks embed clinical trial opportunities and data directly within EHR platforms to transform research from a separate process into a seamless component of patient care—enabling clinical trials that never end. This model will allow for real-time patient recruitment, continuous data capture, and rapid trial initiation, making routine clinical interactions opportunities for discovery.
In 2025, patients will become partners in their care, or they will go elsewhere for it. While everyone talks about putting patients first, that reality does not exist everywhere today. Patients want to be empowered with more information about their health and their options. They want to know what trials they are eligible for and make decisions about participation. Patients also increasingly want to influence the design and objectives of clinical research as a condition for participation. The industries are recognizing how technology is empowering patients with information about their health and tools to track — in real time and over time — changes in bodily functions and performance, from sleep to exercise. Progress toward full interoperability is making it possible to assemble, store, and transmit increasingly complete, longitudinal patient medical histories – and patients will justifiably expect the right to own and manage those records, including decisions about who has access to parts or all of the information they contain.”
Filed Under: Biotech, Cardiovascular, Data science, Drug Discovery, Drug Discovery and Development, Genomics/Proteomics
