What role is precision medicine playing in transforming cancer treatment — and how are developers adapting?

Precision medicine is playing a large role in transforming cancer treatment by bringing individualized therapeutics to the patient. The driver is scientific advancements in therapeutic modalities, such as cell and gene therapies. Progress is also being driven by significant advances in data science.

To keep pace, developers are evolving how they work. Driving innovation in cancer treatment through precision medicine demands cross-functional collaboration across scientific disciplines and throughout the product life cycle. Teams can no longer operate in silos. For example, insights from gene therapy programs are informing cell therapy strategies, and monoclonal antibody experts are working alongside antibody–drug conjugate teams to optimize payload delivery and targeting.

At the core of precision medicine is data that link biology, therapy design, and clinical outcomes. To bring personalized cancer treatments to market, developers must integrate genomic, clinical, and manufacturing data to inform decisions across the development life cycle. This includes aligning analytical characterization, dose design, and supply chain planning with individual patient or disease subtypes. Success depends on coordinated, cross-functional teams that apply modality-agnostic thinking and deep technical expertise to translate complex biological insights into scalable, patient-specific therapies.

The use of patient-derived organoids (PDOs) in lead molecule selection offers several significant advantages. PDOs capture the biological and genetic characteristics of the original tumor, facilitating more accurate predictions of patient drug responses. Unlike traditional cell lines or animal models, organoids preserve tumor heterogeneity and structural complexity, providing a more clinically relevant evaluation of drug efficacy and toxicity. By modeling drug responsiveness within three-dimensional cell–cell interactions and the surrounding microenvironment, organoid-based studies allow drug developers to validate target specificity with greater precision.

Samsung Organoids, our PDO-based drug screening services, advances precision medicine strategies by identifying co-mutations in non-responding organoids. Derived from over 10 different cancer types, the Samsung Organoids Biobank provides information on specific mutations and their protein expression profiles. High-quality clinical data sets help elucidate the mechanistic relevance of drug responses.

This approach not only accelerates the development of precision medicine but also reduces the risk of failure in clinical trials, ultimately saving time and costs in the drug development process. Furthermore, PDOs have the potential to replace or supplement animal models, offering a more ethical and physiologically relevant preclinical platform.

Advances in cancer biology, knowledge of the immune microenvironment, and testing technologies now enable the creation of detailed molecular and genetic tumor profiles, including biomarker identification. Precision medicine has revolutionized cancer treatment by moving away from a one-size-fits-all approach to highly targeted, personalized strategies, resulting in treatments with greater effectiveness and less toxicity.

In response to this paradigm shift, pharmaceutical companies, investigators, and regulatory agencies now prioritize therapies linked to specific biomarkers. Clinical trials focus on smaller, more defined biomarker-selected populations, identified using new technological companion diagnostics, rather than employing blockbuster drugs across broad patient populations. New models for clinical trial designs have emerged, which enable adaptation throughout the development of the compound. For instance, basket trials target common molecular alterations across different tumor types permitting “tumor-agnostic approvals” and allowing new drugs to reach patients faster. Dose-optimization requirements established by regulatory agencies are also helping researchers to reach the right balance between drug doses that are toxic and doses that are truly needed to achieve a specific goal. Collectively, these innovations are advancing the effectiveness and precision of cancer treatment.

Next-generation sequencing (NGS) has unleashed the full force of precision medicine, transforming oncology forever, shifting us from a one-size-fits-all model to targeted, data-driven decisions. Yet too often, these critical clinical choices are stalled by the long turnaround times associated with molecular profiling. In 2025, this is an avoidable reality, especially for patients with advanced cancer, when every moment truly matters.

To unlock the full potential of precision therapies, we must bring genomic testing closer to the point of care, particularly into community hospitals, where most cancer patients are treated. Indeed, the demand for in-house NGS is growing rapidly, and for good reason. Patient groups have more recently played an increasing part in advocating for fast biomarkers testing. When testing happens locally, turnaround times can shrink from weeks to as little as 24–48 hours. The implications for patients and clinicians are profound: decisions become faster, better informed, and more confident.

But speed alone is not enough. Instruments enabling ultra-rapid NGS must be both technically reliable and operationally accessible. That means fully automated, user-friendly platforms that fit seamlessly into existing workflows; systems that the current staff can operate without the need for highly specialized expertise. Still, a machine capable of delivering fast results is only truly valuable if it can be used consistently, day after day. That solution now exists.

The next challenge is scale. Access to rapid genomic testing still varies dramatically between regions, creating unacceptable disparities, even within the United States. Closing this gap requires building an even stronger bond between laboratories, medical associations, and payers supporting validation efforts and aligning with evolving regulatory requirements to ensure both quality and sustainable widespread adoption.

The true promise of precision medicine will be fulfilled when rapid genomic testing becomes the rule, not the exception, available to every patient, in every setting, without delay. That is the future we are committed to building

Cancer care is shifting from non-selective cytotoxic drugs toward targeted therapies driven by molecular profiling. Genomic and immunologic characterization now allows treatment to be matched to the unique drivers of disease in each patient. To make this possible, modalities must be selective, tunable, and able to accommodate patient heterogeneity. Drug discovery alone is insufficient; platforms must support complex molecules and advanced delivery systems while maintaining safety, efficacy, and reproducibility. At CPTx, we focus on single-stranded DNA vectors as a foundation for precision medicine. ssDNA supports episomal expression with low innate immune activation, making it compelling for personalized approaches. Compared with mRNA, which yields short-lived expression, episomal ssDNA can sustain activity for longer periods without genomic integration, offering a balance of persistence and safety well suited to in vivo applications. Our scalable platform enables rapid transition from sequence design to clinical-grade material, a critical factor when tailoring therapies to patient-specific profiles. We are extending this platform into in vivo CAR-T, where ssDNA vectors are delivered directly to immune cells to program tumor targeting inside the patient. This brings together precision vector design, controlled expression, and delivery science into a single system. The convergence of ssDNA and in vivo CAR-T represents the next phase of precision oncology, focused on building the capabilities required to deliver individualized therapies reliably and at scale.

Targeted alpha therapy (TAT), a form of radionuclide therapy, is transforming cancer treatment by using alpha-emitting isotopes to deliver highly potent, precise radiation directly to tumor cells. Alpha particles cause irreparable double-stranded DNA breaks, driving tumor cell death and increasing the likelihood of meaningful clinical responses by offering significantly greater cytotoxicity than traditional beta-emitters, which primarily induce less effective single-strand breaks.

For years, TAT’s potential to treat solid tumors has been recognized, but progress was stifled by limited access to high-quality alpha-emitting isotopes. Developers are now addressing this bottleneck by building end-to-end infrastructure that integrates isotope production, radiolabeling, and scalable manufacturing. By ensuring reliable, clinical-grade supply, these efforts accelerate clinical trials, expand research access, and enable rapid development of next-generation radiotherapeutics. Overcoming the supply challenge allows precision nuclear medicine to reach its full potential, delivering safer, more effective therapies to patients with aggressive, treatment-resistant cancers.

Precision medicine has shifted the focus of cancer treatment development away from drugs to treat a specific cancer defined by its organ of origin toward treatment that is focusing on phenotypic characteristics, including the molecular make-up. This has led to new formats of clinical trials, such as basket or umbrella trials, that define their inclusion criteria based on molecular targets or other biomarkers. In the preclinical space, patient-derived models that mimic the complete bandwidth of the disease play an incremental role in the development of new targeted drugs and predictive biomarkers, both for response and patient selection. Besides their relevance as additional read-out in co-clinical trials, patient-derived xenografts (PDX) and patient derived organoids (PDO) serve as valuable tools in the development of innovative compounds. As both platforms are characterized by retaining molecular signatures and clonal diversity of the donor patient, they enable drug testing in a clinically relevant system. With the latest developments in precision medicine, the identification of druggable targets, not only on tumor cells but other cells in the tumor microenvironment (TME), cancer modelers have to adapt their preclinical platforms to include immune cells and other components of the TME. Interestingly, the presence of different cell types not only shapes the efficacy of these new drugs but also influences the efficacy of drugs that were intended to target tumor cells directly. With the use of more sophisticated, fully human preclinical models, the identification of the most promising drug candidates as well as combination strategies has improved tremendously.

The way we treat disease today is significantly different than approaches at the beginning of the millennium. Long gone are the days of one-size-fits-all approaches; today, treatments are becoming highly specialized and targeted for each patient, depending on the biology of their disease. Antibody–drug conjugates (ADCs) are emerging on the front lines of the battle against cancer and other diseases.

As the market for this novel modality grows and the medical community adopts them as first-line treatments, it may mean that fewer patients need invasive treatments like chemotherapy and radiation that cause significant side effects.

ADCs have experienced remarkable growth, with eight out of the 15 total commercially approved ADCs being approved in the past five years. There are over 200 new ADCs in active clinical trials encompassing a wide variety of tumor types. For clients interested in working with fewer suppliers, our company offers streamlined CDMO services for linker–payloads and conjugation across its global network and deep expertise to tailor each molecule’s unique journey, while creating the dynamic client partnerships drug manufacturers need to help reach their critical milestones. Our goal is to provide the tools/offerings and services needed for a world where more cancer patients become cancer survivors.

Precision medicine hinges on an ability to identify patients most likely to respond to a targeted therapy. With a disease as heterogeneous as cancer, insights at single-cell resolution are proving uniquely capable of uncovering patient-specific disease characteristics that can be used to guide therapy and prevent relapse.

Recent examples include assays that can track the clonal evolution of myelodysplastic syndrome, with the potential to identify specific targets for intervention to disrupt the development of cancers, as well as measurable residual disease assays intended to predict relapse and drive treatment recommendations for diseases like acute myeloid leukemia (AML).

Precision medicine is increasingly playing a pivotal role in transforming cancer treatment. By leveraging a targeted approach, treatment can be stronger and also more effective, as it preserves the normal function of cells and tissues. This approach minimizes adverse effects and maintains patients’ overall strength throughout treatment.

Developers are adapting by both leveraging new technologies and innovations to become more targeted, and by focusing on prevalent treatments and advancing them to become more precise. For example, radiation therapy is one of the most widely-used cancer treatments, with the most common delivery being external beam radiation therapy (EBRT). EBRT is constrained by low doses, which are used to mitigate potential harm to healthy tissues and organs. Consequently, it necessitates frequent treatment sessions, which can be inconvenient and time-consuming for the patient. At Plus Therapeutics, we have developed a targeted radiotherapeutic that addresses these limitations. Our drug candidates allow for the precise delivery of 15–20 times the radiation dose directly into the tumor compared with traditional EBRT while sustaining cell and tissue function.

Precision medicine is transforming cancer treatment by enabling therapies tailored to each patient’s genetic tumor profile. But speed is critical — every day counts in the therapeutic window. Traditional plasmid-based DNA production struggles to keep pace, especially for complex, patient-specific designs. With ENFINIA™ cell-free DNA, Elegen delivers NGS-verified, IVT-ready templates in days, helping developers move from design to manufacturing faster and make personalized cancer therapies a reality at scale.

Developers are playing a critical role in transforming precision medicine by reimagining how cell therapies are manufactured and delivered. One example is the move toward patient-adjacent manufacturing. By building GMP facilities next to medical centers, developers reduce logistical complexity, conserves resources, shortens vein-to-vein time, and minimizes thaw cycles, ultimately preserving potency and improving patient outcomes.

In response to this, CTMC has created the Network Alliance with cancer centers around the world that operates through a hub-and-spoke model. By offering specialized training, educational programs, and readiness assessments, these collaborations empower hospitals and treatment centers to adopt cell therapies more effectively. Together, these programs not only accelerate access to lifesaving treatments but also build the scalable infrastructure needed to bring precision medicine to more patients worldwide.

Precision medicine is transforming cancer treatment by shifting from a “one-size-fits-all” approach to one that is tailored to each patient’s cancer. On the one hand, the integration of multi-omics data enables the creation of detailed genetic and molecular profiles of patients. On the other hand, technologies like CRISPR and mRNA allow developers to rapidly adapt treatments to new mutations or rare cancer subtypes, with small-scale, modular, and fast manufacturing.

CRISPR has exhibited potential in boosting the efficacy of autologous cell therapies like CAR-T cells, reducing tumor growth and sensitizing cancer cells to treatments, by selectively activating or deactivating genes within the tumor cells and microenvironment. This technology is also being harnessed in precision disease modeling and drug screening to validate the best tumor antigens as well as identify effective treatments and resistance mechanisms.

With 60 treatments in development and 120 cancer vaccine trials across various malignancies, mRNA has enabled unprecedented advances in oncology. Notable examples of personalized cancer vaccines encoding patient-specific neoantigens are BioNTech’s BNT122 (Autogene Cevumeran) and Moderna’s mRNA-4157/V940, which have shown promising recurrence-free survival in pancreatic cancer and melanoma, respectively.

While the transformation in individualized care is enabling more effective and less toxic therapies, costs remain a challenge.