Precision oncology has reshaped how we diagnose, treat, and manage cancer. By moving beyond a one-size-fits-all approach, it allows therapies to be tailored to the unique biological characteristics of each patient’s tumor. Once considered a novel frontier, precision oncology is now a foundational part of cancer care—driving better outcomes, improving clinical trial design, and expanding access to more personalized treatments.
What Is Precision Oncology and Why Is It Important?
Cancer treatment has evolved from relying solely on tumor location—such as lung, breast, or colon—to focusing on molecular characteristics that drive tumor growth. While traditional treatments like chemotherapy and radiation remain important, they are often applied broadly across diverse patient populations—despite important biological differences that can influence treatment response.
Precision oncology represents a major shift toward biology-based care. By analyzing a tumor’s genetic mutations, biomarkers, and molecular pathways, clinicians can identify therapies more likely to benefit a specific patient—while avoiding those unlikely to help.
This approach not only improves outcomes and reduces toxicity but also transforms how trials are designed. With biomarker-driven enrollment strategies, biotech companies can better identify eligible patients, reduce variability, and improve trial efficiency. CRO partners help navigate these complexities by supporting biomarker integration, data interpretation, and regulatory alignment.
How Does Precision Oncology Work?
At the core of precision oncology is molecular profiling—analyzing the DNA, RNA, and protein expression of a patient’s tumor to identify key biomarkers that influence cancer growth and treatment response. The increasing use of molecular profiling in cancer has enabled more precise, data-driven approaches to both treatment selection and clinical trial design.
These insights allow clinicians and drug developers to:
- Classify tumors not only by location, but by molecular subtype
- Characterize a tumor’s molecular traits or biomarkers to inform diagnosis, prognosis, and therapeutic strategy
- Leverage the tumor-specific insights to align patients with therapies that target their tumor’s vulnerabilities
For background on how tissue‑agnostic approaches helped shift focus from tumor site to molecular characteristics, see our historical perspective on tissue‑agnostic therapeutics and biomarkers.
Advances in next-generation sequencing and biomarker discovery have made this process faster and more efficient, enabling more timely and data-driven treatment decisions in both clinical care and trial enrollment.
Precision Oncology Treatments: Types and Technologies
Precision oncology includes several types of cancer treatments and diverse therapeutic approaches—many of which are already integrated into standard care—designed to target cancer more precisely based on molecular and immunologic features, including genetic mutations, antigen expression, and tumor microenvironment dynamics.
Targeted Cancer Therapies
Targeted therapies are designed to inhibit specific molecular alterations that drive cancer growth, such as activating mutations or overexpressed receptors. Examples include EGFR inhibitors for non-small cell lung cancer and HER2-targeted therapies for HER2-positive breast cancer, which block specific overexpressed or mutated receptors that drive tumor growth. Targeted therapy for cancer minimizes damage to healthy cells by focusing on tumor-specific drivers, improving clinical outcomes in selected patient
Immunotherapy
In discussions around immunotherapy vs chemotherapy, a key distinction lies in how each approach targets cancer—traditional chemotherapy attacks all rapidly dividing cells, while immunotherapy activates the body’s immune system to seek out and destroy tumor cells. Immunotherapy engages the patient’s own immune system to recognize and eliminate cancer cells. A key strategy involves checkpoint inhibitors, which block immune-regulating proteins such as PD-1, PD-L1, or CTLA-4. These proteins normally act as safeguards to prevent excessive immune activity—but many tumors evade immune detection by overexpressing PD-L1, effectively turning off the immune response. By interrupting these inhibitory signals, checkpoint inhibitors help restore immune activity against the tumor. Therapies targeting PD-1/PD-L1 have shown durable responses in cancers such as melanoma, non-small cell lung cancer, and bladder cancer. To learn more, see our overview of immunotherapy for cancer treatment.
Antibody-Drug Conjugates (ADCs)
Antibody-drug conjugates (ADCs) are targeted therapies that link a monoclonal antibody to a cytotoxic payload via a specialized chemical linker. The antibody binds to a tumor-associated antigen, is internalized by the cancer cell, and then releases its drug payload to induce cell death—minimizing impact on surrounding healthy tissue. The specificity of the antibody and stability of the linker are critical to the safety and efficacy of the therapy. Several ADCs are now approved, including therapies for HER2-positive breast cancer, urothelial carcinoma, and certain hematologic malignancies. For deeper insight into the evolution of ADCs and their impact on cancer treatment, see our ADCs expert overview featuring Dr. Brian Barnett.
Radionuclide Therapies
Radionuclide therapies use radioactive isotopes linked to tumor-targeting molecules—such as antibodies or small ligands—to deliver radiation directly to cancer cells. Once bound to the tumor-associated antigen or receptor, the radiopharmaceutical delivers localized radiation that damages tumor DNA while sparing surrounding healthy tissue. This approach is especially useful in metastatic or treatment-resistant cancers. Approved therapies include radioligand treatments for prostate cancer targeting PSMA and neuroendocrine tumors targeting somatostatin receptors.
Cell and Gene Therapies (CGTs)
CGTs represent some of the most personalized approaches in oncology. A leading example is CAR T-cell therapy, in which a patient’s own T cells are collected, genetically modified to express a chimeric antigen receptor, and reinfused to target cancer cells. These therapies have demonstrated remarkable efficacy in hematologic malignancies, including certain leukemias and lymphomas. Research is ongoing to expand their use into solid tumors, while newer platforms are exploring donor-derived (allogeneic or “off-the-shelf”) therapies that eliminate the need to use each patient’s own cells, potentially improving scalability and accessibility. To explore how cell-based therapies are transforming cancer care, see our overview of cell therapy and how it works.
For a deeper look at CAR T-cell therapy in hematologic cancers, see our white paper on CAR T advances.
Together, these modalities reflect the growing diversity of approaches in precision oncology—ranging from targeted therapies and immunotherapies to antibody-drug conjugates (ADCs), radionuclide therapies, and emerging cell-based platforms.
The Role of Technology in Precision Oncology
The continued advancement of precision oncology is closely tied to enabling technologies:
- Genomic testing for cancer, including next-generation sequencing, has become faster, more scalable, and more cost-effective, enabling real-time molecular characterization of tumors in both research and clinical care.
- AI in oncology is increasingly central to data-driven care. Artificial intelligence and machine learning support interpretation of complex molecular data, power predictive modeling, and aid clinical decision-making.
- Data integration platforms bring together clinical, genomic, and real-world data to support patient matching, treatment optimization, and trial design.
These tools are also transforming trial design—moving beyond tumor-type-based enrollment to biomarker-driven stratification, where patients are matched based on genetic or molecular profiles. This approach helps ensure that participants are more likely to benefit from the treatment being studied, accelerating recruitment and increasing the likelihood of trial success.
In addition, companion diagnostics—such as genomic or protein-based tests—are increasingly used to identify the specific cancer biomarkers required to determine whether a patient is eligible for a targeted therapy. As these tests become more central to trial design, regulatory agencies are placing greater emphasis on the analytical and clinical validation of these diagnostic tools.
CROs that understand how to operationalize biomarker-driven trials, including diagnostic integration and evolving regulatory requirements, play a key role in helping biotech companies navigate this complexity.
Benefits of Precision Oncology for Cancer Patients
Precision oncology offers meaningful advantages that are already transforming cancer care across a wide range of tumor types:
- Improved outcomes: Patients receiving biomarker-driven treatment often experience longer survival and higher response rates.
- Fewer side effects: Targeted therapies and immunotherapies are generally more selective, reducing damage to healthy cells.
- Faster treatment decisions: Molecular profiling enables clinicians to avoid trial-and-error prescribing by identifying the most likely effective treatments earlier in the process.
- More informed choices: Patients and their care teams can make decisions grounded in a deeper understanding of the tumor’s biology.
As precision oncology reshapes the treatment landscape, it’s delivering more effective therapies and personalized cancer treatment to patients—and enabling faster, more confident decisions for the biotech companies working to bring those new therapies to the patients who need them.
Challenges and Future Directions in Precision Oncology
While precision oncology continues to evolve rapidly, several challenges remain—particularly when it comes to broader clinical implementation and trial scalability.
One key barrier is the limited availability and adoption of molecular testing across all treatment centers. When testing capabilities vary widely by institution or region, it can hinder consistent patient identification and complicate site selection for biomarker-driven clinical trials. For biotech companies, this can translate into slower enrollment, reduced access to eligible patients, and variability in data quality.
Another challenge is the lack of well-characterized, actionable biomarkers in many tumor types. Although some cancers have established molecular targets, others still lack reliable predictors of response—limiting opportunities for targeted therapy development.
At the same time, the growing complexity of multi-omic data requires advanced analytic platforms and specialized expertise to support real-time interpretation and decision-making, both in the clinic and during drug development.
Despite these challenges, the outlook for precision oncology remains highly promising. Innovation continues to expand the boundaries of what’s possible, with several areas showing particular momentum:
RNA-based therapies
These emerging treatments aim to disrupt cancer growth by targeting disease-driving mechanisms at the RNA level—such as by silencing mutant transcripts, correcting splicing errors, or degrading aberrant RNA molecules. By intervening earlier in the gene expression process, they offer new therapeutic avenues for cancers with molecular drivers that have historically been difficult to target.
Bispecific antibodies
These next-generation immunotherapies are engineered to simultaneously engage a tumor-associated antigen and an immune effector cell—most often a T cell—facilitating direct tumor cell recognition and destruction. While already demonstrating clinical success in hematologic malignancies, bispecifics are increasingly being adapted for the complexities of solid tumors.
Tumor microenvironment (TME) modulation
The TME includes not only cancer cells but also surrounding immune cells, stromal cells, blood vessels, and signaling molecules that can promote tumor survival and resistance. Emerging therapies aim to reprogram this environment—boosting immune infiltration, reversing immunosuppressive signals, and dismantling resistance-supporting structures—to improve therapeutic response.
While precision oncology continues to evolve rapidly, key challenges remain—including limited access, biomarker variability, and therapeutic resistance. Yet as scientific advances accelerate, the future lies in overcoming these hurdles through smarter trial designs, improved diagnostic strategies, and next-generation therapies that expand the range of treatable tumors, refine patient stratification, and bring more effective options to those who need them most.
Conclusion: Precision Oncology in Practice
Precision oncology is no longer an emerging concept—it’s a cornerstone of modern cancer care and drug development. By using molecular insights to guide patient selection, therapeutic strategy, and trial design, it offers a more targeted, efficient, and impactful path forward. As science continues to evolve, biotech companies have an opportunity to lead the next wave of innovation—advancing smarter treatments, faster trials, and better outcomes for patients worldwide.
At Allucent, we support biotech companies in navigating the complexities of precision oncology—from biomarker strategy and diagnostic integration to patient selection and regulatory alignment. Our oncology experts work across a broad range of innovative modalities, helping accelerate smarter trial designs and bring targeted therapies to market faster. Learn more about our oncology drug development services.
About the Allucent Editorial Team
The Allucent Editorial Team is composed of experienced professionals in drug development, spanning preclinical research, clinical trials, regulatory strategy, and scientific communications. As part of Allucent’s content team, we collaborate with subject matter experts to deliver insightful, industry-leading perspectives on emerging trends and scientific advancements. Our goal is to provide biotech innovators with clear, informative content that supports strategic decision-making in a complex development landscape.
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