With the adoption of medical advancements such as artificial intelligence (AI), new human-relevant alternatives to animal testing, genetic-based precision medicine, and predictive 3D culture-based assays, staying up-to-date on trends in drug discovery is more important than ever.
In this article, we examine how recent trends in drug discovery are transforming the time-consuming and resource-intensive drug development process.
The U.S. Food and Drug Administration (FDA) reports receiving more than 500 submissions that employed AI components from 2016 to 2023. AI systems are becoming more prevalent in multiple stages of drug development, according to an article in Nature Medicine, streamlining the complexities of trial-and-error experimentation.
An article published in Drugs in Context reports that AI can facilitate, optimize, and guide simulations for clinical trials and drug development. Drug developers are utilizing AI systems to identify promising drug targets, predict the bioactivity and toxicity of new molecules, plan syntheses of novel compounds, design and prioritize drug leads, and explore potential ways to repurpose existing drugs.
Moreover, AI tools are helping scientists predict protein structures and understand how drug candidates might bind to a protein of interest. Researchers are using machine learning and physics-based computational drug design to screen billions of compounds digitally. Other AI platforms aim to help drug developers identify safety concerns and focus their laboratory and clinical trial efforts on the compounds most likely to succeed.
For example, a study published in Scientific Reports revealed that researchers developed a deep learning model called AiKPro, which accounts for 3D-structural information from kinases and compounds to achieve more accurate kinase profiling. This tool demonstrated superior performance across various phases of kinase drug discovery, including binding affinity prediction, outperforming conventional tools such as AutoDock Vina. The researchers noted that AiKPro may help identify highly selective kinase inhibitors, pinpoint drug candidates for experimental validation, guide productive drug design, and fast-track drug discovery and development of targeted therapies.
Regulatory agencies in many countries require animal toxicity testing, sometimes called preclinical testing, to estimate the toxicity and safety of substances and prevent public health crises that may be caused by contaminated medicines. However, as the need for more humane and efficient methods for safety testing becomes widely understood, regulators and the scientific community are embracing new policies.
In 2023, the United States became the first country to pass a bill eliminating the requirement for animal toxicity testing in drug development. More recently, in April 2025, the FDA announced that its "animal testing requirement will be reduced, refined, or potentially replaced" with more effective human-relevant alternatives, including AI-based computational models and laboratory-grown organoid toxicity testing, also called New Approach Methodologies (NAMs).
Researchers can develop human-based lab models using 3D cell culture technologies such as Corning Matrigel® matrix and spheroid microplates, which can more closely mirror the complex tissues and organs of humans, revealing a more accurate picture of a drug's safety profile.
This approach promises to expedite the drug testing phase, enhance drug safety, minimize the use of lab animals, and reduce drug development times and costs.
In the clinic, precision medicine aims to select the most effective therapies for each patient based on their genetics, metabolic profile, lifestyle, comorbidities, environment, and other relevant factors. This quest for precision medicine is making its mark in preclinical drug discovery. Pharmaceutical companies are increasingly focusing on developing precision medicine drugs, such as those targeting specific mutations in patients' tumors. In fact, 43% of the 198 FDA-approved oncology drugs between 1998 and 2022 were precision oncology therapies guided by biomarker testing, according to a study published in Cancer Discovery.
Companies and academic labs are designing drugs for specific subsets of patients based on their genetic profiles or to specifically target their tumors' mutations. This precision is important in oncology because of the high heterogeneity of many tumor types, both within one patient and between patients who have the same type of cancer, such as melanoma or breast cancer.
For example, researchers designed the tumor-agnostic drugs larotrectinib, entrectinib, and repotrectinib to target NTRK gene fusion, which drives growth in a subset of tumors, regardless of the organ from which the tumor originated. FDA approved these drugs in 2018, 2019, and 2024, respectively, and also approved diagnostic tests to identify patients with the NTRK fusion protein who can benefit from these treatments.
Clinicians, industry and academic researchers, and regulators are working together to overcome challenges and pave the way for more gene therapies that can contribute to precision medicine.
Meanwhile, researchers are increasingly turning to 3D cell cultures for a more physiologically relevant view of drug candidates' potential for success and a more predictive understanding of drug effects in the body. Compared to 2D cell cultures, 3D cultures such as spheroids and organoids can better mimic the in vivo environment of cells within an organ or a tumor. This allows 3D cultures to capture aspects of the in vivo environment, such as concentration gradients for drugs, oxygen, and metabolites; cell-cell and cell-extracellular matrix (ECM) interactions; and heterogeneity within an organ or tumor—any of which can affect how drugs will function within the bodies of real patients.
In line with recent legislation that reduces the requirements for preclinical animal testing, FDA has signaled its support for alternative techniques, such as organoid models. These changes underscore the importance of developing robust 3D culture models that capture key aspects of human biology while mitigating the effects of interspecies differences.
In preclinical drug development, 3D culture enables researchers to move toward precision medicine and future therapies. In oncology, 3D cultures enable researchers to better replicate the complexity of real tumors, such as by culturing organoids that contain multiple cell types to mimic tumors with multiple cell types or multiple mutations in vivo. Exciting technological developments, like those detailed in the journal Stem Cells, enable the miniaturization of 3D cultures, including scaffold-supported organoids, so they can be used in high-throughput applications.
The increasing ease of using 3D culture systems also allows scientists who wish to perform more realistic assays to scale up patient-derived cells to the large volumes needed for drug screening or molecular studies, rather than waiting for rarely available clinical samples. Combined with miniaturization techniques, researchers can screen libraries with hundreds of thousands of compounds directly against 3D spheroids derived from patient cells.
Trends in drug discovery, like AI in drug development, animal-testing alternatives, precision medicine, and 3D cell cultures are opening new possibilities for research directions, including ways of using these strategies together. One example is research matching cancer patients to the most effective drugs by growing patient-derived tumor cells or tumoroids, which are then screened against approved drugs for effectiveness.
Life sciences manufacturers and scientists must collaborate to keep these exciting trends moving forward. Because technologies like organoids rely on complex inputs to be used in a scalable way, drug development labs are collaborating with companies that have expertise in setting up 3D culture systems to solve problems as they arise. In turn, manufacturers can collect feedback from industry and academic researchers on the problems they're trying to solve and design products accordingly.
Corning Life Sciences offers a wide variety of tools and expert support to help you solve drug development challenges. With more than 35 years as a leader and innovator in 3D cell culture, we offer gold-standard extracellular matrices (ECMs) like Matrigel matrix, established permeable supports, like Corning Transwell® and other innovative scaffold-free technologies like 3D culture spheroid flasks and microplates, along with a comprehensive line of products and tools for drug discovery.
Stay informed with Corning's extensive library of protocols, webinars, and articles to stay up-to-date on the latest trends in drug discovery, drug screening, and 3D culture tools. Learn more about 3D cell culture and drug discovery solutions.
