Can gene editing be used as a life-saving treatment? Could antiviral drugs that target human proteins be more effective than those than target the viruses themselves? The constant evolution of drug discovery is one of the most exciting aspects of modern medicine, and new research is leading to breakthroughs in precision medicine, cancer immunotherapy, neurodegenerative disease detection, and much more. Let’s take a closer look at eight key trends happening in drug discovery today.
Want an overview of important drug discovery breakthroughs to watch in 2025? Register today for our September 23 expert panel, where speakers will discuss specific technologies as well as their context within the larger drug research environment.
Driving E3 ligase discovery through commercial PROTAC momentum
PROteolysis TArgeting Chimeras (PROTACs) are small molecules that drive protein degradation by bringing together the target protein with an E3 ligase. To date, more than 80 PROTAC drugs are in the development pipeline, and over 100 commercial organizations are involved in this field of research. We’ve seen in the CAS Content CollectionTM, the largest human-curated repository of scientific information, a sharp increase in PROTAC-related publications in less than 10 years, which demonstrates their therapeutic potential. Cancer is the leading disease in PROTAC-related literature, but neurodegenerative, infectious, and autoimmune diseases are represented as well.
Despite the diversity of E3 ubiquitin ligases, however, most designed PROTACs act via one of four E3 ligases: cereblon, VHL, MDM2 and IAP. Efforts are now underway to identify new ligases and utilize others already known beyond the main four. These include DCAF16, DCAF15, DCAF11, KEAP1, and FEM1B.
New insights into the structure and functionality of different ligases could enable targeting of various proteins that were previously inaccessible, and it may lead to fewer off-target effects. Expect to see new PROTAC drug designs entering the preclinical pipeline as researchers continue expanding the E3 ligase toolbox.
Expanding probiotic use beyond gut health for systemic diseases
The human microbiome — the vast community of bacteria, viruses, fungi, and other microbes living in and on our bodies — plays a crucial role in maintaining health. Far from being passive bystanders, these microbial ecosystems influence digestion, immunity, mental health, and even chronic disease risk. Researchers are harnessing the power of gut bacteria to treat antibiotic-resistant infections, metabolic disorders, and mental health conditions.
For example, fecal microbiota transplants (FMT) have been approved by the FDA to treat recurrent Clostridioides difficile infections. As of 2025, over 180 microbiome-targeted therapies were in development for amany conditions. A greater understanding of the microbiome’s role in chronic diseases may also result in early-life interventions and dietary recommendations that are low-cost, long-term improvements to many aspects of human health.
Delivering precision oncology through radiopharmaceutical conjugates
Drug conjugates are innovative molecules that combine a targeting moiety (such as an antibody, peptide, or small molecule) with a potent therapeutic payload (e.g., chemotherapy agents, toxins, or radionuclides), enabling selective delivery to diseased cells while sparing healthy tissues. There are several types in existence, such as antibody-drug conjugates, and now researchers are making progress with radiopharmaceutical conjugates.
This form of nuclear medicine combines targeting molecules with radioactive isotopes for imaging or therapy. These conjugates offer dual benefits — real-time imaging of drug distribution and highly localized radiation therapy. For cancer treatments, radiopharmaceutical conjugates can reduce off-target effects and toxicity by directing drugs to specific cells. These drugs can also improve efficacy through better targeting of tumors with a lethal payload. We expect to see increased use of these theranostic approaches as several radiopharmaceuticals have entered late-stage clinical trials or received regulatory designations.
Scaling CAR-T therapy for solid tumors with next-generation platforms
Immunotherapy has become a pillar of cancer treatment, along with surgery, chemotherapy, and radiation. The number of immunotherapy drugs is constantly growing, and CAR-T therapies, which use a patient’s own genetically engineered cells to attack and kill cancer cells, have been found particularly effective. Yet their cost and thedevelopment time from an individual’s cells make them prohibitive for most cancer patients. Advances in allogeneic and armored CAR-T cells are overcoming problems of cost and scale as well as drug efficacy, and they may hold the key to expanding the use of these cancer treatment options to more patients:
- Allogeneic CAR-T: These are donor-derived or gene-edited cells that are faster and more affordable to develop than autologous (patient-derived) CAR-T cells. By providing an off-the-shelf option, these treatments could become more accessible to a larger pool of patients.
- Dual-target and armored CAR-T: Dual-target CAR-T cells recognize two antigens, while armored CAR-Ts are engineered to secrete cytokines or resist immunosuppression, which enhances efficacy and durability. Several dual-target CAR-Ts (e.g., AUTO1/22, CD19/CD22) are in clinical trials (CAR-T for pancreatic cancer; CAR-T for solid tumors), and armored CAR-Ts like ATA3271 are being developed to overcome tumor escape and exhaustion. These innovations aim to reduce relapse rates and improve outcomes in cancers with high antigen variability or immunosuppressive environments.
Enabling early diagnosis of neurodegenerative diseases with biomarkers
Biomarkers are measurable biological indicators in blood, tissue, or bodily fluids that reflect normal or pathological processes, and they play a pivotal role in detecting diseases at their earliest, most treatable stages. In cancer treatment, for example, BRCA1/2 genetic mutations are an important component of preventive care for breast and ovarian cancers.
Now, blood-based and imaging biomarkers are being developed to detect early signs of neurodegenerative diseases like Alzheimer’s and Parkinson’s before clinical symptoms appear. Recent studies have also validated plasma biomarkers (e.g., phosphorylated tau) that correlate with early Alzheimer’s pathology, enabling earlier diagnosis and trial enrollment. Early detection could allow for timely intervention, improve clinical trial design, and shift the focus from symptom management to disease prevention.
Transforming drug development with AI-powered trial simulations
AI in healthcare is pushing all sorts of new boundaries, and the technology is constantly improving. Not only are AI models and platforms capable of designing novel drug candidates and predicting protein structures, but they’re now accelerating the clinical trial process.
Quantitative systems pharmacology (QSP) models and “virtual patient” platforms simulate thousands of individual disease trajectories, allowing teams to test dosing regimens and refine inclusion criteria before a single patient is dosed. AI-powered digital twins are also transforming clinical development and translational research. For example, Unlearn.ai has validated digital twin-based control arms in Alzheimer’s trials, demonstrating that AI-augmented virtual cohorts can reduce placebo group sizes considerably, thereby ensuring faster timelines and more confident data without losing statistical power.
Launching rapid-response gene editing with personalized CRISPR therapy
In 2025, a seven-month-old infant with CPS1 deficiency received personalized CRISPR base-editing therapy developed in just six months. This treatment was delivered via lipid nanoparticles to correct a life-threatening mutation and marked the first use of CRISPR tailored to a single patient.
By demonstrating the feasibility of rapid, individualized gene editing, even for life-threatening conditions, this breakthrough could lead to new options for rare diseases that have no existing treatments. Beyond this type of personalized medicine, in vivo CRISPR therapies may be the next evolution in treating cardiovascular and metabolic diseases. For example, CRISPR Therapeutics’ CTX310 reduced LDL by 86% in Phase 1 trials, and Intellia’s NTLA-2002 for hereditary angioedema has entered Phase 3 with strong early efficacy.
Accelerating antiviral discovery with AI to fight future pandemics
Antiviral drugs work by disrupting a virus’s ability to infect and replicate, targeting stages like cell entry, replication, or release. Traditionally tailored to specific viruses, these treatments are now evolving into broad-spectrum antivirals and host-directed therapies — approaches that target shared viral mechanisms or the human cellular pathways that viruses exploit. To accelerate development, researchers are leveraging AI and machine learning to identify and design promising compounds, often before a new virus even appears.
- Broad-spectrum antivirals (BSAs) are designed to target conserved viral elements or host pathways shared across multiple virus families, enabling a single drug to work against diverse pathogens. These could serve as a first line of defense in future outbreaks by buying time until pathogen-specific treatments are developed.
- Host-derived antivirals (HDAs) target human proteins or pathways that viruses require, rather than the virus itself. They may provide more durable antiviral protection from rapidly mutating viruses.
- Machine learning is screening compound libraries, predicting viral protein structures, and identifying host-virus interaction networks before new pathogens emerge. Global initiatives like PANVIPREP in the EU and the U.S. Antiviral Program for Pandemics are investing in AI-driven platforms to preemptively identify antiviral candidates. These innovations can enable a proactive rather than reactive response to a new outbreak or pathogen.
Additionally, lastmonth (August 2025), researchers at MIT reported the invention of antibiotics using generative AI against drug-resistant strains of gonorrhea and Staphylococcus aureus. While still in the early stages, this breakthrough is likely to be a beacon of hope for antibiotic research.
Drug discovery is always in motion, and with AI-driven tools and the rise of personalized medicine, we can expect to see more breakthroughs in 2025 and beyond. At CAS, we’re keeping our finger on the pulse of new innovations in drug discovery, and you can stay up-to-date on the latest research here.
