Top 5 Clinical Trials Shaping Medicine in 2025 

Thousands of clinical trials aim to push the boundaries of modern medicine each year—improving our understanding of disease mechanisms, biomarkers, and diagnostic tools, and refining approaches to new therapies. In 2025, many of the most promising trials center innovations in precision diagnostics, genetic testing, and computational modeling, especially in high-need areas like rare diseases and oncology.

While weight-loss drugs like GLP-1 receptor agonists continue to dominate headlines, other trials are having a quieter but potentially more lasting affect on the diagnostic and therapeutic landscape. 

Drawing from recent expert perspectives published in Nature Medicine, we’ve selected five ongoing clinical trials that are poised to shape the scientific and clinical landscape in 2025. 

Here's what clinical laboratory professionals should be watching—and why it matters.

Drawing from published in Nature Medicine, we’ve selected five clinical trials that stand out for their implications in lab testing, personalized medicine, and the future of integrated care. 

Here’s what clinical lab professionals should keep on their radar this year:

 1. Gene therapy for prion disease 

Prion diseases contributed to the death of 1 in 6,000 people in the US in 2022 alone. Now, a Phase 1/2a trial by Ionis Pharmaceuticals, PrProfile, could change that. 

Prion diseases are caused by misfolded proteins in the brain, leading to progressive neurological damage that can affect movement, cognition, and personality, and eventually cause death. These conditions can be genetic—such as Creutzfeldt-Jakob disease (CJD), the most common form—or acquired from infected meat products. 

The PrProfile trial is evaluating ION-717, an investigational antisense oligonucleotide that inhibits the production of prion proteins. The drug is administered intrathecally, and the study is assessing its safety, tolerability, and pharmacokinetics across 16 global clinical trial sites. 

The trial completed enrolment within a year of its launch in 2024 and aims to collect its first set of data by the end of this year. 

If successful, ION-717 could not only offer hope for prion disease patients but also set a precedent for early treatment for a range of neurodegenerative conditions beyond prion disease.

2. Base editing for sickle cell disease

Sickle cell disease (SCD) affects about 100,000 people in the US and can shorten life expectancy by up to 20 years. Beam Therapeutics saw promising early results in its phase 1/2 trial, BEACON, which is the first base-editing clinical trial targeting hematopoietic stem cells (HSCs), offering some hope for an effective treatment. 

The investigational therapy, BEAM-101, uses adenine base editors to introduce single-base changes in the promoter regions of the γ-globin genes HBG1 and HBG2. This disrupts the BCL11A repressor, reactivating fetal hemoglobin (HbF) expression, a strategy that aims to increase the proportion of HbF in red blood cells vs sickle hemoglobin to reduce SCD symptoms.

Early results published in Nature Biotechnology show that in four patients treated with BEAM-101, functional HbF levels increased by more than 60 percent within one to six months. This increase was accompanied by reduced red cell sickling and adhesion, as well as improved flow properties. 

Though no serious adverse effects were reported in these patients, there was one death due to lung injury. Rather than a consequence of base editing, this lung injury was likely due to the toxicity of busulfan, the chemotherapy drug used to deplete the patient’s existing stem cells prior to stem cell transplantation, which is part of the current standard of care for sickle cell disease.

BEAM-101 is one of at least 10 base-editing therapies undergoing clinical trials, including two additional approaches being tested by Beam Therapeutics, including 

• a CD117-targeting antibody approach, used with base editing of HBG1 and HBG2 to improve conditioning, and 
• BEAM-201, an allogeneic CAR-T cell therapy for blood cancers that base edits four genes. 

If successful, these trials could pave the way not only for treating SCD but also other genetic disorders caused by single-base mutations.

3. Radiopharmaceuticals for prostate cancer

Prostate cancer remains one of the most prevalent cancers in men, with an estimated 191,000 new cases diagnosed and 33,000 deaths reported in the US in 2020. For a subset of patients, the disease progresses to metastatic castration-resistant prostate cancer (mCRPC), a late-stage cancer with limited treatment options. 

The Phase 3 PSMAfore trial, led by Novartis, is investigating whether lutetium-177-labeled PSMA-617 (Pluvicto) can be used as a therapy for cells that express prostate-specific membrane antigen (PSMA) earlier in the treatment paradigm, i.e., for patients with PSMA-positive metastatic hormone-sensitive prostate cancer who are treatment-naive or minimally treated. 

This radioligand therapy targets PSMA, delivering a radioactive payload directly to cancer cells. By the end of 2025, the trial aims to determine whether Lu177-PSMA-617 could be used in combination with the standard of care to improve radiographic progression-free survival (rPFS).

One of the trial’s designers, Oliver Sartor, MD, commented, “This could be a potential game-changer for hundreds of thousands of patients with prostate cancer globally.” However, Sartor also remarked that even if Lu177-PSMA-617 proves effective, its high cost may limit its availability globally.

If successful, the trial could shift the timing and use of radioligand therapy in prostate cancer care, with important implications for diagnostics and lab-based PSMA screening workflows.

4. AI ChatBot to aid cervical cancer screening

Each year, about 12,000 new cases of human papillomavirus (HPV)-associated cervical cancer are reported in the US. Thanks to decades of cervical cancer screening programs, mortality rates have decreased by more than 70 percent, but recent trends show fewer women participating in screening. 

To address barriers to screening, the AppDate-You study, led by the International Agency for Research on Cancer (IARC), is evaluating a new strategy to improve screening uptake. The approach combines a smartphone-based, AI-powered decision aid with at-home HPV self-sampling kits. 

The decision aid—a multilingual, AI-based ChatBot—was developed using qualitative research into women’s knowledge gaps, attitudes toward cervical cancer screening, and smartphone usage data, as well as recommendations from healthcare professionals. 

The ongoing trial provides study participants with the decision aid along with a mailed HPV self-sampling kit to enable them to make informed decisions about screening and complete the process from home. The aim is to assess whether this combined strategy increases participation in the HPV-detection-based cervical cancer screening pathway. 

If successful, this model could offer a scalable solution to improve public health outcomes not only in cervical cancer but also for other conditions where early detection through screening is key to prevention or improved prognosis.

5. Personalized breast cancer screening

Breast cancer accounts for about 30 percent of all new cancers in women and is the second leading cause of cancer death in the US. Despite widespread screening programs, breast screening, which typically starts after age 50, has led to limited reductions in mortality and high rates of overdiagnosis. 

To improve the effectiveness of breast cancer screening, Unicancer is leading an international randomized, open label, multicentric, study called MyPeBs. The study is assessing whether a risk-based breast cancer screening strategy can more accurately detect stage 2 or higher breast cancers, compared to current standard country-specific screening guidelines.

The personalized approach incorporates DNA testing from saliva samples to calculate a polygenic risk score, which is then combined with known risk factors such as family history and breast density. Participants will undergo four years of risk-adapted screening, after which participants will return to routine national screening schedules. Long-term outcomes, including breast cancer incidence and survival, will be monitored over a 15-year follow-up period. 

If the trial demonstrates evidence that personalized risk-based screening significantly improves detection and outcomes, it could mark a major shift in how breast cancers, and potentially other cancers with known risk factors, are prevented and managed around the world. 

Leveraging emerging technologies for better health outcomes

In 2025, a host of clinical trials will assess the feasibility of translating the latest laboratory and computational advances into real-world practices and health care. The five clinical trials highlighted in this article represent a diverse spectrum of innovation, from intrathecally-administered antisense oligonucleotide for prion disease, and base-editing stem cell therapy for sickle cell disease, to a radiopharmaceutical targeting prostate cancer. 

Rounding out these treatment-focused trials are two studies aimed at improving cancer screening: one leveraging an AI-powered decision aid to boost cervical cancer screening participation, and the other investigating a personalized risk-based screening approach to breast cancer detection. 

While these clinical trials differ in their disease focus, endpoints, and methods, they all share the potential of reshaping how we detect, treat, and ultimately prevent disease. Their outcomes could redefine standard practices in clinical laboratories and clinics alike, paving the way for more personalized, effective, and accessible health care.