In the realm of medicine, the year 2025 has been a challenging one, marked by political decisions that have significantly impacted the U.S. research enterprise. Despite these obstacles, science has continued to forge ahead, leading to several groundbreaking advancements in the field of medicine.
As we navigate through what many refer to as biology’s century, we are witnessing an era of unparalleled progress in medical science. This progress is being driven by a wealth of data, a deep understanding of biology, and a surge of investment from the private sector. What sets 2025 apart is that these advancements have been achieved amidst government decisions that may hinder future scientific breakthroughs. Let’s take a closer look at some of the key developments of 2025.
A gene edit and a baby
One of the most notable medical innovations of the year came in the form of a gene-editing procedure performed on a 6-month-old baby named KJ Muldoon. Using CRISPR technology, researchers at the University of Pennsylvania were able to edit the genes of cells in KJ’s liver to correct a rare mutation. While the treatment did not cure KJ, it did allow him to resume a normal diet and avoid the need for a liver transplant. The case of KJ Muldoon raises important questions about the future of personalized medicine and how the healthcare system can adapt to make such treatments more accessible.
A strong crop of new medicines
In 2025, the Food and Drug Administration approved 44 new drugs, showcasing a robust pipeline of innovation in the pharmaceutical industry. Among these new medicines, a few standout for their significant impact. Yeztugo, a long-acting medicine developed by Gilead for the prevention of HIV infection, represents a major breakthrough in the fight against the disease. Journavx, a non-opioid painkiller from Vertex Pharmaceuticals, offers a promising alternative for pain management without the risk of addiction. Additionally, the approval of two new antibiotics to treat gonorrhea addresses a critical need in combating antibiotic resistance.
Progress against cancer
Advancements in cancer treatment have been steadily progressing, with the FDA approving five new drugs targeting different subtypes of lung cancer in 2025. These targeted therapies have extended the lives of patients with metastatic disease, offering new hope for those battling the illness. Among the new approvals, Enhertu, an antibody-drug conjugate, has emerged as a new standard of care for patients with HER2-positive tumors, significantly delaying tumor progression.
Vaccines delivering more for less
Vaccines have proven to be powerful tools in preventing disease, with emerging evidence suggesting that vaccines like the HPV vaccine may have additional benefits beyond their intended purpose. Studies have shown that vaccines for shingles and HPV have the potential to prevent conditions like Alzheimer’s and certain types of cancer, highlighting the far-reaching impact of vaccination programs.
The GLP-1 pills
GLP-1 agonists, such as Wegovy and Zepbound, have revolutionized weight loss treatment and cardiovascular risk reduction. The approval of a higher dose of semaglutide as a pill for weight loss represents a significant advancement in the field of obesity management. With the availability of these medications as pills, they are poised to reshape healthcare and societal norms.
What to watch
Looking ahead to the future, there are several promising developments on the horizon. A new gene therapy shows promise in treating Huntington’s disease, while research on genetically engineered pigs for organ transplantation could alleviate the organ shortage crisis. Notably, an Italian nonprofit successfully brought a gene therapy to market, highlighting the potential for non-profit organizations to drive medical innovation.
As we reflect on the achievements of 2025, we are hopeful for more breakthroughs in the coming year and beyond. May the field of medical science continue to progress, overcoming challenges and paving the way for a healthier future.
