A gene edit and a baby<\/p>\n
Ask anyone in biotech for the defining moment of medical innovation this year, and they\u2019ll point you to KJ Muldoon. When he was just 6 months old, the genes of cells in KJ\u2019s liver were edited using CRISPR<\/a> \u2014 the technology that won researchers the Nobel Prize in chemistry in 2020 \u2014 to correct a unique mutation.<\/p>\n The treatment did not cure KJ, but it allowed him to resume a normal diet and means he is no longer facing the liver transplant that he likely would have needed otherwise. What was striking about the case was that KJ\u2019s treatment had been developed by researchers at the University of Pennsylvania on a one-off basis. The question now is whether \u2014 and how \u2014\u00a0the medical system can adapt to make more cases like this possible.\u00a0<\/p>\n \t\t\t \t\t\t\t Regulators have moved to lower barriers<\/a> for the development of these \u201cn of 1\u201d treatments, but there is no commercial model to encourage them. The only approved gene-editing treatment, for sickle cell disease and beta thalassemia, is used to treat a larger population and still has a list price of more than $2 million.\u00a0<\/p>\n A strong crop of new medicines<\/p>\n The Food and Drug Administration has approved 44 brand new drugs this year, down six from a year ago but still a bumper crop \u2014 and more than twice as many as were approved in 2010, when the pharmaceutical industry saw itself facing an innovation crisis.<\/p>\n Most new medicines are incremental advances, but every year a few stand out for either their immediate impact or their potential. Here are some that meet those criteria.<\/p>\n Yeztugo: Gilead\u2019s long-acting medicine for the prevention of HIV infection is the closest thing yet to a vaccine<\/a> against the disease. The Trump administration\u2019s dismantling of USAID and cuts to other HIV-prevention programs could blunt its impact, but deals Gilead has made with generics makers could still lead to broad distribution. An agreement with the Global Fund to Fight AIDS, Tuberculosis, and Malaria could provide the drug to 2 million people in resource-limited countries over three years.<\/p>\n Journavx: A non-opioid painkiller developed by Vertex Pharmaceuticals was approved at the start of the year<\/a>. There are questions about how effective it is, but if patients, doctors, hospitals, and insurers embrace it, Journavx could spare some people from being exposed to addictive treatments. It\u2019s one to watch.<\/p>\n New antibiotics: Two new antibiotics were approved to treat gonorrhea. Nuzolvence was produced by the partnership<\/a> of Innoviva Specialty Therapeutics and the Global Antibiotic Research & Development Partnership, and Blujepa by GSK. That\u2019s important because even as bacteria have become more resistant to common antibiotics, antibiotic drug development has been stymied by scientific and commercial challenges<\/a>.<\/p>\n Progress against cancer<\/p>\n Sometimes it is not a single drug that changes everything, but advances that add up one after another. Take lung cancer, for instance, for which the FDA in 2025 approved five new drugs targeting different subtypes of the disease. They are among a broader group of targeted medicines that have in recent years extended the lives of patients with metastatic disease.<\/p>\n \t\t\t \t\t\t\t Among new approvals, for instance, was one for the combination of Rybrevant and Lazcluze, from Johnson & Johnson, which was shown to extend the time before tumors grow significantly in non-small cell lung cancer caused by a mutation in the gene EGFR. So did the targeted therapy Tagrisso, from AstraZeneca, when given with chemotherapy.<\/p>\n Other advances register here, too. Enhertu<\/a>, an antibody-drug conjugate<\/a>, which combines a genetically targeted therapy with a chemotherapeutic warhead in a single molecule, became the new standard of care for patients\u2019 whose tumors test positive for a protein called HER2 when added to an existing drug, Perjeta. Adding Enhertu delayed tumor progression by a median seven months.<\/p>\n Vaccines delivering more for less<\/p>\n Being vaccinated against shingles is already important; it is, quite simply, a painful disease no one wants. But there is building evidence of another reason: The herpes zoster virus that causes shingles may also contribute to the development of Alzheimer\u2019s. A study published using data in Wales, where there was a specific birthdate cut-off for getting a shingles vaccine, helped back up this idea.<\/a><\/p>\n Another example of a vaccine doing more than was intended: HPV vaccinations have nearly eliminated cervical cancer<\/a> in some countries, and can also prevent head and neck cancer. Emerging data say the vaccine may work well as one dose, instead of two<\/a>. It\u2019s not clear whether the U.S. will move in that direction, but the news could be a big deal in areas where vaccines are in short supply.<\/p>\n \t\t\t \t\t\t\t The GLP-1 pills<\/p>\n GLP-1 agonists \u2014 weight loss drugs like Wegovy and Zepbound \u2014 are changing society\u2019s health and our culture. In December, a higher dose of semaglutide, the active ingredient in Wegovy, was approved for weight loss (and the reduction of cardiovascular risks) as a pill instead of an injection<\/a>. The drug was developed by Novo Nordisk. In some ways, it\u2019s a small thing; Novo previously had a GLP-1 pill approved to treat type 2 diabetes. But another new such pill is coming soon from Eli Lilly, and the availability of these medicines will be one more way that they reshape the world.<\/p>\n What to watch<\/p>\n There was also some more futuristic progress afoot.<\/p>\n A new gene therapy showed promise in Huntington\u2019s disease<\/a>, although it seems to have run into hurdles at the FDA<\/a> and full data have not yet been presented.<\/p>\n Researchers have made significant progress in creating genetically engineered pigs whose organs can be transplanted into people, potentially alleviating the organ shortage. Work is moving forward with kidneys<\/a>, and a gene-edited lung<\/a> was transplanted into a brain-dead patient as a first step toward trials.<\/p>\n![\"\"](\"https:\/\/www.newsbeep.com\/us\/wp-content\/uploads\/2026\/01\/Father-Kyle-Playing-with-KJ-post-infusion-768x432.jpeg\")
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\n\t\t\t\tSTAT Plus: What Baby KJ means for the CRISPR gene-editing industry<\/a><\/p>\n![\"\"](\"https:\/\/www.newsbeep.com\/us\/wp-content\/uploads\/2026\/01\/STAT_autoimmune_investment_f2_2000x1125-768x432.jpg\")
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\n\t\t\t\tSTAT Plus: Chasing CAR-T, biotech finds its next gold rush in autoimmune disease<\/a><\/p>\n![\"\"](\"https:\/\/www.newsbeep.com\/us\/wp-content\/uploads\/2026\/01\/NOVO_PILL_01-768x432.jpg\")
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\n\t\t\t\tSTAT Plus: With the Wegovy pill, Novo Nordisk undercuts Eli Lilly in direct-to-consumer market<\/a><\/p>\n