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1250 articles, sorted by publication time.
As BioNTech shifts from the global prominence of its COVID-19 vaccine toward the clinical and commercial delivery of cancer drugs, it is bringing in rare-disease pharmaceutical veteran Guido Oelkers to steer its goal of becoming a multi-product company by 2030.
Once-daily enlicitide reduced LDL cholesterol by 56% and 59% more than placebo in two Phase 3 trials. It may lower barriers to use, but whether it can also prevent heart attacks and strokes remains to be answered by a large trial.
Although median progression-free survival was more than five months longer with selinexor than with placebo in patients with TP53 wild-type disease, the difference did not reach statistical significance; Karyopharm will reduce investment in this indication and shift its focus to myelofibrosis and multiple myeloma.
EchoNext identifies the risk of six types of structural heart disease from routine 12-lead ECGs, helping triage patients for further imaging; validation in more than 100,000 people supports its performance, but a positive notification is not a diagnosis, and it has not yet been shown to improve long-term outcomes.
An Indian team used an artificial intelligence-generated nuclease to perform gene knockout, base editing, and prime editing in rice, with performance broadly matching commonly used Cas9. The results expand the crop-breeding toolbox, but several validation hurdles remain before field-ready varieties can be developed.
Moving from reducing proteinuria to preserving kidney function, the Phase 3 trial’s 24-month results provide the most critical piece of evidence that was missing when Voyxact received accelerated approval; however, the full magnitude of effect and risk data still await public scrutiny.
A dual-AAV gene-editing therapy developed for a single patient was followed by a fatal immune response seven days after injection; the death, family funding, and trial results were not disclosed promptly, and the related animal study is now under investigation by Nature.
When arginine is insufficient, cells may struggle to produce the MHC-I proteins that enable recognition by the immune system; increasing dietary intake improved tumor and infection outcomes in mice, but key gaps remain before it can become a human therapy.
Two drug candidates face FDA decision deadlines this year, while another HER2 lung cancer program remains in early clinical development; the deal gives GSK a near-commercial growth opportunity while also taking on uncertainties surrounding efficacy, safety, and the timeline to market.
A nanofiber implant slowly released two immune-stimulating drugs inside tumors and, when combined with radiotherapy, led to complete tumor regression in 60% of mice with triple-negative breast cancer; however, evidence of efficacy and safety remains limited to the preclinical stage.
Using protein structure and evolutionary information to constrain AI design, a research team created streamlined gene-editing enzymes that do not exist in nature. Some versions surpassed the activity of natural TnpB, but multiple hurdles remain before animal testing and medical applications.
A peer-reviewed review outlines the potential of lanthanide materials in deep-tissue imaging, targeted imaging, and drug delivery; before they can truly enter preclinical development, safety, mass production, and algorithm validation remain major hurdles.