Genética, CRISPR, biologia sintética e biomanufatura.
Atualizado
In the BE BOLD Phase 3b trial of 553 adults with psoriatic arthritis, numerically greater ACR50 rates for BIMZELX held from Week 16 through Week 24, UCB reported.
In a 50-adult Phase 2a challenge study, one dose of ETH47, an mRNA encoding interferon lambda, gave numerical reductions in rhinovirus load and symptoms; no p-values were reported.
In a small Phase 2 trial, four monthly doses of PepGen's experimental drug for myotonic dystrophy type 1 repaired splicing errors in six patients; the company's shares rose over 90%.
In a phase I trial in 10 healthy adults, injected volar fibroblasts increased skin thickness and elasticity at treatment sites. The approach is studied for amputees' residual limb skin.
FITdb integrates 43 screens covering 20,696 mouse and 22,293 human genes across 199 immune cell types and states. Researchers can query how gene perturbation affects immune cell function.
In the phase 1 CARLYSLE trial of 15 patients, Autolus reported SLEDAI-2K improvement and DORIS remission at 6+ months. The sample is small; phase 2 data are expected in 2028.
In humanized lupus mice, podocytes supply palmitic acid to T cells and push them into a kidney-resident CD103+ state. Blocking podocyte fatty acid oxidation reduced renal inflammation.
Researchers found that a chimpanzee-specific retroviral insertion keeps the LINC00662 gene silent, while it stays active in human brain organoids and supports neuronal development.
Beam reported that risto-cel, a base-editing therapy for sickle cell disease, met its primary endpoint in a pivotal trial of 31 patients. The company plans an FDA application by year-end.
Early data on a bispecific antibody combination in metastatic castration-resistant prostate cancer come from a small group; the combination now moves into Phase 3.
In a phase 1b trial in allogeneic stem cell transplant recipients, oral SER-155 had a safety profile like placebo and was linked to fewer bloodstream infections.
A review of earlier lab work describes proteins, DNA and other biomaterials as light guides, emitters and converters; medical sensors look most promising, but integration is hard.
Universities in Volgograd and Bashkortostan are finishing a new antiplatelet substance for myocardial infarction; the developers report efficacy comparable to eptifibatide.
Tablets based on monophosphorylated starch kept the probiotic detectable in the feces of calves with diarrhea longer than a liquid suspension did.
London-based Oyster Therapeutics is developing tau protein degraders for Alzheimer's disease. The lead candidate has not entered the clinic, and the company has not said when it will.
The method uses antibodies to bring a deaminase enzyme to a target protein and reads binding sites by sequencing. According to the authors, it works with standard single-cell multiomic platforms.
Indian researchers mapped protein secretion in the TB bacterium and showed that SecA1 and Tat are essential for its growth. The work may help identify drug targets; it is at the laboratory stage.
Cryo-EM structure shows how the phage protein SglPP7 locks MurJ, a bacterial lipid transporter. The work is basic research relevant to the search for new antibacterials.
In a Cancer Cell study, a tumor-targeted TGF-β blocker loosened the fibrous barrier around tumors in mice and let T cells in. A human version has entered safety testing.
Latus Bio presented preclinical data in non-human primates: AAV capsids reached 40–60% of cortical neurons and about 80% of glomeruli, outperforming AAV9 on several measures.
