Archive
1250 articles, sorted by publication time.
The AEGIS program will combine base editing, prime editing, and bone marrow-targeted delivery in an effort to use shared manufacturing, toxicology, and clinical frameworks to rapidly create one-time treatments for different ultra-rare mutations; treating 10 children within five years is a goal, not a proven outcome.
In osteosarcoma that has recurred or progressed after at least two lines of treatment, risvutatug rezetecan has defeated chemotherapy for the first time in a randomized Phase 3 trial; a regulatory filing in China is expected to move forward, but the magnitude of benefit, maturity of survival data, and full safety data have yet to be disclosed.
A nanofiber drug seed slowly releases two immune-stimulating agents inside tumors and, when combined with radiation therapy, shows potential for localized drug control. However, the findings remain limited to mice with triple-negative breast cancer, with manufacturing and safety hurdles still to be cleared before human trials.
ISM6331 targets TEAD transcription factors downstream of the Hippo signaling pathway, and its first-in-human trial has been selected for an ESMO rapid oral presentation. However, its safety, dosing, and antitumor efficacy remain undisclosed, and the real clinical test will come when the data are presented in October.
After 22 children ages 2 to 11 received preventive treatment for one year, most had no further hereditary angioedema attacks; CSL is preparing to apply for an expanded indication but has not yet disclosed attack rates or details of adverse events.
Akeso has advanced its HER3 antibody-drug conjugate AK138D1 into a trial evaluating it as monotherapy and in combination with ivonescimab, enrolling patients with advanced non-small cell lung cancer in both the first-line setting and after treatment failure. This 265-patient study has only just begun and is currently designed to answer questions about safety and preliminary response, rather than superiority in efficacy.
A retrospective study at four hospitals in China shows that, after site-specific data adaptation, a large language model can accurately identify patients at high risk of acute kidney injury and provide individualized risk explanations; whether it can actually improve treatment outcomes still requires prospective trials.
A University of California San Diego team continuously measured levodopa in fingertip sweat, obtaining results comparable to laboratory blood tests. The technology could help close the information gap in dose adjustment, but larger-scale validation is still needed before it can support clinical decisions or automated drug delivery.
The HIF-2α inhibitor casdatifan showed durable tumor responses in patients with advanced clear-cell renal cell carcinoma, and changes in blood erythropoietin may also offer clues to drug activity; however, small, single-arm phase 1 data are not yet sufficient to establish an efficacy advantage.
After reanalyzing 414 diagnostic slides from a phase 3 trial, the research team found that patients with higher tumour cell density may benefit from preoperative chemoradiotherapy with added irinotecan; however, this post hoc subgroup finding is not yet sufficient to change clinical management.
The research team used a programmable Cas13 system to correct messenger RNA processing in tumor cells, restoring MHC-I antigen presentation and enhancing immune checkpoint therapy in mice; the strategy has not yet entered human trials, and delivery and long-term safety remain to be clarified.
A U.S. randomized trial found that participants who continued the anti-IgE antibody were more likely to pass high-dose challenges with three foods; however, the difference was driven mainly by more adverse reactions and withdrawals with oral immunotherapy, rather than a large disparity in efficacy among those who completed treatment.