Prime Medicine dosed the first participant in its global Phase 1/2 study of PM577a, an LNP-delivered prime editor designed to correct ATP7B p.H1069Q in hepatocytes. This is a meaningful execution milestone for in vivo…
The Nobel Assembly awarded the 2026 Prize in Physiology or Medicine to Karl Deisseroth, Peter Hegemann and Georg Nagel for discoveries concerning light-gated ion channels and optogenetics. The October 5 recognition marks the field-shaping…
A peer-reviewed pediatric AML study integrates paired diagnosis–relapse single-cell data with larger outcome cohorts to identify rare chemoresistant cell states that improve risk stratification and nominate drug vulnerabilities. The framework is translationally credible, but…
Osaka University researchers report a modular mRNA–lipid-nanoparticle system that generates transient CAR activity in vivo and uses mRNA methylation state to tune immune programs. Target substitution across FAP, EpCAM, LGR5 and CD133 supports platform…
An online-first, peer-reviewed Nature Genetics study maps two cis-regulatory brakes on embryonic ζ-globin (HBZ) and uses base editing to reactivate it in mouse and patient-derived erythroid models of severe α-thalassemia. This is a compelling…
A peer-reviewed Leukemia letter reports increased ROR1 expression in mantle-cell lymphoma specimens after CD19 CAR-T relapse and shows that experimental ROR1/CD3 T-cell engagers kill resistant patient-derived cells and control mouse tumors. The work identifies…
Monte Rosa reported randomized Phase 1 GFORCE-1 data in 108 obese adults with elevated cardiovascular risk. Four weeks of oral NEK7-directed molecular glue MRT-8102 produced 80–90% median NEK7 degradation and broad reductions in inflammatory…
Link Cell Therapies initiated clinical dosing of LNK001, an autologous logic-gated CAR-T therapy that requires coexpression of CAIX and ENPP3, in a Phase 1 clear-cell renal-cell-carcinoma study at MD Anderson. The design directly addresses…
C4 Therapeutics reported biomarker evidence that cemsidomide plus dexamethasone activated CD8 T cells and reprogrammed natural-killer cells across 62 heavily pretreated myeloma patients, with stronger effects at 75 and 100 micrograms. In the elranatamab…
A peer-reviewed Cancer Discovery study evaluated 11 large B-cell lymphoma patients who received one lot of cemacabtagene ansegedleucel and identified two separable determinants of allogeneic CAR-T performance. High pre-existing recipient alloreactive CD8 T-cell frequencies…
