In vivo BCMA CAR-T phase 1: 15 months of follow-up, one ongoing sCR
Green CAR-T cells attacking a red myeloma cell at right
Science
Briefs on new papers and preprints across organoids, disease models, AI drug design, tumor immunology and cell therapy, autoimmunity, vaccines, antibody engineering, nucleic-acid medicines and precision oncology. Each brief is read from the original paper and gives the study design, key numbers, limitations and what it could mean for drug development, with a DOI link. Preprints are labelled and have not been peer reviewed.
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A pIgR bispecific raises cynomolgus BAL exposure 5.5-fold
A bispecific crossing airway epithelial cells and released at the airway surface
Synthetic TCR library: on average only 56% of VDJdb TCRs reproduce their annotated reactivity
Green peptide-MHC complexes binding synthetic TCRs, with one non-reproducing case marked in red
Targeting ZMYND8 increases effector-like exhausted T cells in mice and enhances immunotherapy responses
Green immune cells attacking red tumour cells
TSC2 loss produces astrocyte reactivity in organoids at all 3 time points
Astrocytes in a brain organoid cross-section, with reactive cells in red
Human cortical organoids in apallial mice: grafts make up 91.9% of cortical volume
The green area in a brain cross-section is the transplanted human cortical organoid
Human DPP9 loss causes HSPC depletion through CARD8 pyroptosis
A normal haematopoietic stem cell at left and a pyroptotic, ruptured cell at right
A single inhaled dose of ARO-RAGE lowered BALF sRAGE by 90.2%
Alveoli and droplets at left, with the siRNA cleaving messenger RNA at right
Charge-switchable LNPs did not raise IL-6 and other inflammatory cytokines in LPS-pretreated mice
Lipid particles and a macrophage, with sparse red inflammatory cytokines at right
Rentosertib phase 2a: 21/54 aging-clock comparisons significant
A cell cluster with red-marked molecules, showing proteomic aging-related changes
Four humanized DMD lines: deletions of exon 44, 45, 51 or 53
The red exon in the row is the one being skipped, with muscle fibres at right
Anti-CD19 CAR-T in refractory MG: 3 patients in treatment-free remission for at least 19 months
The neuromuscular junction at left, CAR-T cells clearing red B cells at right
Cortical organoids at 5 years: methylation clocks track time with r=0.88–0.90
Organoids growing progressively larger, with red dots on the DNA strand below marking methylation sites
511 AI antibodies benchmarked: only 9.8–13.8% beat the control in Challenge 2
Many antibodies pointing at a green antigen, with the best binder in red
Triple therapy at 72 hours: 8 infant macaques aviraemic for 6 months off treatment
Virus particles and three drug chains, with red marking suppressed virus
A 256-model tumour organoid biobank, with 162 genome-wide CRISPR screens completed
Clustered tumour organoids, with red dots marking dependency genes hit in CRISPR screens
HCMI resource: 97.8% of 421 matched pairs retain at least two classes of DNA features
Tumour and model tissue masses linked by a DNA strand, with red dots marking retained DNA features
A 156-member bispecific screen selects a low-affinity SIRPα×CD38
A macrophage phagocytosing the tumour cell at right via a red bispecific
RAG-17 phase 1 in 6 patients: cerebrospinal fluid SOD1 down 69% and 56%
A red strand inside a motor neuron cell body representing the silenced messenger RNA
A trispecific IL-2R antibody reaches an EC50 of 0.015 nM with Treg bias
A green trispecific antibody binding a dark green regulatory T cell, with the rest quiescent
12 diet models: 4/6 obesogenic diets associated with anti-PD-1 response
Intestinal villi and microbiota at left, T cells and a red tumour at right
44% of rhesus macaques developed serum bnAb activity after the complete sequential HIV vaccine regimen
Three generations of B cells maturing step by step, with antibodies binding the viral spike at right
NISE zero-shot design of drug-binding proteins: APEX affinity of 80 pM
A green designed protein enclosing a red small-molecule drug
Germinal validation: 43–101 designs across 4 antibody targets
Four green targets each bound by a designed antibody, with one epitope in red
251 samples across 11 ASD mouse models: a transient radial glia delay
Red marks the delay among radial glial fibres, with multiple models in the right column
A single dose of PE-LNP achieved 49% precise editing across the mouse liver
A lipid particle carrying red editing components into a hepatocyte
Observational study: mRNA-1010 recipients had larger increases in memory B cells
A cross-section of a lymph node germinal centre, with memory B cells at right
A 160-member bispecific library: surface properties predictable from parents, ρ up to 0.95
Two parental antibodies combining into a bispecific, with red dots marking surface properties
GPR15 guides regulatory CD8 T cells to the colon, with a 42% reduction in sporadic UC
Green regulatory T cells homing to the intestinal epithelium, with red dots marking homing receptors
Two highly sensitized patients transplanted after dual-target CAR-T
Five green CAR-T cells and one red target cell, with a kidney at right
SEZ6-targeting ADC phase 1: a 52% response rate in relapsed/refractory SCLC
An antibody binds a cell surface receptor and carries a red drug into the cell
Transient confinement culture gives small intestinal organoids a 100% engraftment rate with early transplantation
Ring-shaped small intestinal organoids in a dish, with one on the right engrafted into the intestinal wall
A fungal glycan adjuvant extends COVID-19 mRNA vaccine protection to day 500
Lipid particles with mannan chains, a dendritic cell and a B cell producing antibodies
Intratumoural anti-CTLA4 + anti-PD1: 24.3% grade 3–4 toxicity at 6 months
Intratumoural antibody injection, antibody delivered through a vessel, T cells infiltrating the tumour
Agent-designed nanobodies: 46 confirmed, best KD 0.66 nM
An AI network at left, nanobodies in the middle, binding a red target at right
Hashimoto's thyroiditis: 135 distinct TNFRSF14 mutations in a single donor
Thyroid follicles at left, B cell clones at right, with red dots marking mutations
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