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Natera ctDNA MRD outperforms PET: end‑of‑treatment positivity prognostic across lymphomas
Natera presented real‑world data at ASH showing that circulating tumor DNA (ctDNA) minimal residual disease testing at end‑of‑treatment was prognostic across lymphoma subtypes and outperformed PET...
Pig-to-human liver: genetically modified graft sustains organ function in proof-of-concept surgery
Researchers implanted a genetically engineered pig liver into a human patient and demonstrated the graft could support key liver functions for an extended period before complications required its...
Gilead, Arcellx CAR-T: pivotal anito-cel shows deep, durable responses — launch ramps up
Gilead Sciences and partner Arcellx reported pivotal-stage data showing deep and durable responses for anito-cel in multiple myeloma, with a 96% overall response rate and 74% complete remission...
Vertex CRISPR in kids: Casgevy/exa-cel data push pediatric label and access talks
Vertex reported early results showing its CRISPR-based therapy Casgevy produced clinical benefit in pediatric patients with sickle cell disease and transfusion-dependent beta-thalassemia,...
ctDNA and MRD: liquid biopsy and pooled data push MRD closer to regulatory surrogate status
At ASH, Natera presented real-world data showing circulating tumor DNA (ctDNA) — a minimal residual disease (MRD) assay — was prognostic across lymphoma subtypes and outperformed PET scans at end...
Cosmo’s clascoterone: Phase III toplines trigger stock surge after large hair-count gains
Cosmo Pharmaceuticals released topline results from two Phase III SCALP trials for clascoterone 5% topical solution in androgenetic alopecia, reporting a combined 252% relative improvement in...
Higher-dose sickle-cell pill: Fulcrum’s pociredir raises fetal hemoglobin in early study
Fulcrum Therapeutics reported that a higher dose of pociredir raised levels of fetal hemoglobin from 7.1% to 16.9% in 12 participants, presented at ASH. The company argued the data support...
Incyte targets calreticulin: experimental myelofibrosis drug shows spleen and symptom benefits
Incyte presented preliminary results from a study of an investigational agent that targets calreticulin-mutant myelofibrosis, showing meaningful spleen responses and symptom improvements in...
Rusfertide data: Protagonist and Takeda report Phase 3 durability in polycythemia vera
Protagonist Therapeutics and Takeda presented longer-term Phase 3 data showing rusfertide reduced the need for therapeutic phlebotomy in patients with polycythemia vera. Results were disclosed at...
Star Therapeutics: monthly antibody cuts bleeding rates — pivotal dosing underway, IPO considered
Star Therapeutics reported preliminary phase 1/2 data showing its once-monthly injection substantially reduced bleeding rates in patients with von Willebrand disease and has started dosing in a...
Hengrui, BeOne top China innovation index — new ranking maps R&D leaders
IDEA Pharma and SAI MedPartners released the inaugural China Pharmaceutical Innovation and Invention Index, ranking Hengrui and BeOne at the top for invention and innovation. The report evaluated...
Pig liver transplant: Genetically engineered graft supports human but complications follow
Researchers implanted a genetically modified pig liver into a human and demonstrated that the xenograft could perform core hepatic functions for an extended period before surgical removal was...
Gilead, Arcellx CAR‑T posts 96% response rate – pivotal data
Gilead Sciences and partner Arcellx reported pivotal-stage results for anito-cel in multiple myeloma showing deep, durable responses with acceptable safety. In the analysis of 117 patients...
Vertex CRISPR: Casgevy meets early targets in pediatric blood disorders
Vertex reported that its CRISPR-based therapy Casgevy reached early efficacy goals in children with sickle cell disease and beta-thalassemia, supporting efforts to expand the label and accelerate...
Casgevy for kids? Pediatric data point to label expansion – ASH preview
Presentations at ASH preview data suggesting exa-cel (Casgevy) could provide a one-time functional cure for children aged 5–11 with transfusion-dependent beta-thalassemia or sickle cell disease....
Higher-dose pill boosts fetal hemoglobin — Fulcrum posts encouraging sickle cell signal
Fulcrum Therapeutics presented early ASH data showing a higher dose of its oral candidate pociredir increased levels of fetal hemoglobin in patients with sickle cell disease. In the small cohort...
Incyte drug shows spleen responses in myelofibrosis – early data
Incyte disclosed preliminary results for an experimental myelofibrosis agent that produced meaningful spleen-volume reductions and symptom improvements in advanced patients, according to ASH...
MRD inches toward surrogate endpoint status in AML – pooled ASH dataset
Researchers presented the largest prospective pooled dataset to date linking multiparameter flow cytometry minimal residual disease (MRD) status with overall survival in acute myeloid leukemia,...
AstraZeneca dual‑target CAR‑T replicates China results in Western patients
AstraZeneca reported preliminary Western-patient data for its dual-target CAR‑T showing response rates consistent with earlier China-only studies, according to ASH disclosures. Early investigators...
Semaglutide fails to reduce CNS inflammation — trial readout
A BioCentury report detailed that semaglutide, a GLP‑1 analogue laterally explored for neuroinflammation, did not demonstrate the expected anti-inflammatory effects in the central nervous system...