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Decitabine combination improves outcomes for high‑risk AML
A study in Biochemical Genetics reported that adding the DNA methyltransferase inhibitor decitabine to a modified chemotherapy regimen enhanced responses in patients with high‑risk karyotype acute...
CFTR triple therapy benefits children — ETI shows pediatric gains
Real‑world and clinical data reaffirm that the CFTR modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) delivers measurable clinical benefits in pediatric cystic fibrosis patients. The...
Metabolic therapies advance — Glutamine‑driven bladder cancer and autophagy activator for NSCLC
Two translational studies identified metabolic vulnerabilities that could be exploited therapeutically. Ding, Zhang, and Huang reported in the Journal of Translational Medicine that glutamine...
Merck to buy Cidara for $9.2B: bolsters long‑acting influenza program
Merck agreed to acquire Cidara Therapeutics for about $9.2 billion to add CD388, a long‑acting neuraminidase inhibitor–Fc conjugate designed to prevent influenza, to its antiviral portfolio....
Iambic raises $100M: advances AI‑discovered cancer drug into trials
Iambic closed a $100 million financing round to advance clinical trials of cancer candidates discovered using its AI platform; the company recently presented breast‑cancer data at the European...
Point‑of‑care AAV8 antibody test... eases gene‑therapy screening bottleneck
Researchers reported a point‑of‑care test for AAV8 binding antibodies using Chembio’s Dual Path Platform (DPP), enabling rapid detection of anti‑AAV8 antibodies in plasma, serum and whole blood....
Intranasal nanogel vaccine slows HPV‑driven cervical tumors – preclinical
Chiba University researchers published preclinical data in Science Translational Medicine showing a cationic cholesteryl‑group‑bearing nanogel (cCHP) nasal vaccine delivering HPV16 E7 antigen plus...
FDA restricts Elevidys label – adds boxed warning for DMD gene therapy
Regulatory reporting from BioCentury indicates the FDA has moved to restrict the label and add a boxed warning to Sarepta’s microdystrophin gene therapy Elevidys. The label change responds to...
Biomimetic mRNA delivery restores PTEN in colorectal tumors – targeted immunotherapy advance
Researchers reported a biomimetic mRNA delivery system designed to restore PTEN expression in colorectal tumors and potentiate targeted immunotherapy. The platform delivers therapeutic mRNA with...
Engineered cell strategies amplify target clearance: CAR‑T IDRs and microglial CARs
Two separate preclinical studies reported engineered cellular strategies to improve target engagement. A Nature Chemical Biology paper showed intracellular delivery of multivalent Intrinsically...
Targeted genetic assay improves diagnosis for X‑linked dystonia‑parkinsonism
Teams at Brigham and Women’s Hospital and Harvard Medical School developed a targeted genetic test to improve diagnostic precision for X‑linked dystonia‑parkinsonism (XDP), a neurogenetic disorder...
ctDNA‑NGS shows clinical utility in stage III/IV NSCLC – large Chinese cohort
A BMC Cancer study validated a circulating tumor DNA (ctDNA) next‑generation sequencing platform across a large cohort of stage III and IV non‑small cell lung cancer patients in China,...
AAV gene therapy delivering BDNF/GAS6 delays ALS onset in mice
Researchers used AAV vectors to deliver brain‑derived neurotrophic factor (BDNF) and GAS6 to muscle tissue in SOD1G93A mouse models of ALS and observed delayed symptom onset and slower disease...
Merck to buy Cidara for $9.2B — secures late‑stage flu antiviral
Merck agreed to acquire Cidara Therapeutics for about $9.2 billion to add CD388, a long‑acting antiviral designed to prevent seasonal and pandemic influenza, to its pipeline. Cidara reported...
FDA narrows Elevidys label: boxed warning, ambulatory‑only use
The U.S. FDA revised the label for Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy Elevidys, adding a boxed safety warning and restricting use to ambulatory patients. The agency...
AAV bottleneck: point‑of‑care antibody screening and high‑tropism capsids emerge
Researchers introduced two advances targeting key barriers to AAV gene therapy: a point‑of‑care (POC) test for AAV8 binding antibodies using Chembio’s Dual Path Platform, and discovery of AAV...
Nasal nanogel vaccine slows HPV tumors: therapeutic approach advances
Researchers at Chiba University reported a cationic nanogel‑based intranasal therapeutic vaccine that targets HPV16 E7 oncoprotein and slowed tumor growth in preclinical models. The...
Gene therapy in neurology: hearing restored and ALS onset delayed in mice
Two preclinical gene therapy advances target neurodegeneration and sensory loss. One team delivered VGLUT3 to inner hair cells and restored hearing in aging mice, highlighting a pathway to address...
MD Anderson launches $2.5B campaign — 'Only Possible Here' aims to transform cancer research
The University of Texas MD Anderson Cancer Center announced a $2.5 billion philanthropic campaign titled 'Only Possible Here' to expand research, clinical innovation and translational capacity....
DeepTarget: AI maps small‑molecule cancer mechanisms — computational discovery scales
Sanford Burnham Prebys and collaborators unveiled DeepTarget, an AI‑driven tool that predicts anti‑cancer mechanisms of small molecules and proposes candidate targets beyond the...