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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...
Bovine cell immortalization without GM: cultivated beef barrier falls
Researchers at Hebrew University and Believer Meats reported a method to naturally immortalize bovine cells without genetic modification by activating telomerase and PGC1α, enabling stable...
Third Arc licenses Adagene Safebody — $840M deal to build masked CD3 engagers
Third Arc Bio licensed Adagene’s Safebody platform in a deal worth up to $840 million to generate two masked CD3 T‑cell engagers against tumor‑associated antigens. The agreement grants Third Arc...
CHMP recommends multiple new medicines — EMA advances gene therapy and oncology candidates
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued positive opinions on several new products, including a gene therapy for Wiskott‑Aldrich syndrome...
Merck buys Cidara for $9.2B — bolsters influenza prevention push
Merck agreed to acquire Cidara Therapeutics for approximately $9.2 billion to add a late-stage, long-acting antiviral designed to prevent seasonal and pandemic influenza. The deal centers on...
FDA tightens Elevidys label: boxed warning added — use narrowed
The U.S. Food and Drug Administration revised the label for Sarepta Therapeutics’ microdystrophin gene therapy Elevidys, adding a boxed safety warning and restricting use to ambulatory patients....
Gene therapy toolkit expands: new AAV capsids and bedside antibody test
Researchers reported discovery of novel AAV variants that substantially improve transduction of human vascular endothelial cells, and separately a point-of-care test was unveiled to detect...
Lundbeck jumps into Avadel fight — Alkermes faces tougher bid
Lundbeck submitted an unsolicited, higher cash offer for Avadel Pharmaceuticals, intensifying a takeover contest that previously favored Alkermes. The new bid increases the cash component per...
Triple CFTR combination benefits children with cystic fibrosis — pediatric data
New clinical data confirm that the CFTR modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) delivers measurable clinical benefit in children with cystic fibrosis, extending the therapy’s...