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AAV variants boost vascular delivery: Two studies report improved transduction
Teams publishing in Gene Therapy reported engineered AAV variants that substantially improve transduction of human vascular endothelial cells. Stamataki et al. screened and identified capsid...
Microglial CARs... Enhance amyloid clearance in preclinical models
Researchers engineered microglia with chimeric antigen receptors (CARs) to selectively phagocytose Aβ1‑42, reporting increased uptake of pathogenic amyloid species in vitro and in vivo models. The...
Condensing CAR‑T potency: IDR strategy boosts activity against low‑antigen tumors
A Nature Chemical Biology study showed that intracellular delivery of multivalent binding agents using intrinsically disordered regions (IDRs) promotes condensate formation that enhances CAR‑T...
AAV microdystrophin therapy update: Elevidys signals gene‑therapy momentum
Regulatory and clinical history around AAV microdystrophin therapies continues to shape the Duchenne muscular dystrophy (DMD) landscape after the FDA granted accelerated approval to...
Prime‑editing inversion method: PIE enables chromosome‑scale DNA flips
Researchers introduced Prime‑Editing‑Based Inversion with Enhanced Performance (PIE), a technique that enables efficient, large‑scale inversion of genomic DNA in mammalian cells. PIE improves on...
Merck to acquire Cidara for $9.2B — Adds long‑acting flu antiviral
Merck agreed to buy Cidara Therapeutics for approximately $9.2 billion to add the long‑acting influenza prophylactic CD388 to its antiviral portfolio. Cidara’s CD388—a small‑molecule neuraminidase...
Sofinnova closes €650M fund — Capital XI backs preclinical drugmakers and medtech
Sofinnova Partners raised €650 million (about $750 million) for its Capital XI fund to invest in preclinical life‑science startups, allocating roughly 20%–30% toward medical devices. The firm has...
Korro stock collapses 81% after underwhelming RNA‑editing data
Korro Bio’s shares plunged 81% after topline data from its Phase I/IIa REWRITE trial showed KRRO‑110 produced functional M‑AAT protein but failed to reach the protective 11 µM AAT threshold. The...
Iambic raises $100M to move AI‑designed oncology candidates into trials
AI drug‑discovery startup Iambic closed a $100 million financing to fund clinical development of oncology candidates discovered by its generative chemistry platform. The round follows...
AAV vector toolkit expands — Point‑of‑care antibody test and new capsids for vascular transduction
Researchers introduced two advances that address key bottlenecks in AAV gene therapy: a rapid point‑of‑care test for AAV8 binding antibodies and discovery of AAV capsid variants that improve...
Gene‑editing toolkit widens — Collagen disorder therapies and chromosomal‑scale inversions
Two independent papers advance genome‑editing approaches: a review outlining progress and prospects for gene editing in collagen disorders and a methods paper describing Prime‑Editing‑Based...
CAR platform advances: intracellular condensation and microglial CARs
New preclinical studies report two distinct routes to improve chimeric antigen receptor (CAR) strategies. A Nature Chemical Biology paper shows that intracellular delivery of multivalent...
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...