Eli Lilly and Company has agreed to acquire Orna Therapeutics for up to $2.4 billion in cash. The deal includes ORN-252, a clinical-trial-ready CD19-targeting in vivo CAR-T therapy for B cell-driven autoimmune diseases.
A lipid nanoparticle gene-editing approach inserted a full healthy CFTR gene into human airway cells and restored 88% to 100% of normal CFTR function in lab tests. The nonviral strategy is intended as a mutation-agnostic path for cystic fibrosis.
University of Missouri researchers released PSBench, a database of 1.4 million annotated protein structure models verified by independent experts. The resource aims to improve AI assessment of protein models as biologic drug discovery moves toward data-driven design.
Researchers have developed lipid nanoparticles that deliver mRNA therapeutics to simultaneously treat lung cancer and associated muscle wasting. The nanoparticles target lung tumors while preserving muscle mass, showing 2.5 times greater tumor reduction in preclinical models. This dual-action approach addresses both the primary tumor and debilitating cachexia syndrome.
Researchers have engineered immune cells to sense cancer metabolites, improving tumor infiltration in solid cancers. Separate studies identify Kappa and Lambda Myeloma Antigens as specific targets for multiple myeloma treatment, while mRNA-based personalized cancer vaccines show promise through AI-driven neoantigen selection.
Scientists have developed multiple breakthrough delivery systems for gene editing and mRNA therapies, including a simple amino acid supplement that increases delivery 20-fold and a CRISPR gene drive that reverses antibiotic resistance in bacterial populations.
Researchers discovered that adding three common amino acids to lipid nanoparticles dramatically improves mRNA and CRISPR gene editing delivery, boosting efficiency up to 20-fold and raising gene editing success rates from 25% to nearly 90%.
Scientists have developed two breakthrough approaches to dramatically enhance gene editing and mRNA therapy delivery: a simple amino acid supplement that increases CRISPR efficiency to nearly 90 percent, and a self-replicating CRISPR system that spreads between cells like a virus.
Researchers have created a nasal vaccine platform that protects mice against flu, COVID-19, SARS, and bacterial infections by activating both innate and adaptive immune systems, offering a fundamentally different approach to disease prevention.
UCLA scientists developed a lipid nanoparticle-based gene editing system that successfully inserts a full-length healthy CFTR gene into human airway cells, offering potential treatment for cystic fibrosis patients unresponsive to current therapies.
