Tag

Rna Therapeutics

All articles tagged with #rna therapeutics

health1 month ago•44 min saved

RNA-tRNA therapy aims to bypass stop signals to treat multiple genetic diseases

Scientists at the University of Toronto engineered suppressor tRNAs delivered in lipid nanoparticles to bypass premature stop signals, restoring full-length proteins in cystic fibrosis models. A specific chemical modification (m1A) boosted tRNA activity and persistence, while a lung-targeted delivery system (TTP-3) improved tissue reach. Because stop signals occur across many genes, this RNA-based approach could become a disease-agnostic platform, potentially treating numerous genetic disorders, though clinical safety and organ-specific delivery remain key hurdles before use in people.

via thebrighterside.news|

#cystic-fibrosis #genetic-diseases #health

biotech2 months ago•2 min saved

Silence Therapeutics' divesiran shows strong Phase 2 signals in polycythemia vera

In a 48-patient Phase 2 study for the rare blood cancer polycythemia vera, Silence Therapeutics' RNA drug divesiran achieved a clinical response in 88% of treated patients versus 19% on placebo, suggesting robust efficacy and potential competitiveness with Takeda's near-approval therapy.

via STAT|

#biotech #divesiran #drug-development

business11 months ago•11 min saved

Novartis to Acquire Avidity Biosciences for $12 Billion to Strengthen Neuroscience Pipeline

Novartis plans to acquire Avidity Biosciences for $12 billion to strengthen its neuroscience pipeline with late-stage RNA therapeutics targeting neuromuscular diseases, including a new platform for muscle-directed delivery of RNA therapies, aiming for product launches before 2030 and increasing its sales growth forecast to 6% CAGR for 2024-2029.

via Novartis|

#acquisition #avidity-biosciences #business

business11 months ago•1 min saved

Novartis to Acquire Avidity Biosciences for $12 Billion in Cash

Novartis is acquiring U.S. biotech firm Avidity Biosciences for about $12 billion in cash, aiming to expand its RNA therapeutics portfolio and boost growth, with the deal expected to close in the first half of 2026 after Avidity spins out some of its business units.

via CNBC|

#acquisition #avidity-biosciences #biotech

science-and-technology2 years ago•24 min saved

"Unlocking the Healing Power: Mastering the Art of Therapeutic mRNA Design"

Researchers are exploring the art of designing therapeutic mRNA molecules for vaccines and immunotherapies. They are investigating various factors that influence mRNA stability and translation efficiency, such as codon usage, RNA structure, and nucleotide modifications. By optimizing these parameters, scientists aim to enhance the effectiveness and safety of mRNA-based therapies. Deep learning models are also being developed to predict RNA secondary structure, aiding in the design process. Understanding the intricacies of mRNA design could lead to improved treatments for various diseases.

via Nature.com|

#mrna-design #mrna-stability #rna-structure

biotech3 years ago•6 min saved

Amit Munshi's Next-Gen RNA Therapeutics Startup: Thinking Big with $300M+

Amit Munshi, former CEO of Arena Pharmaceuticals, is now leading ReNAgade Therapeutics, a startup focused on next-gen RNA therapeutics. Meanwhile, Indivior has acquired a new nasal spray for opioid overdose, Icosavax is pushing forward with a bivalent RSV-hMPV vaccine, and Ironwood Pharmaceuticals is set to acquire VectivBio in a $1 billion all-cash deal. Quanta Therapeutics has also raised $50.7 million to develop KRAS inhibitors for clinical trials in 2024.

via Endpoints News|

#biotech #kras-inhibitors #manda