Nucleic Acid, Nucleoside, or Nucleotide
Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy
Gene editing, CRISPR, Rare liver disease, Primary hyperoxaluria type 1 (PH1), Funding
Avidity’s Del-zota Shows Promise in Duchenne Muscular Dystrophy Treatment
Del-zota, Duchenne muscular dystrophy (DMD), RNA therapeutics, exon 44 skipping, dystrophin production, creatine kinase reduction, safety profile
Gilead Faces Legal Challenges Over Alleged HIV Drug Patent Extension Strategy
Product hopping, Patent extension, HIV drugs, Tenofovir, Antitrust, Truvada, TAF vs TDF
Ono Pharmaceutical Acquires Global Rights to Ionis’ Sapablursen for Polycythemia Vera in $940M Deal
Ono Pharmaceutical, Ionis Pharmaceuticals, sapablursen, polycythemia vera, licensing agreement, RNA-targeted medicine
Daiichi turns to Nosis for AI-designed RNA delivery tech
Nosis, Daiichi, RNA, Sankyo, Partnership, Biosciences, Cells, Chronic disease
FDA Pauses BioNTech’s Malaria Vaccine Trial Amid Regulatory Scrutiny
BioNTech, malaria vaccine, FDA clinical hold, RNA vaccine, regulatory challenges
Entrada given go-ahead to test DMD therapy after two-year hold
Entrada, Muscular Dystrophy, Duchenne, DMD, United States Food and Drug Administration, Adult, Exons
Merck & Co. Partners with Epitopea to Explore ‘Junk DNA’ for Novel Cancer Antigens
Merck & Co., Epitopea, cancer immunotherapy, Cryptigen tumor-specific antigens, CryptoMap platform, junk DNA, off-the-shelf immunotherapies
Biogen and Stoke Therapeutics Partner on Promising Dravet Syndrome Treatment
Biogen, Stoke Therapeutics, zorevunersen, Dravet syndrome, antisense oligonucleotide, rare epilepsy, drug development, pharmaceutical partnership