March 18, 2025
IRA round 2 begins as drugmakers sign on to price negotiation process with CMS
Myasthenic Syndromes, Congenital, Mediation, Prices, Medicare, Ira (eukaryote), drugmakers
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
Arch-backed HC Bioscience closes doors after seeing early data for tRNA asset
Vision, hydrocortisone, Bioscience
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
AstraZeneca’s $1bn hypoparathyroidism drug achieves success at Phase III
eneboparatide, hypoparathyroidism, Phase III trial, Calypso study, Amolyt Pharma acquisition
Lonza’s Vision for the Future: A Fireside Chat with New CEO Wolfgang Wienand
Lonza, Wolfgang Wienand, CDMO, Pharmaceutical manufacturing, Biologics, Cell and gene therapy, Small molecules, Leadership transition, Industry outlook
Curevo Secures $110M for Advanced Shingles Vaccine Development
Curevo Vaccine, amezosvatein, shingles vaccine, Series B funding, improved tolerability, Shingrix competitor
AstraZeneca deepens cell therapy expansion with $1B EsoBiotec buyout
AstraZeneca, EsoBiotec, cell therapy, acquisition, ENaBL platform, in vivo cell engineering, oncology, autoimmune diseases
Incyte’s oral HS drug hits study goals, but fails to excite investors
povorcitinib, JAK1 inhibitor, hidradenitis suppurativa, Phase 3 trials, efficacy, investor reaction, regulatory submission