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Aptar Pharma and Aceso Therapeutics have partnered to advance ACT-101, an antisense oligonucleotide targeting the underlying mechanisms of cystic fibrosis.

Moonwalk Biosciences has closed a $70 million Series B to advance its adipose-targeted RNAi platform toward first-in-human studies.

Sebastian Arana, head of process solutions at MilliporeSigma, discusses the operational tradeoffs facilities face as they move toward multi-modality production and the persistent cost and scale-up challenges shaping cell and gene therapy, mRNA, and ADC manufacturing.

Back Bay’s director discusses why IBD remains underserved — JAK inhibitor constraints, emerging oral mechanisms, and the durability gap.

Christian Thienel on the oral IBD mechanisms generating the most excitement — SIK2, oral IL-23, TYK2 — and why multiple could succeed.

Carelon Research's Mark Cziraky on why analyzing RWD in place — rather than licensing it out — is reshaping how evidence gets generated.

Ionis wins FDA approval for Zanvastro, the first disease-modifying treatment for Alexander disease, Pharmaceutical Executive’s weekly biotech roundup gathers analyst reaction to the week's biggest stories, and Nate Hill of Strive Pharmacy talks through the regulatory uncertainty around compounded peptides.

FDA has approved Ionis Pharmaceuticals' Zanvastro (zilganersen) as the first disease-modifying treatment for Alexander disease.

CAR-T safety halts, a rare disease trial failure, a China licensing deal, an mRNA flu vaccine advance, and nine new MFN agreements.

Nate Hill, co-founder & CEO, Strive Pharmacy, breaks down looming questions around the completed FDA PCAC hearing on peptides.

Ultragenyx shares plunge after apazunersen fails its Phase III trial in Angelman syndrome, Mark Cziraky of Carelon Research talks through the mechanics of analyzing linked real-world data sets, and Melanie Whittington joins The Ron Lanton Report to argue biopharma policy needs to stay tethered to patients, not abstractions.

Ultragenyx shares plunge more than 40% after apazunersen fails to meet both endpoints in a Phase III trial for Angelman syndrome.

Melanie Whittington, managing director and head of the Leerink Center for Pharmacoeconomics, joins Ron Lanton to argue that biopharmaceutical policy only makes sense when traced back to its purpose.