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Nonprofit rescues abandoned gene therapy — can the model scale?
A nonprofit effort brought an orphaned rare‑disease gene therapy to market by rescuing development and facilitating access, according to BioCentury’s reporting. The case involved Fondazione...
FDA issues two Form 483s to Catalent’s Maryland gene‑therapy sites
The FDA issued two Form 483 observations to Catalent’s Maryland facilities that manufacture gene‑therapy products prior to a round of mass layoffs at the sites. The notices relate to manufacturing...
BioMarin buys Amicus for $4.8B — rare-disease portfolio enlarged
BioMarin agreed to acquire Amicus Therapeutics for $4.8 billion, buying two approved therapies for Fabry and Pompe disease and gaining U.S. rights to a late‑stage candidate for focal segmental...
FDA clears Cytokinetics’ heart drug — company gets first U.S. approval
The FDA approved Cytokinetics’ Myqorzo (first U.S. approval), a therapy for obstructive hypertrophic cardiomyopathy, marking the company’s first marketed product after nearly three decades....
Partial hold on Merck‑Daiichi ADC trial after fatal events
Daiichi Sankyo and Merck paused enrollment and the FDA placed a partial hold on the Phase III IDeate‑Lung02 study of the ADC ifinatamab deruxtecan following a higher‑than‑anticipated incidence of...
CEPI backs Moderna H5 mRNA program — Phase 3 push funded
The Coalition for Epidemic Preparedness Innovations (CEPI) agreed to fund Moderna’s mRNA H5 avian influenza vaccine candidate with up to $54.3 million to advance the program into Phase 3 testing....
Single‑cell assays and variant mapping: tools for translation
Two technology stories spotlight efforts to scale single‑cell and variant‑effect assays for translational discovery. Swedish spinout Epigenovo is commercializing scFFPE‑ATAC, a single‑cell...
Galux and Boehringer ink AI protein‑design pact
Galux, a South Korean AI‑driven protein therapeutics startup, signed a research agreement with Boehringer Ingelheim to explore AI‑powered precision protein design. The collaboration will evaluate...
AI life‑science funding heats up: Insilico IPO, Edison seed round
AI drug‑discovery and research‑software financing accelerated as Insilico Medicine filed to raise HK$2.27 billion (about $292M) in a Hong Kong IPO to advance its clinical pipeline and generative...
White House finalizes nine MFN drug deals — pricing and policy move
The White House announced 'most favored nation' (MFN) style agreements with nine major drugmakers — including Amgen, BMS, Gilead and Novartis — in an end‑of‑year policy push to align some U.S....
JAX completes NYSCF unification — new nonprofit research engine
The Jackson Laboratory (JAX) completed its acquisition of the New York Stem Cell Foundation (NYSCF) and launched a unified organization in New York City, integrating JAX’s genetics and mouse‑model...
Gene‑therapy access vs. manufacturing scrutiny: nonprofit rescue, Catalent 483s
A nonprofit organization successfully brought an abandoned rare‑disease gene therapy to market, illustrating alternative models to rescue small or orphan programs that big pharma has shelved....
BioMarin to buy Amicus: $4.8B bet on Fabry and Pompe franchises
BioMarin agreed to acquire Amicus Therapeutics for $4.8 billion in an all-cash transaction that the companies expect to close in the second quarter of 2026. The deal transfers two marketed...
FDA halts Merck–Daiichi ADC trial: deaths trigger partial hold
Regulators and sponsors paused a Phase III study of the antibody‑drug conjugate I‑Dxd after an unexpectedly high incidence of fatal interstitial lung disease (ILD) events. Daiichi Sankyo...
Cytokinetics wins FDA green light: first U.S. drug approved
The FDA approved Cytokinetics’ Myqorzo, the company’s first U.S. drug, for obstructive hypertrophic cardiomyopathy (oHCM). The approval closes a 27‑year effort by the biotech and establishes...
Takeda’s TYK2 drug clears Phase 3—regulatory filings planned
Takeda reported that zasocitinib, a TYK2 inhibitor acquired via a major deal, met co‑primary and key secondary endpoints in two Phase 3 trials for plaque psoriasis. The company said more than half...
Lilly’s oral GLP-1 hits maintenance goal — review accelerated
Eli Lilly disclosed positive top‑line results from the Phase 3 Attain‑Maintain trial: its oral GLP‑1 receptor agonist orforglipron met primary and key secondary endpoints for weight maintenance...
CEPI backs Moderna’s H5 mRNA shot: funds Phase 3 push
Moderna secured up to $54.3 million from CEPI to advance its mRNA H5 influenza vaccine candidate, mRNA‑1018, into Phase 3 testing. The funding restores external support after the company’s earlier...
Parse and Codebreaker join to map variant effects with single‑cell readouts
Parse Biosciences and Codebreaker Labs launched a collaboration to combine engineered variant libraries with high‑throughput single‑cell transcriptomics to experimentally characterize how genetic...
Epigenovo pushes scFFPE‑ATAC: single‑cell chromatin assay for FFPE tissue
Epigenovo, a Uppsala University spinout, is commercializing scFFPE‑ATAC, a single‑cell chromatin accessibility assay compatible with formalin‑fixed paraffin‑embedded (FFPE) samples. The startup...