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FDA faults controls and surrogates — Regenxbio’s Hunter filing rejected
The FDA issued a complete response letter rejecting Regenxbio’s Biologics License Application for RGX‑121, citing inadequate controls, heterogeneous patient sampling, and insufficient evidence...
Phase‑3 failure forces restructuring — Theravance ends R&D, halves workforce
Theravance Biopharma confirmed a Phase 3 failure for ampreloxetine in multiple system atrophy and announced an immediate wind‑down of its R&D organization and a 50% workforce reduction. The...
Moderna pays up — $950M upfront, deal could swell to $2.25B
Moderna agreed to settle long‑running patent claims over its Covid‑19 vaccine by paying $950 million upfront to Arbutus Biopharma and Roivant’s Genevant, with contingent payments that could push...
GSK doubles down on oligonucleotide and PAH bets — two asset deals
GlaxoSmithKline struck two deals to broaden its pipeline: a licensing agreement with Frontier Biotechnologies for two siRNA immunology assets (up to $1 billion in milestones) and a $950 million...
FDA grants accelerated approval — Ascendis’ weekly dwarfism shot cleared
The FDA granted accelerated approval to Ascendis Pharma’s once‑weekly TransCon CNP (Yuviwel) for children aged two and older with achondroplasia, offering an alternative to the daily injection...
Prime editing edges toward clinic — company files for rare immune disease approval
Prime Medicine reversed an earlier decision and will file for FDA approval of a prime‑editing gene therapy after treating two patients in a bespoke trial, marking the first regulatory push for a...
Roche posts third Phase 3 MS win — liver safety questions follow
Roche reported a third positive Phase 3 readout for its BTK inhibitor fenebrutinib in multiple sclerosis but acknowledged a safety signal: a higher number of deaths in the fenebrutinib arm...
Aardvark halts Phase 3 HERO after cardiac signals — shares tumble
Aardvark Therapeutics voluntarily paused dosing and enrollment in its Phase 3 HERO trial of ARD‑101 for hyperphagia in Prader‑Willi syndrome after safety monitors observed reversible cardiac...
Big pharma bets on AI for biomarkers and trial ops — Merck, Tempus and Evinova deals
Merck expanded a multi‑year collaboration with Tempus to use Tempus’ de‑identified multimodal datasets and AI platform for biomarker discovery in oncology and other disease areas. Separately,...
Theravance collapses: Phase 3 flop triggers R&D shutdown
Theravance announced a decisive strategic retrenchment after its norepinephrine reuptake inhibitor ampreloxetine failed a pivotal Phase 3 study in multiple system atrophy and related neurogenic...
FDA rebukes Regenxbio: rejection cites controls and surrogate endpoints
The FDA issued a complete response letter rejecting Regenxbio’s gene therapy for Hunter syndrome, explicitly faulting trial population heterogeneity, inadequate external controls and reliance on...
FDA pressures uniQure: sham‑controlled trial urged for Huntington’s program
The FDA has strongly recommended that uniQure conduct a prospective, randomized, double‑blind, sham surgery‑controlled study before it will consider approval of AMT‑130 for Huntington’s disease....
Intellia cleared with conditions: CRISPR trials resume under tighter monitoring
The FDA lifted clinical holds on two Intellia Phase 3 trials of its CRISPR‑based gene editing therapies after a prior safety event, allowing enrollment to resume with added liver‑safety measures....
Ascendis wins FDA nod: weekly dwarfism shot cleared on accelerated basis
The FDA granted accelerated approval to Ascendis Pharma’s once‑weekly TransCon CNP (Yuviwel) for children with achondroplasia, clearing the company to compete with BioMarin’s Voxzogo. The decision...
GSK doubles down: siRNA license and $950M buy amplify pipeline bets
GSK struck multiple deals this week to bulk up its pipeline: a global license for two Frontier Biotechnologies siRNA programs worth up to ~$1 billion, and a $950 million cash acquisition of...
Merck ends Gardasil runs at Durham plant — vaccine demand slump forces cuts
Merck notified state authorities it will stop producing Gardasil and Gardasil 9 at its Durham, N.C., facility and lay off roughly 154 employees as a result of weakened global demand, particularly...
Aardvark pauses Phase 3 HERO trial after cardiac signals in volunteers
Aardvark Therapeutics voluntarily paused dosing and enrollment in its Phase 3 HERO trial for ARD‑101 in Prader‑Willi syndrome after routine safety monitoring in a separate healthy‑volunteer study...
AGBT fallout: sequencing vendors tee up a high‑throughput showdown
Announcements and demos at AGBT signaled intensifying competition at the high end of sequencing: Element Biosciences, Ultima Genomics, Roche (Axelios nanopore), and Complete Genomics all showcased...
FDA Seeks Sham-Controlled Study – UniQure's Huntington Program Stalls
The FDA has told UniQure it expects a randomized, double-blind, sham surgery–controlled trial before it will consider a marketing application for AMT‑130, the company’s gene therapy for...
FDA Lifts Hold on Intellia’s CRISPR Trial... Safety Conditions Apply
Regulators have lifted an FDA hold on Intellia’s Magnitude Phase 3 study of nexiguran ziclumeran for transthyretin amyloidosis with cardiomyopathy, allowing the trial to resume under new safety...