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GSK doubles down on oligonucleotides: siRNA license and $950M buy
GlaxoSmithKline expanded its oligonucleotide and pulmonary portfolios with two major deals: a global license for two siRNA assets from Frontier Biotechnologies and a $950 million acquisition of...
FDA lifts hold on Intellia CRISPR trials — safety caveats attached
The FDA has lifted a clinical hold on Intellia Therapeutics’ Phase 3 CRISPR gene‑editing trials for transthyretin amyloidosis after reviewing safety data from a prior hepatic adverse event that...
Moderna settles patent dispute: $950M upfront to Arbutus and Roivant
Moderna agreed to pay $950 million upfront to Arbutus Biopharma and Roivant’s Genevant to settle long‑running patent claims tied to its Covid‑19 mRNA vaccine. The settlement closes a multi‑year...
Candid to list via reverse merger — $505M cash to push T‑cell engager pipeline
Candid Therapeutics announced a reverse merger with RallyBio to take the AI‑driven T‑cell engager developer public on Nasdaq, supported by a $505 million private financing. The combined company...
AGBT sparks high‑throughput sequencing race — rivals circle Illumina
Advances unveiled at the Advances in Genome Biology and Technology (AGBT) meeting signaled a renewed scramble for high‑throughput genomics: Element Biosciences, Ultima Genomics, Roche (Axelios 1),...
Prime Medicine pivots to file — prime editing therapy moves toward FDA
Prime Medicine said it will seek FDA approval for a previously shelved rare‑disease therapy after agency interactions signaled a regulatory environment for gene editing that may still be...
Pharma leans on AI: Tempus‑Merck biomarker pact and Evinova trial deals
Pharma companies are expanding AI partnerships to accelerate biomarker discovery and clinical development. Tempus AI and Merck signed a multi‑year deal to mine de‑identified clinical and molecular...
FDA urges sham‑surgery control — uniQure’s Huntington program stalls
The FDA has pushed back on uniQure’s Huntington’s disease program, recommending a randomized, double‑blind, sham‑surgery–controlled trial before it will consider approval. Company disclosures and...
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....