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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...
FDA Clears Ascendis’ Weekly Dwarfism Shot – Market Battle Begins
The FDA granted accelerated approval to Ascendis Pharma’s TransCon CNP (now Yuviwel) for children two years and older with achondroplasia, using annualized growth velocity as the surrogate basis...
Cardiac Signal Halts Aardvark’s Phase 3 Hunger Trial – Data Review Underway
Aardvark Therapeutics voluntarily paused dosing and enrollment in its Phase 3 HERO trial of ARD‑101 for hyperphagia in Prader‑Willi syndrome after routine safety monitoring in a healthy volunteer...
Candid to Go Public via Rallybio Reverse Merger – $505M War Chest
Candid Therapeutics agreed to a reverse merger with Rallybio that will leave the combined company publicly traded and well‑capitalized after a $505 million private financing. The deal transfers...
Merck Expands Biomarker Hunt With Tempus AI – Precision Oncology Focus
Merck has expanded a multi‑year collaboration with Tempus AI to accelerate discovery of precision‑medicine biomarkers across oncology and potentially other therapeutic areas. Under the agreement,...
Asia Deals: GSK Buys siRNA Programs; Astellas Taps Vir for a T‑Cell Engager
GlaxoSmithKline struck a global exclusive licensing deal with China’s Frontier Biotechnologies for two siRNA therapeutic candidates—one IND‑ready and one preclinical—paying $40 million upfront and...
Earendil Locks Payload-Linker Tech With WuXi XDC in Up‑to‑$885M Pact
Earendil Labs signed a licensing and development agreement with WuXi XDC to access payload‑linker technology and end‑to‑end CRDMO services for antibody‑drug conjugates, in a deal valued at up to...
Hepion Pivots to Liquid Biopsy – Licenses ctRNA Assay for Early HCC Detection
Hepion Pharmaceuticals licensed rights from Cirna Diagnostics to develop a circulating tumor RNA (ctRNA) assay aimed at detecting early, high‑risk hepatocellular carcinoma (HCC). The deal includes...
Theravance Winds Down R&D After Phase 3 Failure – Major Jobs Impact
Theravance Biopharma announced it will wind down its R&D organization and cut roughly 50% of its workforce after a Phase 3 failure for ampreloxetine, its experimental candidate. The company said...
FDA Demands Sham-Controlled Study: uniQure’s AMT-130 Approval Path Blocked
The FDA has told uniQure that existing single-arm data for its Huntington’s gene therapy AMT‑130 are insufficient and has strongly recommended a randomized, double‑blind, sham surgery–controlled...
Phase III Halted: Aardvark Pauses PWS HERO Trial After Cardiac Signals
Aardvark Therapeutics voluntarily paused dosing and enrollment in its Phase III HERO trial of ARD‑101 for hyperphagia in Prader‑Willi syndrome after routine safety monitoring in a...
FDA Grants Accelerated Approval: Ascendis’ Yuviwel Cleared for Achondroplasia
The FDA granted accelerated approval to Ascendis Pharma’s TransCon CNP (now Yuviwel, navepegritide) for children aged two and older with achondroplasia, ending BioMarin’s practical monopoly on the...
FDA Lifts Hold: Intellia Restarts Phase 3 CRISPR Trial With Safety Limits
The FDA has fully lifted a clinical hold on Intellia’s Magnitude Phase 3 trial of CRISPR gene editing candidate nexiguran ziclumeran (nex‑z) for ATTR‑CM, allowing enrollment to resume under...