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Intellia’s in vivo CRISPR trial faces clinical hold after liver safety signal
Intellia Therapeutics disclosed voluntary pauses in dosing and screening across its Phase III MAGNITUDE trial and a second Phase III study after a patient experienced Grade 4 transaminase...
AAVantgarde raises $141M Series B to advance retinal gene therapies
AAVantgarde closed a $141 million Series B, led by Amgen Ventures, to advance gene‑therapy programs for inherited retinal diseases. The Milan‑based biotech will deploy the financing to accelerate...
Mass General Brigham researchers use tumor cells to kickstart antitumor immunity in models
Mass General Brigham researchers published preclinical data showing engineered tumor cells can be leveraged to restore innate immune sensing and trigger robust antitumor responses in animal...
TScan cuts workforce and halts solid‑tumor TCR program to focus on blood cancers
TScan Therapeutics announced a workforce reduction of roughly 30% and the discontinuation of a Phase I solid‑tumor T‑cell receptor (TCR) trial, shifting focus to hematologic indications. The...
UniQure’s Huntington Hopes Hit: FDA Signals Data Shortfall
UniQure said the FDA no longer agrees that its Phase I/II data for AMT-130—an experimental one-time gene therapy for Huntington’s disease—are adequate to support a Biologics License Application....
FDA Drug Office in Turmoil: CDER Director Resigns After Leave
George Tidmarsh resigned as director of the FDA’s Center for Drug Evaluation and Research (CDER) after being placed on administrative leave amid an HHS probe into his conduct. The department said...
Pfizer Files Suit to Block Novo Nordisk–Metsera Deal — Antitrust Fight Starts
Pfizer sued Metsera and Novo Nordisk in Delaware court seeking to stop Novo’s counter-offer to acquire obesity biotech Metsera, alleging breach of contract and an anticompetitive conspiracy....
Thermo Fisher Expands Into Clinical-Trial Tech: Clario Deal Agreed
Thermo Fisher Scientific agreed to acquire clinical trial data firm Clario in a transaction reported at roughly $8.8–$9 billion to broaden its digital capabilities in clinical research. Clario...
Roche Taps Manifold Bio’s Platform: $55M Deal to Pilot Brain Shuttles
Roche struck a strategic collaboration and license deal with Manifold Bio to develop next-generation 'brain shuttle' technologies for neurological diseases. The pact includes an upfront payment...
Off‑the‑Shelf CAR‑T Shows Promise: Caribou Posts Early Efficacy Signals
Caribou Biosciences reported early data suggesting its allogeneic, off‑the‑shelf CAR‑T candidate can induce deep remissions in B‑cell lymphoma. The preliminary results showed high overall and...
Intellia Halts Dosing: CRISPR Heart Program Faces FDA Clinical Hold
Intellia Therapeutics paused dosing and screening in Phase III and Phase II trials of its in vivo CRISPR candidate nexiguran ziclumeran after a patient experienced severe liver injury. The FDA...
Clinical Program Cutbacks: TScan Lays Off Staff, Stops Solid‑Tumor Trial
TScan Therapeutics announced workforce reductions of roughly 30% and the termination of a Phase I solid‑tumor T‑cell receptor program as the company refocuses resources on blood‑cancer...
Cyclin A2 Reboots Human Heart Cells: New Gene Therapy Drives Division
Researchers at Mount Sinai demonstrated that delivering the gene Cyclin A2 (CCNA2) via a human‑compatible viral vector can induce cytokinesis in adult human cardiomyocytes and promote cardiac...
Non‑toxic gene‑switch 'Cyclone' Debuts: Precise, Safer Control of Gene Activity
Weill Cornell Medicine investigators introduced a versatile gene‑switch system that uses a non‑toxic, clinically used antiviral molecule to control expression of target genes in cells. The...
uniQure’s Huntington therapy — FDA flags data shortfall
uniQure told investors that the FDA indicated data from its Phase I/II AMT‑130 program—benchmarked against an external historical control—may no longer be sufficient to support a Biologics License...
Roche bets on brain shuttles: $55M Manifold tie‑up to speed BBB delivery
Roche struck a discovery and licence collaboration with Manifold Bio to develop next‑generation brain shuttles, paying roughly $55 million upfront and structuring potential payments that could...
Italian gene‑therapy startup raises $141M — Amgen joins Series B
Milan‑based AAVantgarde closed a $141 million Series B round led by investors including Amgen Ventures to advance gene therapies for inherited retinal diseases. The company said proceeds will...
Intellia’s CRISPR program paused — FDA places clinical hold after liver signal
Intellia voluntarily paused dosing and screening in its Phase III MAGNITUDE program and a companion Phase III trial after a patient experienced Grade‑4 liver transaminase elevations and...
TScan slashes staff, abandons solid‑tumor trial — refocus on blood cancers
TScan announced workforce reductions of roughly 30% and confirmed it will stop a Phase I solid tumor T‑cell receptor (TCR) program to concentrate resources on hematologic indications. The...
Caribou’s off‑the‑shelf CAR‑T posts competitive lymphoma results — needs cash
Caribou Biosciences reported that its allogeneic CAR‑T candidate vespa‑cel produced high complete response (CR) and overall response rates in advanced B‑cell lymphoma, outcomes the company says...