Get the Daily Brief
Latest Biotech News
FDA pivots to one pivotal trial: CROs and developers demand rulebook
The FDA’s newly stated default that one adequate and well‑controlled study may suffice for approval has prompted industry calls for clear guidance. ACRO, the CRO trade group, urged the agency to...
PepGen trial paused: FDA partial hold hobbles rare‑disease program
The FDA placed a partial clinical hold on PepGen’s Phase 2 study for a rare muscle disorder after reviewing preclinical data and raising product‑characterization questions. The stoppage affects...
Gene regulation therapy slashes seizures in Dravet trials — unprecedented readouts
Clinical data released for zorevunersen, a gene‑regulation therapy for Dravet syndrome, showed dramatic seizure reductions and functional gains in pediatric patients. In open‑label cohorts...
GLP‑1s reshape care — prescriptions spike, surgeries fall; FDA targets compounders
Rapid uptake of GLP‑1 receptor agonists such as semaglutide and tirzepatide is shifting clinical practice: prescriptions have surged among patients eligible for metabolic and bariatric surgery,...
Big bets on brain and blood: Cognito raises $105M; Atavistik banks $160M
Cognito Therapeutics closed a $105 million Series C to propel its Spectris non‑invasive brain‑stimulation platform through pivotal development for Alzheimer’s disease and to scale operations for...
UCLA engineers CAR‑T to confront solid tumors — next‑gen design reported
Researchers at UCLA disclosed a next‑generation CAR‑T construct engineered to resist the suppressive tumor microenvironment common in solid cancers. The team reported preclinical data showing...
UCB inks TCE pact: Antengene’s CD19xCD3 program licensed for up to $1.1B
UCB struck a licensing agreement to develop Antengene’s ATG‑201, a CD19/CD3 bispecific T‑cell engager aimed at autoimmune diseases, in a deal valued at up to $1.1 billion. The transaction gives...
Theravance shutters R&D after Phase III failure — mass cuts follow
Theravance Biopharma confirmed it will wind down research and development activities and cut roughly half its workforce after its norepinephrine reuptake inhibitor failed a Phase III study in...
Liquid biopsy goes beyond cancer: cfDNA fragmentomics flags liver disease
Johns Hopkins investigators published pilot data showing genome‑wide cell‑free DNA (cfDNA) fragmentation and fragmentomic signatures can identify liver fibrosis and cirrhosis, expanding...
Moderna to pay up to $2.25B — ends mRNA lipid nanoparticle patent fight
Moderna agreed to settle a long-running patent dispute over lipid nanoparticle (LNP) delivery technology with Roivant subsidiary Genevant and Arbutus, committing up to $2.25 billion in payouts....
Gyre agrees to absorb Cullgen... $300M all‑stock transaction expands TPD, DAC pipeline
Gyre Therapeutics announced an all‑stock deal to acquire Cullgen in a transaction valued at roughly $300 million, giving Gyre immediate access to Cullgen’s targeted protein degradation (TPD)...
Gene therapy zorevunersen dramatically lowers seizures in Dravet — NEJM data
Clinical results published in the New England Journal of Medicine show the investigational gene regulation therapy zorevunersen produced large seizure reductions and functional improvements in...
Prime tests FDA flexibility — seeks approval from two‑patient prime editing study
Prime Medicine reversed a prior deprioritization and said it will pursue FDA approval for PM359, a prime‑editing therapy for chronic granulomatous disease (CGD), based on data from just two...
Theravance halves workforce, ends R&D after pivotal ampreloxetine failure
Theravance Biopharma announced a major restructuring after its phase III ampreloxetine program missed its primary endpoint, triggering a decision to wind down the company’s entire R&D organization...
FDA escalates crackdown on compounded GLP‑1s — 30 warning letters target telehealth firms
The U.S. Food and Drug Administration issued a fresh wave of enforcement letters aimed at telehealth and compounding pharmacies marketing unapproved compounded versions of GLP‑1 diabetes and...
Liquid biopsy fragmentomics extends beyond cancer — cfDNA patterns identify liver disease
Researchers from Johns Hopkins and collaborators published pilot studies demonstrating that genome‑wide cell‑free DNA (cfDNA) fragmentomics and AI analysis can detect liver fibrosis and cirrhosis,...
UCLA engineers CAR‑T cells designed to breach solid‑tumor defenses
UCLA scientists reported development of next‑generation CAR‑T cells engineered to overcome the immunosuppressive microenvironment characteristic of many solid tumors. The platform includes...
Evo 2 DNA foundation model: AI designs genomes across life and flags disease variants
Researchers published Evo 2, a DNA foundation model trained on genomic sequences from over 100,000 species, reporting capabilities to predict disease‑causing human mutations and generate whole...
ARPA‑H backs lymphedema programs — Rice and Georgia Tech win major grants
Two ARPA‑H awards seeded sizable academic efforts to develop regenerative lymphatic therapies. Rice University secured up to $18.2 million to build the first regenerative treatment for lymphedema,...
Gene therapy cuts seizures in Dravet: NEJM reports dramatic drops
An experimental gene-regulation therapy, zorevunersen, produced large reductions in seizure frequency for children with Dravet syndrome in multicenter studies led by University College London and...