Get the Daily Brief
Latest Biotech News
FDA greenlights leucovorin for rare folate disorder — autism claim not endorsed
The U.S. Food and Drug Administration approved leucovorin (Wellcovorin) for cerebral folate deficiency caused by variants in the folate receptor 1 gene, narrowing the drug’s labeled use to a...
FDA consolidates safety signals: one adverse-event database for all centers
The FDA announced a major overhaul of its safety surveillance infrastructure by consolidating multiple adverse-event reporting systems into a single, unified Adverse Event Monitoring System. The...
New in vivo assays map LNP endosomal escape — liver delivery leaps forward
Two complementary Nature Biotechnology reports introduced quantitative in vivo assays that measure endosomal escape of lipid nanoparticles (LNPs) in animal models, providing actionable readouts to...
Immune‑evasive DNA donors enable safe, gene‑sized insertions
Researchers reported an immune‑evasive approach to insert kilobase-scale DNA payloads into mammalian genomes without triggering toxic innate immune responses. Teams developed circular...
Regenxbio posts clean DMD safety ahead of pivotal readout; FDA revisits another DMD program
Regenxbio reported a clean safety profile and functional improvement signals for its Duchenne muscular dystrophy (DMD) gene therapy as pivotal data approach, reinforcing the program’s regulatory...
GSK sells linerixibat to Alfasigma in $690m deal as FDA decision looms
GSK agreed to transfer worldwide rights to linerixibat, an IBAT inhibitor for primary biliary cholangitis (PBC) pruritus, to Italy’s Alfasigma in a deal worth up to $690 million including a $300...
Avidity spinout Atrium raises $270m to deliver RNA to the heart
Atrium Therapeutics launched as a spinout funded with roughly $270 million in cash to advance Avidity Biosciences’ preclinical cardiac RNA programs. The new company is advancing two lead...
Vertex files BLA after Rainier win — povetacicept seeks accelerated approval
Vertex signaled plans for rapid regulatory action after the Phase 3 Rainier trial showed a pronounced reduction in proteinuria for immunoglobulin A nephropathy (IgAN). The company intends to...
Evotec cuts roughly 800 roles as restructuring widens
Evotec announced an expanded restructuring that will close four sites and cut about one‑sixth of its workforce—roughly 800 positions—in a multiyear transformation aimed at rebalancing the German...
BioNTech founders to depart and launch next‑gen mRNA startup
BioNTech co‑founders Ugur Sahin and Özlem Türeci will step down to establish a new company focused on next‑generation mRNA technologies. BioNTech will grant the startup certain technology rights...
Immune‑evasive DNA donors clear kilobase insertion barrier
Researchers reported a new donor design that allows safe insertion of kilobase‑scale DNA into mammalian genomes without triggering toxic innate immune responses. Teams at Massachusetts General...
In vivo endosomal assays pinpoint LNP escape—liver delivery optimized
Two independent teams introduced quantitative in vivo assays that measure lipid nanoparticle (LNP) endosomal escape and used the readouts to improve hepatic delivery. Groups publishing in Nature...
Nanoparticles program CAR T cells in vivo: mice show B‑cell depletion
Johns Hopkins teams reported biodegradable, targeted polymeric nanoparticles that deliver mRNA encoding anti‑CD19 CARs directly to T cells in vivo, producing functional CAR T cells and depleting B...
Securitization and spinouts fuel cell and gene therapy scale‑up
Two financing approaches surfaced as potential solutions to the high capital demands of advanced therapies: a major spinout and an academic analysis of securitization. A new company, Atrium...
FDA narrows leucovorin to rare cerebral folate deficiency—autism claims checked
The FDA approved leucovorin (Wellcovorin) for cerebral folate deficiency due to confirmed FOLR1 variants but rejected broader claims linking the drug to autism treatment. Agency notices and...
Lonza expands capacity to make ZYNTEGLO—gene‑therapy scale‑up
Lonza will expand commercial manufacturing capacity at its Houston cell and gene therapy site to support Genetix Biotherapeutics’ ZYNTEGLO, the only FDA‑approved gene therapy for...
Optical genome mapping finds hidden variants in acute leukemia
A Canadian tertiary care study showed optical genome mapping (OGM) detected additional clinically significant structural variants in nearly 20% of acute leukemia patients when used as a first‑line...
UCB’s Bimzelx bests AbbVie’s Skyrizi in psoriatic arthritis head‑to‑head
UCB reported that its IL‑17A/F antibody Bimzelx achieved statistically significant superiority to AbbVie’s IL‑23 inhibitor Skyrizi in a head‑to‑head psoriatic arthritis trial, the first time an...
Salspera files IPO to fund phase 3 of salmonella IL‑2 cancer therapy
Salspera Health filed for a $91 million IPO to advance saltikva, an engineered Salmonella strain expressing human IL‑2, into phase‑3 studies in metastatic pancreatic cancer and other solid tumors....
Nanoliposomes revive antibiotics against multidrug‑resistant Salmonella
Researchers described nanoliposome formulations that potentiate conventional antibiotics against multidrug‑resistant Salmonella enterica serovar Typhimurium, restoring bactericidal activity in...