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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...
FDA narrows leucovorin use: approves for rare cerebral folate defect, not autism
The U.S. Food and Drug Administration approved leucovorin (Wellcovorin) for cerebral folate deficiency caused by pathogenic variants in the folate receptor 1 gene, but declined broader approval...
Optical genome mapping finds hidden variants in acute leukemia — nearly 1 in 5 cases
A Canadian tertiary‑care study applying optical genome mapping (OGM) as a first‑line test identified previously undetected structural variants in nearly 20% of acute leukemia patients. Published...
Nanoparticles program in vivo CAR T cells: Johns Hopkins reports mouse success
Johns Hopkins researchers reported that targeted biodegradable polymer nanoparticles delivering mRNA encoding an anti‑CD19 CAR can reprogram T cells inside mice to produce functional CAR T cells...
Safe large‑DNA insertion: stealth DNA circles enable kilobase‑scale genome writing
Researchers described a circular single‑stranded DNA donor approach that permits kilobase‑scale, non‑viral insertions while minimizing innate immune activation. The method—reported in Nature and...
Quantifying endosomal escape: new in vivo assays guide LNP design for liver delivery
Two complementary reports in Nature Biotechnology deliver in vivo assays that quantify endosomal escape and endosomal trafficking of lipid nanoparticles (LNPs), pinpointing why some formulations...
Vertex races to FDA: phase‑3 win for povetacicept yields near‑50% proteinuria cut
Vertex reported phase‑3 Rainier results for povetacicept showing a robust placebo‑adjusted reduction in urine protein‑to‑creatinine ratio (~49.8% at interim), and the company is preparing a BLA...
GLP‑1 drugs linked to lower substance‑use risk in large veterans cohort
An observational study of more than 600,000 U.S. veterans reported that GLP‑1 receptor agonist use was associated with reduced incidence of substance‑use disorders and improved outcomes among...
BioNTech founders exit to start next‑gen mRNA company as firm refocuses on oncology
BioNTech announced that co‑founders Ugur Sahin and Özlem Türeci will step down by year‑end to found a new company focused on next‑generation mRNA technologies. BioNTech will grant limited rights...
China firms push lower‑cost rivals to Zolgensma: four gene‑therapy contenders emerge
Chinese biotech companies are developing lower‑cost gene therapies to compete with Novartis’s $2.1M Zolgensma for spinal muscular atrophy (SMA), driven by affordability and access gaps in...
Securitization pitched to fund cell and gene therapy costs — new financing model proposed
Researchers evaluated securitization as a financing mechanism to spread upfront development and manufacturing costs for expensive cell and gene therapies targeting orphan diseases. The analysis...
FDA clears leucovorin for rare folate defect — autism use rejected
The U.S. Food and Drug Administration approved Wellcovorin (leucovorin) for cerebral folate deficiency caused by pathogenic variants in the folate receptor 1 (CFD‑FOLR1), while explicitly...
Vertex files BLA after Phase III Rainier win — race to FDA
Vertex Pharmaceuticals reported Phase III results for povetacicept in immunoglobulin A nephropathy (IgAN) and is moving rapidly toward a BLA submission seeking accelerated approval. The Rainier...