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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...
Quantifying endosomal escape: new in vivo assays point the way for liver LNPs
Two independent reports introduced in vivo methods to quantify endosomal escape — the rate‑limiting step for lipid nanoparticle (LNP) delivery — and applied the assays to optimize liver-targeted...
BioNTech founders exit — next‑gen mRNA spinout in the works
Uğur Şahin and Özlem Türeci, BioNTech’s co‑founders, announced plans to leave the company by year‑end to launch a new venture focused on next‑generation mRNA technologies. BioNTech will grant the...
Atrium Therapeutics launched with $270M — RNA delivery to the heart
A new spinout, Atrium Therapeutics, launched with approximately $270 million in cash and assets carved out from Avidity Biosciences to pursue RNA therapeutics for rare genetic cardiomyopathies....
Evotec deepens restructuring — 800 roles and multiple site closures
German drug discovery and contract services firm Evotec announced a sweeping restructuring that will cut roughly one‑sixth of its workforce—about 800 people—and close four additional sites as part...
Manufacturing scale‑up: Lonza expands gene‑therapy capacity — CSL builds $1.5B plasma plant
Two major manufacturing investments signal expanded capacity for complex biologics: Lonza agreed to expand commercial manufacturing to continue production of Genetix Biotherapeutics' ZYNTEGLO gene...
Investors double down on AI biology — Breakout $114M, Living Models $7M
Venture capital continues to fund AI‑centric biology startups: Breakout Ventures closed a $114 million Fund III to back early‑stage companies translating academic breakthroughs into scalable...
Electrical stimulation advances restore movement and sensation after SCI
Two clinical teams reported advances in electrical neuromodulation that restored motor function and somatosensory feedback in people with chronic spinal cord injury (SCI). A Brown University–led...
FDA consolidates adverse‑event tracking — single monitoring system planned
The U.S. FDA announced plans to merge multiple adverse event (AE) reporting tools into a single Adverse Event Monitoring System that will ultimately centralize reports across agency centers,...