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FDA rejects Ultragenyx gene therapy: Manufacturing issues stall Sanfilippo treatment
The FDA has issued a complete response letter rejecting Ultragenyx's gene therapy UX111 for Sanfilippo syndrome type A due to manufacturing deficiencies observed during facility inspections....
AstraZeneca’s hypertension drug baxdrostat wins phase III trial, validates CinCor acquisition
AstraZeneca reported positive Phase III results for baxdrostat, an aldosterone synthase inhibitor targeting uncontrolled and treatment-resistant hypertension. The trial demonstrated significant...
Takeda’s oral orexin agonist cleares dual Phase III in narcolepsy, filing planned
Takeda’s orexin receptor 2-selective agonist, oveporexton (formerly TAK-861), met all primary and secondary endpoints in two registrational Phase III trials for narcolepsy type 1. The oral therapy...
Waters to merge with BD’s biosciences unit in $17.5 billion deal to expand life sciences reach
Waters Corporation and Becton, Dickinson & Co. (BD) announced a $17.5 billion transaction to combine Waters with BD’s Biosciences and Diagnostic Solutions business. The merger will nearly double...
Sonnet BioTherapeutics pivots to crypto under $888 million deal, becoming Hyperliquid Strategies
Sonnet BioTherapeutics announced a definitive agreement to transform via a $888 million business combination with Rorschach I LLC, affiliated with Atlas Merchant Capital and Paradigm Operations....
Nutcracker and Elegen unveil cell-free RNA manufacturing platform for personalized cancer therapies
Nutcracker Therapeutics and Elegen launched a pilot program demonstrating the industry's first fully synthetic, cell-free manufacturing platform for RNA-based personalized cancer therapeutics...
Ancestry bias skews CRISPR screens, missing critical cancer drug targets
Researchers from the Broad Institute and Dana-Farber Cancer Institute identified that CRISPR-Cas9 guide RNA efficiency varies by ancestry, with off-target effects occurring more frequently in cell...
Viome and Scripps partner to develop non-invasive RNA test for early colon cancer detection
Personalized medicine company Viome Life Sciences and Scripps Research have initiated a collaboration to develop and clinically validate an at-home RNA-based screening test for colorectal cancer...
Integrated Biosciences unveils precision optogenetic drug screening platform targeting age-related diseases
Integrated Biosciences published a Cell paper detailing a novel optogenetic screening platform that enables millisecond precision activation of the integrated stress response (ISR) pathway,...
Genetic variability shapes tumor immunotherapy response in mouse models
Using the genetically diverse Collaborative Cross mouse model, researchers demonstrated that genetic variability influences tumor response to immune checkpoint inhibitors. This work, published in...
Ultragenyx Gene Therapy Rejection Hits Rare Disease Program
The U.S. Food and Drug Administration issued a complete response letter (CRL) to Ultragenyx Pharmaceutical, declining approval for its gene therapy UX111 targeting Sanfilippo syndrome Type A, a...
AstraZeneca's Hypertension Drug Baxdrostat Shows Phase III Success
AstraZeneca reported positive phase III data for baxdrostat, an aldosterone synthase inhibitor acquired through its 2023 purchase of CinCor Pharma. The drug met primary and secondary endpoints by...
Takeda Advances Narcolepsy Treatment with Phase III Wins for Oveporexton
Takeda Pharmaceutical announced successful results from two Phase III studies evaluating its oral orexin receptor 2-selective agonist, oveporexton, for narcolepsy type 1. The drug met all primary...
Mink Therapeutics’ NK T-Cell Therapy Yields Durable Cancer Remission
Mink Therapeutics reported a complete and durable remission in a testicular cancer patient treated with its allogeneic invariant natural killer T-cell therapy Agent-797 combined with nivolumab....
AI Uncovers Novel Antibiotics in Venoms to Combat Resistance
Researchers harnessed artificial intelligence to identify hundreds of potential antibiotics from snake, scorpion, and spider venoms, offering a new avenue to tackle the rising global threat of...
Viome, Scripps Collaborate on At-Home RNA Test for Colon Cancer Detection
Viome Life Sciences partnered with Scripps Research to develop and validate an at-home RNA-based screening test aimed at detecting precancerous colon polyps, facilitating early prevention of...
Cellular Origins-Led Consortium Targets Automated Cell Therapy Manufacturing
Cellular Origins, Resolution Therapeutics, and Cell and Gene Therapy Catapult formed a consortium awarded £1 million by Innovate UK to develop a fully automated, scalable manufacturing platform...
Private Equity Fuels Expansion at PCI Pharma Services in Drug Production
Private equity firms including Bain Capital and Kolberg notably invested in PCI Pharma Services, valuing the company at $10 billion. The contract development and manufacturing organization...
Circular Genomics and WashU Identify Circular RNA Biomarker for Alzheimer’s
Circular Genomics, collaborating with Washington University, discovered a circular RNA-based signature, including circHOMER1, associated with Alzheimer’s disease onset and progression. This...
Nutcracker and Elegen Pilot Cell-Free RNA Manufacturing for Personalized Cancer Therapies
Nutcracker Therapeutics and Elegen initiated a pilot for a fully synthetic, cell-free manufacturing platform to produce RNA-based personalized cancer therapeutics (PCTs). The platform addresses...