Viral Vector– tag –
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UC Riverside-Led Study: Gene Therapy Reverses Fragile X Deficits, Restoring Brain Activity and Improving Behavior in Mouse Model
UCR News - UC Riverside USA Overview A University of California, Riverside-led study demonstrated that a gene therapy designed to replace a missing brain protein successfully restored normal brain activity and improved behavior in a mous... -
SonoThera Raises $125M Series B to Advance Safer Ultrasound-Mediated Gene Therapies for Duchenne Muscular Dystrophy and ADPKD into Clinical Development
BioSpace USA Overview SonoThera has secured $125 million in Series B financing to advance its gene therapy programs for Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) into clinical development.... -
Cell Therapy Weekly: uniQure Plans BLA Submission for Huntington’s Gene Therapy AMT-130; Ernexa Prepares IND for iMSC, Autolus Reports Early Phase I Data for SLE CAR-T
Cell & Gene Therapy Insights USA Overview uniQure plans to submit a Biologics License Application (BLA) for AMT-130, a gene therapy for Huntington's disease, in Q3 2026, based on three-year Phase I/II data. Ernexa Therapeutics has comple... -
Portal Biotechnologies Raises $9M to Expand Cell Engineering Platform, Attracting Merck & Co. and AbbVie
FirstWord HealthTech USA Overview Portal Biotechnologies secured $9 million to advance its cell engineering platform, which has drawn partnerships with Merck & Co. and AbbVie. The platform leverages mechanoporation for efficient intr... -
CCRM, OmniaBio, and Avectas Collaborate to Automate and Scale Cell Therapy Manufacturing
PR Newswire Canada Overview CCRM and its CDMO subsidiary OmniaBio Inc. have partnered with Avectas Limited to evaluate and integrate Avectas' automated, scalable cell therapy manufacturing platform. This collaboration aims to enhance con... -
UT MD Anderson Develops Exosome-Based Therapy for Duchenne Muscular Dystrophy, Delivering Full-Length DMD mRNA to Dramatically Restore Muscle Function In Vivo
Nature Biomedical Engineering (via The University of Texas MD Anderson Cancer Center) USA Overview Researchers at The University of Texas MD Anderson Cancer Center have developed a novel therapeutic platform utilizing engineered extracel... -
uniQure Announces Plan for BLA Submission of Huntington’s Disease Gene Therapy AMT-130, FDA Accepts Phase 1/2 3-Year Data for Accelerated Approval
uniQure Press Release USA Overview uniQure has announced that the FDA agreed in a Type B meeting that 3-year analysis data from its Phase 1/2 trial of gene therapy AMT-130 for Huntington's Disease (HD) would be acceptable as primary evid... -
Lipid Nanoparticles vs. Viral Vectors for Gene Delivery: A Comparative Analysis of Advantages and Applications
Pharma Almanac USA Overview This comparative analysis explores the distinct strengths of lipid nanoparticles (LNPs) and viral vectors in gene delivery. While viral vectors like AAV and lentivirus excel in high-efficiency, durable gene ex... -
Lonza Emphasizes End-to-End Process Scalability for High-Titer AAV Production Using HEK293 Media
Lonza Switzerland Overview Lonza underscores the critical importance of end-to-end process scalability for high-titer AAV production utilizing HEK293 media. The article highlights the pivotal role of benchtop stirred-tank bioreactors in ... -
FDA Releases Draft Guidance to Streamline Gene Therapy Development, Aiming to Accelerate Access to Novel Therapies
BioProcess International USA Overview The FDA has issued draft guidance for gene therapy development, designed to streamline the process by enabling developers to leverage prior knowledge and platform data. This non-binding document from...
