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iPS-Derived Dopaminergic Neuron Transplants Show Early Safety and Efficacy, Offering New Hope for Parkinson’s Disease Treatment
NeurologyLive USA Overview Stem cell therapy involving the transplantation of iPS cell-derived dopaminergic neurons into the brain is emerging as a promising approach for Parkinson's disease (PD) treatment. Initial clinical trial results... -
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... -
Minaris Strengthens Philadelphia GMP Facility, Integrating Cell & Gene Therapy Manufacturing and Testing to Streamline Production
BriefGlance.com USA Overview Minaris, a leading CDMO, has significantly upgraded its GMP cell banking suite at its Philadelphia facility, aiming to streamline cell and gene therapy production. This expansion integrates manufacturing and ... -
Intellia Therapeutics Reports “Paradigm-Shifting” Phase 3 Data for Lonvoguran Ziclumeran, Achieving 87% Reduction in HAE Attacks with Single In Vivo CRISPR Dose
Fierce Biotech USA Overview Intellia Therapeutics announced groundbreaking Phase 3 HAELO trial data for lonvoguran ziclumeran (lonvo-z), an *in vivo* CRISPR gene-editing therapy for hereditary angioedema (HAE). This one-time treatment de... -
Broad Institute Achieves Major Breakthrough in Prime Editing: Enhanced In Vivo Delivery with Optimized LNPs Paves Way for Broader Genetic Disease Therapies
Broad Institute USA Overview Researchers at the Broad Institute have significantly improved the efficiency and specificity of prime editing by optimizing its core components and developing an advanced lipid nanoparticle (LNP) delivery sy... -
Beam Therapeutics Receives FDA IND Clearance for BEAM-304, Advancing Base Editing Therapy for Phenylketonuria (PKU) into Clinical Development
GlobeNewswire USA Overview Beam Therapeutics Inc. announced that the FDA has cleared its Investigational New Drug (IND) application for BEAM-304, a base editing therapy for Phenylketonuria (PKU). PKU is a rare genetic metabolic disorder ... -
Jazz Pharmaceuticals and AbCellera Partner for Next-Gen Multispecific T-Cell Engagers Targeting GI and Solid Tumors, with Up to $848M in Potential Payments
PR Newswire USA Overview Jazz Pharmaceuticals and AbCellera have entered a research collaboration, option, and license agreement to discover and develop next-generation multispecific T-cell engager antibodies for gastrointestinal cancers... -
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... -
Autolus Therapeutics Wins Prix Galien UK for Best Biotech Product, Reports Promising Early Phase 1 Data for obe-cel CAR-T in Refractory SLE
Stock Titan UK Overview Autolus Therapeutics plc received the 2026 Prix Galien UK Award for Best Biotechnology Product, recognizing its pioneering work in next-generation T-cell therapies developed with University College London. Concurr... -
Editas Medicine Unveils Positive Preclinical Data for Gene Editing Candidate EDIT-401, Demonstrating Significant Reduction in LDL, Lp(a), and ApoB in Non-Human Primates
Investing.com USA Overview Editas Medicine announced positive preclinical data for its gene editing therapeutic candidate EDIT-401, showing significant reductions in LDL cholesterol, lipoprotein(a) (Lp(a)), and apolipoprotein B (ApoB) in...
