iPS Cells & Regenerative Medicine– category –
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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.... -
Cellares and Ori Biotech Lead Automated Cell Therapy Production Market with FDA Advanced Manufacturing Technology Designation
Fierce Pharma USA Overview A Tracxn report highlights Cellares and Ori Biotech as dominant leaders in automated cell therapy production, both holding FDA's Advanced Manufacturing Technology (AMT) designation. Their closed systems aim to ... -
Qihan Biotech’s Universal Dual-Target Hypoimmune CAR-T Therapy QT-019B Receives FDA RMAT and Breakthrough Therapy Designations
PackGene Biotech China Overview Qihan Biotech's universal dual-target CAR-T therapy, QT-019B, has received both FDA Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designations (BTD). This off-the-shelf allogeneic ... -
University of Houston Researchers Discover Salt Significantly Enhances Lipid Nanoparticle Delivery Efficiency in Gene Therapy
University of Houston USA Overview University of Houston researchers have discovered that adding salt to lipid nanoparticles (LNPs) dramatically improves the intracellular delivery efficiency of mRNA vaccines and gene therapeutics. This ... -
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... -
Austrian Cell Therapy Startup Innovecell Lists on Tokyo Stock Exchange Growth Market, Raises ¥11.7B for Global Aggregation Model Focused on Incontinence Therapy
Moomoo Japan Overview Innovecell, a cell therapy startup spun out from the Medical University of Innsbruck, specializing in autologous cell therapies for urinary incontinence, listed on the Tokyo Stock Exchange Growth Market in February ... -
FDA Unleashes Real-Time Clinical Trials: An AI-Driven Revolution for Faster Drug Development
ICON plc アイルランド Overview The U.S. FDA has launched its Real-time Clinical Trials (RTCT) initiative, a pivotal program designed to dramatically accelerate drug development. By enabling near real-time data sharing with regulators and... -
Real-World Data for Follicular Lymphoma CAR T-Cell Therapy Maintains Trial-Level Response Rates but Shows Shorter Progression-Free Survival
The Limbic Australia Overview A real-world analysis of commercial CAR T-cell therapies (axi-cel and tisa-cel) for relapsed/refractory follicular lymphoma (FL) demonstrated comparable response rates to those observed in clinical trials, d... -
PwC Report: Biopharma Ecosystem Fully Recovers with 16 M&A Deals Over $1B in Q1 2026, Driven by Next-Gen Modalities and GLP-1 Expansion
Fierce Pharma USA Overview A recent PwC report indicates the biopharma ecosystem has 'returned to full health,' with 16 M&A transactions exceeding $1 billion in Q1 2026. Deals primarily focused on 'differentiated science, GLP-1 expan...
