iPS Cells & Regenerative Medicine– category –
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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... -
FDA Grants RMAT Designation to Cellectis’ Allogeneic CD22 CAR-T Lasme-cel for Relapsed/Refractory B-ALL
Targeted Oncology USA Overview The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Cellectis' lasmecabtagene timgedleucel (lasme-cel; UCART22), an allogeneic CD22-targeting CAR-T cell therapy, for relapsed/re... -
Intellia’s Lonvo-z Achieves 87% Reduction in HAE Attack Rate and 62% Attack-Free Patients in Pivotal Phase 3 In Vivo CRISPR Trial
GlobeNewswire USA Overview Intellia Therapeutics has reported highly positive Phase 3 HAELO clinical trial results for lonvoguran ziclumeran (lonvo-z), an in vivo CRISPR gene editing therapy for hereditary angioedema (HAE). The therapy a... -
iPS Cells & Regenerative Medicine Weekly Report May 13, 2026
📄 Weekly Report June 13, 2026 (PDF) — Download Weekly Report June 13, 2026 (PDF) — DownloadDownload 🎙 Podcast June 13, 2026 (MP3) — Play & Download iPS_RegenerativeMedicineEnglishPodcast_20260613.mp3Download -
Non-Viral CRISPR/Cas9 HDR Platform Enables Stable Genetic Engineering of Solid Tumor Models, Overcoming Viral Vector Challenges
bioRxiv (Preprint) USA Overview This preprint reports research on a non-viral CRISPR/Cas9 Homology-Directed Repair (HDR) platform for stable genetic engineering of solid tumor models. This approach enables precise genome modification and...
