Genome Editing– tag –
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Next-Generation CRISPR Tools Revolutionize Precise Gene Editing, Bypassing Double-Strand Breaks for Enhanced Therapeutic Safety and Consistency
News-Medical.net USA Overview Next-generation CRISPR technologies are fundamentally transforming cell and gene therapy by enabling precise gene editing without relying on double-strand DNA breaks (DSBs). Advances in base editing, prime e... -
REPROCELL Pioneers Integrated iPSC Manufacturing Platform, Accelerating US Phase III Clinical Programs with AI-Enhanced Gene Editing and GMP Compliance
REPROCELL Japan Overview REPROCELL has launched a comprehensive iPSC manufacturing platform designed to accelerate the clinical development of iPSC-derived therapeutics, notably supporting a US Phase III program. The platform integrates ... -
2026 ‘In Vivo CRISPR Medicine’ Symposium to Focus on Commercialization and Scalable Deployment of In Vivo Genome Editing
CRISPR Medicine News (CMN x CSGCT Virtual Symposium) USA Overview The upcoming 'In Vivo CRISPR Medicine: From Scientific Promise to Patients at Scale' virtual symposium on September 3, 2026, will highlight the transition of in vivo genom... -
FDA Lifts Clinical Hold on Intellia Therapeutics’ Phase 3 Trial for nex-z in Transthyretin Amyloidosis
Intellia Therapeutics (via LinkedIn) USA Overview Intellia Therapeutics announced the FDA has lifted the clinical hold on its Phase 3 MAGNITUDE-2 clinical trial for nexiguran ziclumeran (nex-z), a therapeutic candidate for transthyretin ... -
Intellia Therapeutics’ Gene-Editing Therapy lonvo-z Achieves 87% Reduction in HAE Attacks and 62% Attack-Free Rate in Phase 3 Trial
The Motley Fool USA Overview Intellia Therapeutics announced its in vivo gene-editing therapy, lonvoguran ziclumeran (lonvo-z), successfully met all primary and secondary endpoints in the Phase 3 HAELO trial for hereditary angioedema (HA... -
Vertex Pharmaceuticals’ CASGEVY Receives FDA Label Expansion for Over 5,500 US Children with Sickle Cell and Beta-Thalassemia, Driving Soaring Sales
ScanX USA Overview Vertex Pharmaceuticals' gene therapy, CASGEVY (exagamglogene autotemcel), received FDA label expansion for patients aged 2 and older with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). This... -
Vertex Pharmaceuticals Initiates Phase 3 Trial for iPSC Islet Cell Therapy VX-880 for Type 1 Diabetes, Showing High Rates of Insulin Independence
Facebook (Breakthrough T1D) USA Overview Vertex Pharmaceuticals has launched a Phase 3 clinical trial for zimislecel (VX-880), its stem cell-derived islet cell therapy for severe type 1 diabetes. Prior trials demonstrated remarkable effi... -
Editas Medicine Stock Soars 14.4% on Q2 Revenue Beat, Investor Focus on Gene-Editing Pipeline Intensifies
MarketBeat USA Overview Editas Medicine reported a Q2 2026 revenue of $11.89 million, significantly surpassing analyst estimates of $2.85 million, alongside an adjusted EPS loss of $0.15, better than the projected $0.27 loss, leading to ... -
PTC Therapeutics and Eli Lilly to Acquire Sangamo Assets for Up to $264M in Bankruptcy Auction, Bolstering Rare Disease Pipelines
Endpoints News USA Overview PTC Therapeutics and Eli Lilly are set to acquire parts of the bankrupt gene editing biotech Sangamo for up to $264 million following an auction. PTC Therapeutics will acquire Sangamo's Fabry disease candidate... -
Intellia Therapeutics Advances CRISPR Gene Therapy Pipeline, Including NTLA-2002 for HAE
Tracxn USA Overview Intellia Therapeutics is a public company advancing therapeutics based on CRISPR/Cas9 technology, with its lead candidate, NTLA-2002, an investigational CRISPR therapy for hereditary angioedema (HAE). The company also...
