Genome Editing– tag –
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Overcoming Commercialization Hurdles for iPSC Therapeutics: Regulatory Compliance and Accessible Manufacturing Strategies
Technology Networks Unknown Overview Commercializing iPSC-based therapeutics faces significant challenges in regulatory compliance and ensuring patient accessibility, as many therapies originate from academic settings without large-scale... -
CRISPR Therapeutics Reports Deep, Durable Lipid Lowering from In Vivo Gene Editor CTX310 in Phase 1a Trial
MarketScreener (CRISPR Therapeutics) Switzerland Overview CRISPR Therapeutics AG announced compelling one-year durability data from its Phase 1a clinical trial of CTX310, an in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3. A s... -
TScan Therapeutics Pivots to In Vivo TCR-T for Solid Tumors, Cuts 75% of Staff
BioPharm International USA Overview TScan Therapeutics announced a strategic pivot toward in vivo gene-edited TCR-T cell therapy for solid tumors, accompanied by a reduction of approximately 75% of its workforce. The company will halt it... -
REPROCELL Accelerates Clinical Production with FDA/EMA/PMDA-Compliant iPSC Manufacturing Platform
REPROCELL Japan Overview REPROCELL has unveiled a robust iPSC manufacturing scale-up strategy aimed at accelerating clinical development and commercialization of iPSC-based therapeutics. Their StemRNA™ Clinical iPSC Platform offers clini... -
Precision BioSciences Initiates Clinical Trial for ARCUS Nuclease Gene Editing Therapy PBGENE-DMD in DMD
CRISPR Medicine News USA Overview Precision BioSciences has dosed the first patient in its Phase 1/2 clinical trial for PBGENE-DMD, a gene editing therapy for Duchenne muscular dystrophy (DMD). This therapy utilizes two ARCUS nucleases t... -
Hereditary Transthyretin Amyloidosis Market: Intellia/Regeneron’s CRISPR Gene Editor Nexiguran ziclumeran Receives FDA RMAT Designation
GlobeNewswire USA Overview This article summarizes a market research report by DelveInsight on the hereditary transthyretin amyloidosis (hATTR) market. The report highlights Nexiguran ziclumeran (nex-z), a CRISPR-Cas9-based gene-editing ... -
CRISPR-Cas9 Breakthrough: CTX310 Delivers Sustained Lipid Control for Dyslipidemia in Phase 1a Trial, Published in NEJM
The New England Journal of Medicine USA Overview CRISPR Therapeutics' CTX310, a novel CRISPR-Cas9 gene-editing therapy, has demonstrated sustained and profound reductions in key lipid markers—angiopoietin-like 3 (ANGPTL3), triglycerides,... -
FDA Expands CASGEVY Pediatric Indication: CRISPR Gene-Editing Therapy Evaluated for Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia in 5-11 Year Olds
HCA Healthcare Today USA Overview The FDA expanded approval for CASGEVY (exagamglogene autotemcel), a CRISPR-based gene-editing therapy for sickle cell disease and transfusion-dependent beta-thalassemia, to eligible patients aged 2 years... -
ESC Congress 2026 Reveals Breakthrough Gene Therapies for Cardiovascular Disease: AAV8 Reduces LDL-C by 80-90%, siRNA Kylo-11 Achieves 97% Lp(a) Reduction
HCPLive (YouTube) USA Overview Groundbreaking data on multiple gene and molecular therapies for cardiovascular disease were presented at ESC Congress 2026. An AAV8-mediated gene therapy for homozygous familial hypercholesterolemia (HoFH)... -
University of Toronto Develops Next-Gen RNA Therapy Using Engineered tRNA to Bypass Premature Stop Codons, Offering Hope for Thousands of Untreatable Genetic Diseases
University of Toronto Canada Overview Researchers at the University of Toronto have developed a groundbreaking next-generation RNA therapeutic approach that enables cells to 'read through' disease-causing premature termination codons (PT...
