Genome Editing– tag –
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ARPA-H Invests $125M in On-Demand RNA Medicine Manufacturing; ArsenalBio Pivots to In Vivo CAR T Cell Therapies for Solid Tumors
The Pharma Letter USA Overview ARPA-H has committed $125 million to five teams developing on-demand manufacturing capabilities for RNA medicines, a strategic investment to enhance rapid pharmaceutical response to future public health cri... -
Broad Institute Achieves Long-Term Brain Organoid Longevity and Streamlines Prime Editing with AI
Broad Institute USA Overview The Broad Institute announced a significant milestone on August 20, 2026, where an AI model streamlined the prime editing process and enabled the long-term longevity of brain organoids. The institute actively... -
Precision BioSciences Initiates Phase 1/2 FUNCTION-DMD Clinical Trial for PBGENE-DMD Gene Therapy: Applicable to 60% of DMD Patients, Initial Safety Data Expected by Year-End
Precision BioSciences, Inc. (Press Release) USA Overview Precision BioSciences announced the initiation of patient dosing in its Phase 1/2 FUNCTION-DMD clinical trial for PBGENE-DMD, an in vivo gene editing therapy for Duchenne muscular ... -
Mayo Clinic Leads ARPA-H Initiative to Advance Gene-Editing Therapies for Rare Immune Diseases, Integrating CRISPR and Novel Delivery for Over 500 Inborn Errors of Immunity
Mayo Clinic News Network USA Overview Mayo Clinic is participating in a 5-year collaborative research initiative, funded by an ARPA-H grant, to accelerate the development of gene-editing therapies for rare immune diseases. This initiativ... -
CRISPR Therapeutics Diversifies Pipeline into In Vivo Gene Editing and Off-the-Shelf CAR-T Cell Therapies, Leveraging Casgevy Commercialization Experience
Venture Atlas USA Overview CRISPR Therapeutics is strategically diversifying its pipeline, leveraging its commercialization experience with Casgevy, into in vivo gene editing programs (CTX310, CTX320, CTX340, CTX460) and allogeneic off-t... -
AI Accelerates Gene Editing Tool Development: Profluent Unveils OpenCRISPR-1, First AI-Designed Open-Source CRISPR, Heralding a New Era of Genomic Medicine
Facebook USA Overview Artificial intelligence is dramatically accelerating the development of gene editing tools. Profluent announced OpenCRISPR-1, the first AI-designed open-source gene editor, merging CRISPR technology with large langu... -
Comprehensive Review of CRISPR Gene Therapy: Potential Applications and Challenges for Sickle Cell Disease and Beta-Thalassemia
International Journal of Basic & Clinical Pharmacology Global Overview A comprehensive review covered the discovery, mechanism, therapeutic applications, and current limitations of CRISPR-Cas9, a revolutionary genome editing technology t... -
Sickle Cell Gene Therapy Breakthrough: Broad Institute’s Base Editing Permanently Resolves Disease in 42 Patients; FDA Approval & Stanford Trial Expansion Underway
Facebook (Stanford Medicine) USA Overview In a significant advance for sickle cell disease (SCD) gene therapy, Broad Institute's base editing therapy has permanently resolved SCD in 42 patients, with 24-month follow-up data reported. Con... -
CRISPR/Cas9 Evolves into Multi-Tool: Expanding Applications from Leukemia Screening to CAR-T Cell Engineering and Epigenome Editing
NHSJS USA Overview CRISPR/Cas9 technology has significantly evolved, broadening its applications from screening leukemia mutations to enhancing CAR-T cells' cancer recognition and precisely controlling gene expression through epigenome e... -
CRISPR Frontiers 2026: Innovations in DSB-Independent Editing and Delivery Systems Boost Genome Editing Precision and Stability
Addgene Blog USA Overview The 'Genome Engineering: CRISPR Frontiers 2026' conference reported significant evolution in CRISPR technology towards precise, double-strand break (DSB)-independent editing. Researchers are focusing on enhancin...
