Genome Editing– tag –
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Intellia Therapeutics’ In Vivo CRISPR Therapy Lonvo-z Achieves 87% Attack Rate Reduction in HAE Phase 3 Trial, Initiates Rolling BLA Filing with FDA
Intellectia.AI USA Overview Intellia Therapeutics' in vivo CRISPR therapy, lonvo-z, demonstrated an 87% reduction in hereditary angioedema (HAE) attack frequency in its Phase 3 HAELO trial. Following these breakthrough results, the compa... -
CRISPR Gene Editing Advances in 2026: Accumulation of Clinical Data and Evolving Regulatory Framework for Rare Diseases, Cancer, and Autoimmune Disorders
Industry report / Analysis USA Overview The CRISPR gene editing landscape in 2026 shows significant progress across rare diseases, hematologic and solid cancers, and autoimmune disorders. Clinical programs are generating crucial data, wh... -
Beam Therapeutics Receives U.S. FDA IND Clearance for BEAM-304, a Base Editing Therapy for Phenylketonuria (PKU)
Beam Therapeutics USA Overview Beam Therapeutics announced U.S. FDA Investigational New Drug (IND) clearance for BEAM-304, its investigational base editing therapy for Phenylketonuria (PKU). This milestone paves the way for human clinica... -
CRISPR Therapeutics Stock Volatility Driven by CASGEVY Commercialization and CTX112 Clinical Progress
Investing.com USA Overview CRISPR Therapeutics has experienced significant stock volatility due to market reactions to the commercialization of its approved CRISPR gene therapy, CASGEVY, and clinical developments for its off-the-shelf CA... -
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 ... -
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... -
Pharmaceutical and Biotech M&A Surges in 2026, Driven by Pipeline Reinforcement and Expansion into Emerging Therapeutic Areas
DistilINFO Publications USA Overview The pharmaceutical and biotechnology sectors are experiencing a significant surge in M&A activity in 2026, as major drugmakers actively seek to fortify their pipelines and expand into novel therap... -
Intellia Therapeutics Reports “Paradigm-Shifting” Phase 3 Data for Lonvoguran Ziclumeran, Achieving 87% Reduction in HAE Attacks with Single In Vivo CRISPR Dose
Fierce Biotech USA Overview Intellia Therapeutics announced groundbreaking Phase 3 HAELO trial data for lonvoguran ziclumeran (lonvo-z), an *in vivo* CRISPR gene-editing therapy for hereditary angioedema (HAE). This one-time treatment de... -
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 ...
