Intellia Therapeutics– tag –
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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,... -
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... -
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... -
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... -
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... -
Intellia Therapeutics Advances CRISPR Gene Editing: Lonvo-z Achieves Phase 3 Success for HAE, Targeting Mid-2027 U.S. Launch; Nex-z Phase 3 Enrollment Resumes for ATTR
Intellia Therapeutics, Inc. (Press Release) USA Overview Intellia Therapeutics reported positive Phase 3 results for lonvo-z in hereditary angioedema (HAE), with a Biologics License Application (BLA) submission to the FDA planned for H2 ... -
Intellia Therapeutics Reports Positive Phase 3 HAELO Data for lonvo-z in HAE and Resumes Nex-z ATTR Phase 3 Enrollment
Intellia Therapeutics USA Overview Intellia Therapeutics announced positive Phase 3 HAELO clinical data for lonvo-z in hereditary angioedema (HAE), published in the New England Journal of Medicine. The company anticipates FDA Biologics L... -
CRISPR Gene Editing Therapies Limited to Ex Vivo Approvals for Sickle Cell and Beta-Thalassemia in 2026; In Vivo Candidates Show 87% Attack Reduction and 62% Cholesterol Decrease
DeepDNA USA Overview As of August 2026, the only globally approved CRISPR gene-editing therapies are ex vivo treatments for sickle cell disease and transfusion-dependent beta-thalassemia. However, in vivo therapies are demonstrating sign...
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