CRISPR Therapies, Base Editing and Approvals — 2026-09-08
Intellia Therapeutics' in-vivo CRISPR therapy for hereditary angioedema has officially entered FDA priority review, marking a potential milestone for the first approved in-body gene-editing treatment. Simultaneously, new data from Cleveland Clinic demonstrates the safety and efficacy of CRISPR-Cas9 in lowering cholesterol over a one-year period, while CRISPR Therapeutics advances its lipid-lowering candidate CTX310 with robust Phase 1a data.
CRISPR Therapies, Base Editing and Approvals — 2026-09-08
Top developments
Intellia’s Lonvo-z Enters FDA Priority Review for Hereditary Angioedema
On September 8, 2026, Intellia Therapeutics announced that the FDA has accepted its Biologics License Application (BLA) for lonvoguran ziclumeran (Lonvo-z) with Priority Review. This marks a critical step toward potentially becoming the first approved in-body gene-editing therapy. The application is supported by Phase 3 data showing an 87% reduction in monthly attacks compared to placebo, with 62% of patients remaining attack-free and off therapy for six months. A final FDA decision is anticipated by March 10, 2027.
Cleveland Clinic Trial Confirms Long-Term Safety of CRISPR Cholesterol Therapy
A first-in-human clinical trial at Cleveland Clinic reported that a one-time infusion of a CRISPR-Cas9 gene-editing therapy effectively and safely reduced LDL cholesterol and triglycerides in patients with medication-resistant lipid disorders. The results, published on September 4, 2026, show sustained benefits through one year of follow-up, addressing key concerns about the durability and safety of in-vivo editing for common chronic diseases like hyperlipidemia.
CRISPR Therapeutics Advances CTX310 with Strong Phase 1a Data
CRISPR Therapeutics presented updated Phase 1a data for its in-vivo gene-editing candidate CTX310 at the European Society of Cardiology (ESC) Congress 2026. The data demonstrated deep and durable editing of the ANGPTL3 gene, with a mean reduction of 79% (maximum 89%) in circulating ANGPTL3 levels, leading to significant triglyceride and LDL lowering. These results support the company's decision to pursue the highest dose level in subsequent trials.
FDA Proposes New Guidance to Streamline Genome Editing Approvals
The FDA has released a draft guidance titled "Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing." Although the initial press release was from April 2026, the document remains active and is listed among current Cellular & Gene Therapy guidances as of late August 2026. This regulatory move aims to standardize safety assessments and accelerate the development of genome editing therapies by allowing sponsors to utilize existing data from similar products.
Local view
No recent local-language media coverage specific to these September 2026 developments was identified in the available sources.
Context & numbers
The landscape for gene therapy pricing remains anchored by Casgevy (Vertex/CRISPR Therapeutics), which carries a list price of $2.2 million, and Lyfgenia (Bluebird Bio) at $3.1 million. The CMS Cell and Gene Therapy Access Model, which launched in January 2026, continues to test outcomes-based agreements for these sickle cell disease treatments, aiming to improve Medicaid access and manage costs.
On the radar
- March 10, 2027: Expected FDA decision date for Intellia’s Lonvo-z BLA, which could establish the first regulatory precedent for in-vivo CRISPR therapies.
- Q4 2026: Lilly’s VERVE-102 is projected to enter Phase 2 trials, having treated 35 patients so far, adding competition to the in-vivo lipid-lowering space.
This content was collected, curated, and summarized entirely by AI — including how and what to gather. It may contain inaccuracies. Crew does not guarantee the accuracy of any information presented here. Always verify facts on your own before acting on them. Crew assumes no legal liability for any consequences arising from reliance on this content.
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