CRISPR Therapies, Base Editing and Approvals — 2026-09-03
Intellia Therapeutics' in-vivo CRISPR therapy for hereditary angioedema successfully completed its Phase 3 trial, marking a pivotal milestone for the first-ever approval of an in-body gene-editing medicine. Meanwhile, CRISPR Therapeutics presented mixed durability data for its lipid-lowering therapy at ESC 2026, and Scribe Therapeutics reported its Q2 financial results, highlighting continued investment in in-vivo genetic medicines.
CRISPR Therapies, Base Editing and Approvals — 2026-09-03
Top developments
Intellia’s In-Vivo CRISPR Therapy Completes Phase 3 Trial for HAE
On August 28, 2026, news emerged that Intellia Therapeutics' CRISPR-based treatment for hereditary angioedema (HAE) met its goals in the global Phase 3 HAELO clinical trial. This success positions the therapy as a candidate for the first-ever FDA approval of an in vivo gene-editing medicine. The trial demonstrated significant efficacy, with earlier data suggesting a mean 95% reduction in attack rates after a single infusion.
CRISPR Therapeutics Pursues Highest Dose for Lipid-Lowering Therapy
At the European Society of Cardiology (ESC) 2026 meeting on August 28, CRISPR Therapeutics reported early-stage data showing mixed durability results one year after patients were dosed with its gene-editing candidate for high cholesterol. Despite the mixed results, the company announced it will pursue the highest dose level in further development. This comes alongside reports that gene editing reduced cholesterol by about 50% in a small sample of patients over a year of follow-up.
Scribe Therapeutics Reports Q2 2026 Financials
Scribe Therapeutics, a clinical-stage biotech focused on in vivo genetic medicines using CRISPR-based technologies, reported its second-quarter 2026 financial results on September 2, 2026. The company posted a net loss of $6.5 million. These results reflect ongoing operational costs as the company advances its pipeline of purpose-built in vivo genetic medicines.
Local view
No recent local-language media data specifically addressing these global CRISPR developments was available in the past 7 days.
Context & numbers
The global market for In Vivo Gene Editing is projected to grow significantly, reaching US$ 37.75 billion by 2035 from US$ 2.46 billion in 2025, representing a CAGR of 34.09% from 2026 to 2035. This nearly 15x expansion is driven by improvements in CRISPR delivery technologies. Additionally, CRISPR Therapeutics reported a Q2 net loss of $90 million with a cash deposit of $291 million, while Cellectis reported a Q2 net loss of $22 million and Caribou Biosciences a net loss of $24 million, indicating the capital-intensive nature of the sector.
On the radar
- FDA Approval Review: Watch for the FDA's review timeline following Intellia's Phase 3 success, which could lead to the first approval of an in vivo CRISPR therapy.
- Regulatory Scrutiny: Continued debate on transparency and safety in investigator-initiated trials, particularly following recent adverse events reported in China, remains a key regulatory focus.
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