CRISPR Therapies, Base Editing and Approvals — 2026-09-04
CRISPR Therapeutics presented durable Phase 1a data for its in vivo ANGPTL3 editor CTX310 at the ESC Congress, signaling potential for one-time cholesterol treatments. Meanwhile, Beam Therapeutics dosed the first patient in the pivotal cohort of its base-editing trial for alpha-1 antitrypsin deficiency, advancing base editing toward commercialization.
CRISPR Therapies, Base Editing and Approvals — 2026-09-04
CRISPR Therapeutics Shows Durable Cholesterol Lowering with CTX310
On August 28, 2026, CRISPR Therapeutics presented Phase 1a durability data for CTX310, an investigational in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3, at the European Society of Cardiology (ESC) Congress. The data demonstrated deep and durable editing, resulting in significant lowering of triglycerides and LDL cholesterol. This milestone supports the viability of one-time gene-editing treatments for cardiovascular diseases, a major expansion beyond rare genetic disorders.
Beam Therapeutics Doses First Patient in Pivotal Base-Editing Trial
Beam Therapeutics announced the dosing of the first patient in the global pivotal cohort of its Phase 1/2 BEAM-302 trial. BEAM-302 is a liver-targeted base-editing therapy designed to correct the genetic cause of alpha-1 antitrypsin deficiency (AATD). This step marks a critical transition for base editing technology from early-stage exploration to late-stage clinical validation, potentially paving the way for FDA approval in the near future.
Scribe Therapeutics Reports Q2 2026 Financials and Pipeline Progress
Scribe Therapeutics, a clinical-stage company developing in vivo genetic medicines using CRISPR-based technologies, reported its second-quarter 2026 financial results on September 2, 2026. The company reported a net loss of $6.5 million ($4.08 per share). While financial results were modest, the update underscores ongoing investment in next-generation editing tools designed for in vivo delivery, complementing the broader industry shift toward non-ex-vivo therapies.
Local view
No recent local-language media coverage specifically focused on these developments was identified within the past 7 days.
Context & numbers
The global market for in vivo gene editing is projected to reach US$ 37.75 billion by 2035, growing from US$ 2.46 billion in 2025 at a CAGR of 34.09%. This expansion is driven by improvements in CRISPR delivery technologies and therapeutic potential.
On the radar
- Intellia Therapeutics Conference Call: Intellia previously announced a conference call for August 2026 regarding its pipeline; investors should monitor post-call updates for further details on its lead candidate, lonvo-z, following its historic Phase 3 readout earlier this year.
- FDA Approval Window: The closing stretch of Q3 2026 is a busy regulatory window with target action dates spanning gene therapy and oncology through September.
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