CRISPR TherapeuticsIn Development
A long-acting, double-stranded siRNA that targets Factor XI (FXI) messenger RNA to inhibit FXI protein expression, aiming to provide anticoagulant and antithrombotic protection with less bleeding risk than existing anticoagulants, potentially dosed as infrequently as semi-annually. Co-developed with Sirius Therapeutics (50/50 cost and profit share; CRISPR leads U.S. commercialization, Sirius leads Greater China) across a range of thromboembolic indications including atrial fibrillation, venous thromboembolism, and stroke prevention.
Latest update Sep 15, 2026
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Phase 2 trial for prevention of venous thromboembolism in total knee arthroplasty patients dosed its first patient in September 2025, comparing CTX611 with enoxaparin. Phase 1 data showed peak FXI activity reductions >93% and aPTT increases >2x, sustained up to 6 months. Topline Phase 2 data expected in H2 2026, including high-level VTE-rate comparisons per the September 14, 2026 Morgan Stanley comments; CRISPR will lead global Phase 3 development outside Greater China.
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Announced next steps from CRISPR Therapeutics that mention CTX611 (SRSD107).
H2 2026
Report topline Phase 2 data for CTX611 (SRSD107), the long-acting Factor XI siRNA co-developed with Sirius Therapeutics, in patients undergoing total knee arthroplasty versus enoxaparin. Kulkarni said at Morgan Stanley on September 14, 2026 that the year-end update should include high-level venous-thromboembolism rate comparisons, with more detailed bleeding information later, and that CRISPR will lead Phase 3 development and potential commercialization outside Greater China. No Phase 2 readout has been published as of September 15, 2026.
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The world's first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel). A one-time therapy that edits the BCL11A gene in a patient's own blood stem cells to reactivate fetal hemoglobin, treating sickle cell disease and transfusion-dependent beta thalassemia. Partnered with Vertex Pharmaceuticals.
An in vivo gene-editing therapy delivered by lipid nanoparticles that knocks out the ANGPTL3 gene in the liver to lower triglycerides and LDL cholesterol - a potential one-time treatment for severe dyslipidemia and cardiovascular risk, and the lead of CRISPR's in vivo pipeline.
An allogeneic, gene-edited anti-CD19 CAR-T cell therapy (zugocaptagene geleucel) being developed for autoimmune diseases such as lupus as well as relapsed/refractory B-cell cancers, using CRISPR editing to make an off-the-shelf product.
A next-generation, deviceless beta-cell replacement candidate for type 1 diabetes, made of unencapsulated precursor islet cells derived from gene-edited, hypoimmune induced pluripotent stem cells (iPSCs), designed for direct administration without an encapsulation device or chronic immunosuppression.
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Oklo
Oklo's fast-fission powerhouse product line is designed for build-own-operate deployment, targeting customers that want firm clean power in a smaller footprint than traditional large reactors.
Oklo
Oklo is developing an advanced fuel center to recycle used nuclear fuel and fabricate metal fuel for Aurora and other fast-reactor applications.
TerraPower
Sodium-cooled fast reactor with integrated molten salt energy storage, enabling flexible output from 345 MWe to 500 MWe peak.
TerraPower
Integrated thermal energy storage system using molten salt to decouple reactor output from grid delivery, enabling peak power output.