CRISPR TherapeuticsIn Development
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.
Latest update Sep 15, 2026
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Phase 1/2, with RMAT designation. Oncology data show a 90% overall response rate and 70% complete response; early autoimmune data show drug-free remissions in lupus. As of the Q2 2026 update, zugo-cel is running in three parallel Phase 1 autoimmune basket trials - rheumatology (lupus, systemic sclerosis, inflammatory myositis), hematology (immune thrombocytopenic purpura, warm autoimmune hemolytic anemia), and a newly initiated autoimmune-neurologic basket (progressive multiple sclerosis, NMOSD, MOGAD, NMDAR/LGI1 autoimmune encephalitis, and stiff person syndrome) - plus a combination study with Lilly's pirtobrutinib in aggressive B-cell lymphomas. At Morgan Stanley on September 14, 2026, Kulkarni said additional autoimmune data by year-end will help decide whether one or more indications can advance toward pivotal development.
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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.
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.
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.
Wholly owned in vivo gene-editing therapy that uses lipid nanoparticles to deliver Cas9 mRNA and a guide RNA to the liver and reduce expression of Lp(a).
Wholly owned in vivo gene-editing therapy that uses lipid nanoparticles to deliver Cas9 mRNA and a guide RNA to the liver and reduce hepatic angiotensinogen (AGT), for refractory hypertension.
SyNTase editing candidate, the first from that platform, targeting the E342K mutation in SERPINA1 inside a proprietary lipid nanoparticle, for alpha-1 antitrypsin deficiency.
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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.
Oklo
Oklo's isotope business combines the Idaho Radiochemistry Laboratory, a planned pilot reactor in Texas, and a future multi-reactor foundry to supply critical medical, industrial, research, defense, and space isotopes.
TerraPower
Sodium-cooled fast reactor with integrated molten salt energy storage, enabling flexible output from 345 MWe to 500 MWe peak.