CRISPR TherapeuticsIn Development
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.
Latest update Sep 10, 2026
01
Phase 1a complete; Phase 1b underway in severe hypertriglyceridemia and refractory hypercholesterolemia (US trials initiated, ex-US ongoing). One-year Phase 1a follow-up presented at ESC Congress on August 28, 2026 and published in NEJM: at the highest dose, mean reductions were 79% ANGPTL3, 48% triglycerides, and 53% LDL, with no treatment-related serious adverse events across 15 participants. A separate Phase 1b clinical update remains due in H2 2026.
02
03
Announced next steps from CRISPR Therapeutics that mention CTX310.
H2 2026
TargetH2 2026 (Phase 1b, Lp(a) update)
Having presented one-year CTX310 (ANGPTL3) Phase 1a follow-up at the European Society of Cardiology Congress on August 28, 2026 (15 participants; highest-dose mean LDL -53% and triglycerides -48% through 12 months; no treatment-related serious adverse events) - simultaneously published in NEJM - CRISPR still owes a separate CTX310 Phase 1b clinical update by year-end 2026, with Kulkarni at Morgan Stanley on September 14 saying the immediate focus is severe hypertriglyceridemia (triglycerides plus biomarkers such as LDL, HbA1c and hepatic fat) to inform pivotal-trial discussions. The company also continues progressing the Lp(a)-lowering program CTX321.
2026
TargetMonday, November 9, 2026, 8:00 a.m. CST (AHA LBS.05); embargoed until then
04
CRISPR Therapeutics presented a late-breaking Phase 1a durability update for CTX310, its in vivo gene-editing therapy targeting ANGPTL3, at the European Society of Cardiology Congress in Munich on August 28. Across 15 participants with average follow-up of at least 60 days, the company reported no treatment-related serious adverse events and no grade 3-or-higher liver-transaminase changes, with a separate Phase 1b clinical update still due in the second half of 2026.
CRISPR Therapeutics reported second-quarter 2026 results on August 3, ending the quarter with $2,364.4 million in cash and investments and narrowing its net loss to $91.2 million from $208.5 million a year earlier. The company said it initiated Phase 1 trials for CTX340, an in vivo editing candidate for refractory hypertension, and CTX460, the first candidate from its new SyNTase editing platform for alpha-1 antitrypsin deficiency, broadening its in vivo pipeline beyond the cardiovascular program CTX310.
05
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.
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.
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.
06
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.