CRISPR TherapeuticsIn Development
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).
Latest update Oct 20, 2026
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Preclinical. The Aug. 3, 2026 update said CTX321 is progressing through IND/CTA-enabling studies and uses an optimized guide that delivered about two-fold greater potency in preclinical models, paired with the same lipid nanoparticle used previously. An Lp(a) program update is anticipated in 2026, with no date. No product image is deployed.
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Announced next steps from CRISPR Therapeutics that mention CTX321.
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.
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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.
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.
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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