CRISPR TherapeuticsIn Development
SyNTase editing candidate, the first from that platform, targeting the E342K mutation in SERPINA1 inside a proprietary lipid nanoparticle, for alpha-1 antitrypsin deficiency.
Latest update Oct 20, 2026
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Phase 1. The Aug. 3, 2026 update said CTX460 is the first SyNTase candidate and that a Phase 1 trial in alpha-1 antitrypsin deficiency had been initiated. The pipeline page says it targets the E342K mutation in SERPINA1. No product image is deployed.
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Announced next steps from CRISPR Therapeutics that mention CTX460.
2027
At Morgan Stanley on September 14, 2026, Kulkarni said clinical data from CTX340 (in vivo AGT editing for refractory hypertension) and CTX460 (SyNTase editing for alpha-1 antitrypsin deficiency) are expected in 2027, after both programs entered Phase 1 in 2026. He said blood-pressure changes on CTX340 could potentially be observed within days after gene editing.
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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.
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 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.
06
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