CRISPR TherapeuticsActive
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.
Latest update Sep 15, 2026
01
Approved in 39 countries across North America, Europe, and the Middle East and priced at ~$2.2M per treatment. In July 2026 the FDA expanded the US label to patients ages 2+. Uptake is ramping - >500 cumulative patient starts, >100 initiations per quarter as of September 2026, and >75 US treatment centers - though the treatment journey can still take six to nine months. Vertex booked $76M of Casgevy sales in Q2 2026.
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03
Announced next steps from CRISPR Therapeutics that mention Casgevy.
2026
Convert the 500+ started patients into infusions and grow revenue as the authorized-treatment-center network expands. Casgevy is now described as approved in 39 countries. Revenue booked by Vertex reached $76M in Q2 2026, up 78% quarter-over-quarter and 151% year-over-year. At Morgan Stanley on September 14, 2026, Kulkarni said initiations now exceed 100 per quarter, that most early reimbursement, contracting and manufacturing bottlenecks have been addressed, and that the remaining 6-to-9-month treatment journey is driven more by exchange transfusions and scheduling than by manufacturing. The pediatric ages-2+ label is a tailwind. The next official sales figure is expected with Vertex's early-November Q3 2026 results.
04
The U.S. FDA on July 1 approved expanded use of Casgevy (exagamglogene autotemcel) - the world's first approved CRISPR/Cas9 gene-editing medicine, developed by Vertex Pharmaceuticals with CRISPR Therapeutics - for patients ages 2 and older with sickle cell disease or transfusion-dependent beta thalassemia, lowering the prior age floor of 12. Vertex said the expansion makes roughly 5,500 additional U.S. children newly eligible for the one-time therapy. CRISPR shares rose about 8% and Vertex about 6% on the news; CRISPR retains roughly 40% of Casgevy's economics under the partnership.
05
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.
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