The first CRISPR medicine, and what comes next
CRISPR Therapeutics exists to answer one question: can gene editing, the tool that won a Nobel Prize, become a repeatable medicine business rather than a single miracle drug. In December 2023 the company and its partner Vertex Pharmaceuticals answered half of that question. Casgevy became the first CRISPR/Cas9-edited therapy ever approved by a regulator, a one-time treatment that reprograms a patient's own stem cells to functionally cure sickle cell disease and, weeks later, transfusion-dependent beta thalassemia. It was the moment a decade of academic breakthroughs and biotech promises turned into an approved drug a patient could actually receive.
The harder half of the question is still being answered. Casgevy's real-world rollout has been slow, gated less by demand than by the sheer logistics of delivering a personalized, hospital-administered cell therapy at scale, and CRISPR Therapeutics splits its economics on that drug with Vertex rather than owning it outright. The company's next several years hinge on whether it can turn a single landmark approval into a broader, wholly-owned pipeline of in vivo editing therapies, injectable medicines with none of Casgevy's manufacturing overhead, spanning cardiovascular disease, autoimmune disease and cancer.
From a Nobel-winning discovery to a public biotech
CRISPR Therapeutics was incorporated in Zug, Switzerland in October 2013 (initially as Inception Genomics AG, renamed the following year) by Emmanuelle Charpentier, the microbiologist who co-invented the CRISPR/Cas9 gene-editing system and would go on to share the 2020 Nobel Prize in Chemistry for it, alongside Rodger Novak and Shaun Foy. The company built a dual base, corporate headquarters in Zug and research and commercial operations centered in Boston, betting that Charpentier's 2012 discovery could be turned into medicines faster than biotech's usual decade-plus timelines.
The turning point came in 2015, when CRISPR Therapeutics signed a strategic research collaboration with Vertex Pharmaceuticals to pursue CRISPR-based treatments for the genetic root causes of disease. That partnership produced exagamglogene autotemcel, later marketed as Casgevy, and the companies subsequently amended their agreement so Vertex leads global development, manufacturing and commercialization while the two split program costs and profits worldwide roughly 60/40 in Vertex's favor. CRISPR Therapeutics listed on Nasdaq in October 2016, years before it had an approved product, entirely on the strength of that platform bet.
Casgevy: editing a cure into a patient's own cells
Casgevy works by removing a patient's blood stem cells, editing them outside the body with CRISPR/Cas9 to switch off part of the BCL11A gene, and infusing the edited cells back after chemotherapy conditioning clears space in the bone marrow. Silencing BCL11A reactivates fetal hemoglobin, the oxygen-carrying protein the body makes before birth, which compensates for the defective adult hemoglobin that causes sickle cell disease and beta thalassemia. Unlike earlier gene therapies that add a working gene copy, Casgevy permanently rewrites the patient's own DNA, and it is designed to be given once.
The FDA approved Casgevy for sickle cell disease on December 8, 2023 and extended it to transfusion-dependent beta thalassemia in January 2024; regulators in the UK, European Union, Saudi Arabia and Bahrain, among others, have since authorized it as well, and in July 2026 the FDA expanded the US label down to patients as young as two. Priced at roughly $2.2 million per treatment, Casgevy is not just a drug but a proof of concept: the first time CRISPR gene editing has been shown, in an approved medicine, to durably change a person's biology.
A one-time cure meets an assembly-line problem
Casgevy's science was never the obstacle; its logistics have been. Administering the therapy requires a specialized treatment center to collect enough of a patient's blood stem cells, often across three or four collection sessions, ship them to a manufacturing site for editing, and then hospitalize the patient for weeks of chemotherapy conditioning and recovery, a process closer to a bone marrow transplant than a pill or infusion. More than two years after approval, only around 60 patients across the US, Europe and the Middle East had been treated by early 2026, a rollout STAT News and other outlets have characterized as bottlenecked by treatment-center capacity rather than by patient demand.
The trend line has since turned upward. Vertex reported roughly 300 patients began the treatment process in 2025, with about 150 completing their initial cell collection, and roughly 90% of eligible US patients now have reimbursed insurance access. Vertex and CRISPR Therapeutics have guided to Casgevy revenue nearly tripling in 2026 as more of that backlog converts into completed infusions, a real-world test of whether a curative but operationally heavy gene therapy can still scale into a meaningful commercial franchise.
Betting the next act on off-the-shelf editing
Casgevy's commercial friction is a large part of why CRISPR Therapeutics has spent the past several years building a pipeline the company owns outright and that does not require harvesting a patient's cells at all. Its furthest-along in vivo programs, CTX310 and CTX320, are injectable CRISPR therapies that edit genes directly inside the body to lower ANGPTL3 and lipoprotein(a) respectively, targeting inherited forms of high cholesterol, high triglycerides and cardiovascular risk that current drugs manage poorly; both are in Phase 1 trials, with CTX310 data set for presentation at the European Society of Cardiology Congress in Munich in late August 2026. Two newer in vivo candidates, CTX340 for refractory hypertension and CTX460 for alpha-1 antitrypsin deficiency (the first program built on the company's newer SyNTase editing platform), began Phase 1 dosing in 2026.
In parallel, CRISPR Therapeutics is pushing an allogeneic, off-the-shelf CAR-T cell therapy, zugo-cel (formerly CTX112), into both autoimmune disease and B-cell cancers, including a combination study with Eli Lilly's pirtobrutinib in aggressive lymphomas, and has moved outside gene editing entirely through a 2025 collaboration with Sirius Therapeutics on CTX611, an RNA-interference anticoagulant now in Phase 2. The common thread is diversification away from Casgevy's manufacturing model: every one of these programs is designed to be simpler to deliver and, unlike Casgevy, fully owned by CRISPR Therapeutics rather than split with a partner.
Financial runway and what to watch
CRISPR Therapeutics still operates as a clinical-stage biotech in most respects, funding a wide pipeline mostly on cash rather than product revenue: the company reported a net loss of $91.2 million in the second quarter of 2026, narrowed by more than half from a year earlier, against just $10.2 million of collaboration and grant revenue. It ended the quarter with $2.36 billion in cash and marketable securities, bolstered by a $600 million convertible senior notes offering priced in March 2026, giving it a multi-year runway to fund its cardiovascular, oncology and autoimmune programs without needing near-term Casgevy profits to carry the company.
What determines whether that runway turns into a second approved medicine: whether the Casgevy revenue ramp Vertex and CRISPR Therapeutics have guided to actually materializes in 2026 as treatment centers scale up capacity; whether CTX310 and CTX320's cardiovascular data, due through the second half of 2026, show the clean safety and durability profile in vivo editing needs to win regulators' and doctors' trust; and whether zugo-cel's Phase 1/2 results in B-cell malignancies, also expected in the second half of 2026, hold up well enough to justify a wholly-owned cell therapy franchise. Casgevy proved CRISPR Therapeutics could get one edited medicine to patients. The next 18 months determine whether it can do it again, on its own, at lower cost, and repeatedly.