What they sell
| Product | What it treats | How new is it? |
|---|---|---|
| Casgevy (exa-cel) First medicine made with CRISPR (approved 2023). Sold by Vertex; CRISPR gets 40% of the profit. Approved from age 2 in the US in 2026. | Sickle cell disease, beta thalassemia | First of its kind |
In development
| Drug | For | Stage |
|---|---|---|
| CTX310 One infusion switches off the ANGPTL3 gene in the liver. Effects lasted at least one year. Two FDA "RMAT" fast-track designations. | Very high triglycerides and LDL cholesterol | Phase 1 |
| CTX340 Switches off the AGT gene in the liver. | Hard-to-treat high blood pressure | Phase 1 |
| CTX460 First drug from its new SyNTase editing method, which corrects a gene instead of cutting it off. | Alpha-1 antitrypsin deficiency | Phase 1 |
| Zugo-cel (CTX112) Ready-made ("off-the-shelf") CAR-T cells from donors. | Lupus, systemic sclerosis and other autoimmune diseases; blood cancers | Phase 1 |
| CTX611 (SRSD107) An siRNA medicine (not gene editing), with Sirius Therapeutics. Phase 2 after knee surgery fully enrolled. | Preventing blood clots | Phase 2 |
Dates to watch
| When | What |
|---|---|
| 8 November 2026 | Zugo-cel results in systemic sclerosis at the ACR Convergence meeting. |
| 9 November 2026 | CTX310 Phase 1b results in very high triglycerides, late-breaking talk at the AHA heart meeting. |
| 2H 2026 | CTX611 Phase 2 update and zugo-cel results in blood cancers. |
| 2027 | Decision on licensing up to two more siRNA programs from Sirius. |
The short version
CRISPR Therapeutics was founded in 2013 to turn CRISPR, the gene-editing tool that won the 2020 Nobel Prize in Chemistry, into medicines. It succeeded once already: Casgevy, made with Vertex, became the first CRISPR medicine ever approved in 2023. The company is still loss-making, but it has $2.28 billion of cash (CRISPR Q3 2026 results) and a pipeline that is moving from rare blood diseases to common ones such as high cholesterol and high blood pressure.
What it makes
Casgevy is a one-time treatment: doctors take a patient’s blood stem cells, edit them so the body makes a healthy form of hemoglobin again, and put them back. It generated $99 million in Q3 2026 (+30% on the quarter), and more than 750 patients have started treatment. Vertex sells it and keeps 60% of the profit; CRISPR gets 40%.
The real bet is in vivo editing, inside the body with a single infusion. CTX310 switches off a gene (ANGPTL3) in the liver that raises blood fats. In results shown in August 2026, one treatment gave deep drops in triglycerides and LDL cholesterol that were still there after a year. CTX340 does the same for a gene that raises blood pressure. The company also tests ready-made CAR-T cells (zugo-cel) against autoimmune diseases such as lupus.
Is it really new?
Yes. Casgevy is the first medicine of its kind, and a “one and done” treatment for cholesterol or blood pressure would be a new way to treat the world’s most common diseases. The idea is not unique, though. Several companies are racing to the same goal, and existing pills and injections already lower cholesterol well and cheaply. CRISPR has to show that a permanent edit is safe for decades, not just effective.
The competition
The closest rival is Intellia Therapeutics, the other large CRISPR company, which is further ahead with editing inside the body but had a Phase 3 trial paused in 2025 after a patient died. Beam Therapeutics uses a newer, more precise method (base editing). In heart disease, Eli Lilly bought Verve Therapeutics in 2025 for its one-time cholesterol edits. And there are approved medicines that lower the same blood fats with regular injections, from Regeneron, Arrowhead and Ionis.
Money
CRISPR lost $111.4 million in Q3 2026 and $325.5 million in the first nine months. With $2.28 billion in the bank, that is several years of runway, so it does not urgently need to sell new shares, a common risk for small biotechs. Casgevy’s share of profit should grow as more hospitals start treating patients.
Risks
- Early-stage value. Most of the company’s value rests on Phase 1 trials, and most drugs at that stage never reach the market.
- Slow Casgevy launch. The treatment needs a hospital stay and chemotherapy to prepare the body, which limits how many patients can get it.
- Safety of permanent edits. A long-term problem anywhere in gene editing would hit the whole field.
- Small company, big swings. With a market cap around $5 billion, a single trial result can move the stock 20% or more in a day.
Our take
CRISPR Therapeutics is a pioneer with money in the bank and a first product, which few gene-editing companies can say. It is not a stable business yet. For investors it is a bet that one-time gene edits will treat common diseases. The heart-disease results at the AHA meeting on 9 November 2026 are the next important data point.
Main competitors
| Company | Rival product | How it compares |
|---|---|---|
| Intellia Therapeutics | In vivo CRISPR medicines (Phase 3) | The other big CRISPR company. Further ahead in editing inside the body, but a Phase 3 trial was paused in 2025 after a patient died of liver injury. |
| Beam Therapeutics | Base editing (BEAM-101 for sickle cell) | A newer, more precise editing method; a future rival to Casgevy. |
| Eli Lilly (Verve) | VERVE-102 gene editing for cholesterol | Lilly bought Verve in 2025; one-time cholesterol edits, a direct rival to CTX310. |
| Regeneron, Arrowhead, Ionis | Evkeeza, Redemplo, Tryngolza | Approved drugs that lower the same blood fats, given as regular injections rather than once. |
Frequently asked questions
What does CRISPR Therapeutics make?
It develops medicines based on CRISPR gene editing. Its first, Casgevy, treats sickle cell disease and beta thalassemia and is sold by its partner Vertex. Its other programs, for heart disease, autoimmune disease and cancer, are still in trials.
Is CRISPR Therapeutics profitable?
No. It lost $111.4 million in Q3 2026. It had $2.28 billion of cash on 30 September 2026, enough for several years at that pace.
What is in vivo gene editing?
Editing genes inside the body, with one infusion, instead of taking cells out, editing them in a lab and putting them back as Casgevy does. CTX310 and CTX340 work this way, which could make treatment much simpler and cheaper.
How does CRISPR Therapeutics share Casgevy with Vertex?
Vertex leads development and sales. The two companies split the costs and profits 60/40, with 60% to Vertex.