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CRISPR Therapeutics

PublicFounded 2013🇨🇭Zug, Switzerland
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CEO

Samarth Kulkarni

Data verified as of Aug 30, 2026

Summary

CRISPR Therapeutics is a gene-editing pioneer co-founded in 2013 by Nobel laureate Emmanuelle Charpentier - co-inventor of CRISPR/Cas9 - alongside Rodger Novak and Shaun Foy. Its flagship, Casgevy (exagamglogene autotemcel), is the world's first approved CRISPR/Cas9 gene-editing medicine: a one-time therapy for sickle cell disease and transfusion-dependent beta thalassemia, developed and commercialized with Vertex Pharmaceuticals (which books the sales; CRISPR keeps ~40% of the economics). Casgevy is now approved across 9+ jurisdictions, priced at ~$2.2M per treatment, and in July 2026 the FDA expanded its US label to children as young as two. Commercial uptake has been slower than hoped - constrained by cell-collection bottlenecks - but cumulative patient starts have surpassed 500. Beyond Casgevy, CRISPR is pivoting toward in vivo liver gene editing (cardiovascular programs CTX310 and CTX320), allogeneic CAR-T for autoimmune disease and cancer (zugo-cel/CTX112, now also being tested with Lilly's pirtobrutinib in aggressive B-cell lymphomas), and type 1 diabetes, where a next-generation deviceless beta-cell replacement candidate, CTX213, is progressing toward the clinic on the strength of durable C-peptide data from its predecessor CTX211. It has also expanded beyond gene editing into RNA interference through a 2025 collaboration with Sirius Therapeutics on CTX611 (SRSD107), a long-acting Factor XI siRNA for thromboembolic disorders now in Phase 2, with topline data due in H2 2026. In its Q2 2026 update (August 3, 2026) it reported $2,364.4M in cash and investments, a narrowed net loss of $91.2M, and the start of Phase 1 trials for two next-wave in vivo candidates, CTX340 (refractory hypertension) and CTX460 (alpha-1 antitrypsin deficiency, the first program from its new SyNTase editing platform), pairing a historic scientific position with a still-nascent commercial ramp.

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Main Products

Casgevy

Active

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.

Approved across 9+ jurisdictions 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 and >75 US treatment centers - though gated by cell-collection and manufacturing timelines.

First ApprovalNov 2023 (UK); Dec 2023 (US, SCD)
IndicationsSickle cell disease; transfusion-dependent beta thalassemia (ages 2+)
Patients Started>500 cumulative (Q1 2026)

CTX310

In Development

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.

Phase 1. Positive data reported in November 2025 showed triglycerides down up to ~84% and LDL down up to ~87%, with durable ANGPTL3 editing; published in NEJM. A US IND has been cleared and a further update is expected in H2 2026.

TargetANGPTL3 (in vivo, liver)
Phase 1 EffectTriglycerides −84%, LDL −87% (max)

CTX611 (SRSD107)

In Development

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.

Phase 2 trial for prevention of venous thromboembolism in total knee arthroplasty patients dosed its first patient in September 2025. Phase 1 data showed peak FXI activity reductions >93% and aPTT increases >2x, sustained up to 6 months. Topline Phase 2 data expected in H2 2026; CRISPR will lead global Phase 3 development outside Greater China.

TargetFactor XI (FXI), long-acting siRNA
Phase 1 EffectFXI activity down >93%; aPTT up >2x (6-mo durability)

Zugo-cel (CTX112)

In Development

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.

Phase 1/2, with RMAT designation. Oncology data show a 90% overall response rate and 70% complete response; early autoimmune data show drug-free remissions in lupus. As of the Q2 2026 update, zugo-cel is running in three parallel Phase 1 autoimmune basket trials - rheumatology (lupus, systemic sclerosis, inflammatory myositis), hematology (immune thrombocytopenic purpura, warm autoimmune hemolytic anemia), and a newly initiated autoimmune-neurologic basket (progressive multiple sclerosis, NMOSD, MOGAD, NMDAR/LGI1 autoimmune encephalitis, and stiff person syndrome) - plus a combination study with Lilly's pirtobrutinib in aggressive B-cell lymphomas. Additional readouts expected in H2 2026, supported by a clinical-supply deal with Eli Lilly.

ModalityAllogeneic anti-CD19 CAR-T
Oncology Response90% ORR / 70% CR
Autoimmune Basket Trials3 active Phase 1 baskets: rheumatology, hematology, autoimmune-neurologic

CTX213

In Development

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.

Preclinical. Succeeds the earlier CTX211 (formerly VCTX211/VCTX210) program, whose Phase 1 data showed detectable C-peptide 12 months after implantation; that proof-of-concept informed CTX213's hypoimmune engineering. CTX213 has shown compelling preclinical efficacy via direct administration and is progressing toward the clinic, with no IND filed yet as of Q2 2026.

ModalityDeviceless, hypoimmune iPSC-derived beta-cell replacement
Predecessor ProgramCTX211 - C-peptide detectable 12 months post-implant (Phase 1)

What's Next

Scale the Casgevy commercial ramp

Convert the 500+ started patients into infusions and grow revenue as the authorized-treatment-center network expands and cell-collection bottlenecks ease. Casgevy revenue (booked by Vertex) reached $76M in Q2 2026, up 78% quarter-over-quarter and 151% year-over-year, tracking toward the roughly 3x full-year growth analysts have modeled for 2026.

2026

Advance in vivo cardiovascular programs

Having presented the late-breaking CTX310 (ANGPTL3) Phase 1a durability update at the European Society of Cardiology Congress on August 28, 2026 (15 participants, average follow-up of at least 60 days, no treatment-related serious adverse events or grade 3+ liver-transaminase changes) - simultaneously published as a peer-reviewed NEJM letter - CRISPR still owes a separate CTX310 Phase 1b clinical update in H2 2026 and continues progressing the Lp(a)-lowering programs CTX320 and next-generation CTX321 - establishing one-time in vivo gene editing for cardiovascular disease.

H2 2026 (Phase 1b, Lp(a) update)

Deliver zugo-cel (CTX112) autoimmune & oncology readouts

Report additional autoimmune and hematologic data for zugo-cel in H2 2026, advancing its off-the-shelf CAR-T toward registrational studies in lupus and B-cell cancers. The pipeline now spans three parallel Phase 1 autoimmune basket trials - rheumatology, hematology, and a newly initiated autoimmune-neurologic basket covering progressive MS, NMOSD, MOGAD, autoimmune encephalitis, and stiff person syndrome - plus a combination study of zugo-cel with Lilly's pirtobrutinib in aggressive B-cell lymphomas.

H2 2026

CTX611 (SRSD107) Phase 2 topline data

Report topline Phase 2 data for CTX611 (SRSD107), the long-acting Factor XI siRNA co-developed with Sirius Therapeutics, in patients undergoing total knee arthroplasty - the lead readout for CRISPR's new siRNA modality outside gene editing. CRISPR will lead global Phase 3 development (excluding Greater China) if the data support advancement. Reconfirmed on track for H2 2026 in the Q2 2026 business update.

H2 2026

Track Record

1 of 1 announced date hit

Move new in vivo programs into the clinic

Target 2026 · Resolved Aug 3, 2026

CRISPR Therapeutics confirmed in its Q2 2026 business update that it initiated Phase 1 trials for both next-wave in vivo editing candidates: CTX340 for refractory hypertension and CTX460 (the first candidate from its new SyNTase editing platform) for alpha-1 antitrypsin deficiency.

Hit

Operations & Revenue

StatusCommercial (via partner), pre-profit

CRISPR's first product, Casgevy, is approved and commercial across 10 jurisdictions, but sales are recognized by partner Vertex, with CRISPR taking ~40% of net profits and costs. The company remains loss-making (FY2025 net loss ~$582M) as it funds a broad pipeline. Casgevy's commercial ramp has been gated by lengthy cell-collection and manufacturing timelines, though cumulative patient starts have surpassed 500 globally and the treatment-center network exceeds 75 in the US. A $2,364.4M cash position (Jun 30, 2026) funds the pivot toward in vivo gene editing and cell therapy.

Revenue Streams

Casgevy (Vertex collaboration)

Vertex books all Casgevy product revenue (~$116M globally in 2025); CRISPR is entitled to ~40% of the program's net profits and shares ~40% of its costs, recognized through the net collaboration line rather than as product revenue.

Milestone & upfront payments

Non-recurring cash from the Vertex partnership, including the $900M upfront paid in 2021 and a $200M payment triggered at Casgevy's first approval.

Grant revenue

A small stream of research grant revenue (roughly $1–4M per year) recognized as GAAP revenue.

Key Metrics

Employees

~370

Est. Annual Revenue

Q2 2026 GAAP revenue $10.2M ($10M collaboration revenue plus grants), beating consensus; Casgevy sales booked by Vertex ($76M in Q2 2026, up 151% YoY), with CRISPR's ~40% economics flowing through the collaboration line. Vertex's Q3 2026 results (which would carry the next Casgevy sales figure) are not expected until early November 2026.

Cash & Investments

$2,364.4M (Jun 30, 2026)

Q2 2026 Net Loss

$91.2M (vs. $208.5M in Q2 2025)

Market Cap

~$5.74B (Aug 27, 2026 close; ~$59.37/share) - shares rallied into the Aug 28 ESC Congress CTX310 durability readout, up from ~$5.18B in mid-August

Casgevy List Price

$2.2M per one-time treatment

Patients Started on Casgevy

>500 cumulative globally (Q2 2026)

Authorized Treatment Centers

>75 in the US

Approved Jurisdictions

10 (US, Canada, UK, EU, Switzerland, Saudi Arabia, Bahrain, Qatar, UAE, Kuwait); >60,000 eligible patients (~37,000 in North America/Europe, >23,000 Middle East)

FY2025 Net Loss

$582M (R&D $285M)

Timeline

2026$600M convertible notes strengthen balance sheet

Raises ~$585M net through $600M of convertible senior notes due 2031, lifting cash and investments to ~$2.44B and extending its runway across a catalyst-heavy pipeline.

2026FDA expands Casgevy to children ages 2+

In July 2026 the FDA expands Casgevy's US label to patients as young as two years old with sickle cell disease or transfusion-dependent beta thalassemia, adding roughly 5,500 more eligible US children.

2026Initiates CTX340 and CTX460 Phase 1 trials

Reports in its Q2 2026 business update (August 3, 2026) that it has 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, targeting alpha-1 antitrypsin deficiency - broadening the in vivo pipeline beyond the cardiovascular program CTX310.

2026Zugo-cel plus pirtobrutinib combination study with Lilly

Initiates a combination study evaluating zugo-cel (CTX112) alongside Lilly's pirtobrutinib in aggressive B-cell lymphomas under the companies' clinical-supply collaboration, broadening zugo-cel's oncology development beyond monotherapy ahead of H2 2026 readouts.

2026Diabetes pipeline transitions to next-gen CTX213

Reports that durable Phase 1 data from CTX211 (detectable C-peptide 12 months post-implantation) informed a transition to CTX213, a next-generation deviceless beta-cell replacement candidate made of unencapsulated, edited iPSC-derived precursor islet cells, which has shown compelling preclinical efficacy and is progressing toward the clinic.

2026Zugo-cel expands to a third autoimmune basket trial

Initiates a third Phase 1 basket trial for zugo-cel (CTX112) in autoimmune-neurologic disease - covering progressive multiple sclerosis, NMOSD, MOGAD, NMDAR/LGI1 autoimmune encephalitis, and stiff person syndrome - alongside the ongoing rheumatology basket (lupus, systemic sclerosis, inflammatory myositis) and hematology basket (ITP, warm autoimmune hemolytic anemia), broadening the off-the-shelf CAR-T program well beyond its original oncology and lupus scope.

2026CTX310 durability data presented at ESC Congress

Presents a late-breaking Phase 1a durability update for CTX310 (ANGPTL3 editing) at the European Society of Cardiology Congress in Munich on August 28, 2026. Across 15 participants with average follow-up of at least 60 days, the update reported no treatment-related serious adverse events and no grade 3-or-higher liver-transaminase changes, supporting the therapy's long-term safety profile as the cardiovascular program advances toward a separate Phase 1b update in H2 2026.

2025Sirius Therapeutics siRNA collaboration (CTX611/SRSD107)

Announces a multi-target collaboration with Sirius Therapeutics (May 19, 2025) to co-develop and co-commercialize SRSD107 (CTX611), a long-acting Factor XI-targeting siRNA for thromboembolic disorders, plus an option on up to two further siRNA targets. CRISPR pays $25M cash and $70M in equity upfront, the companies split costs and profits 50/50, and CRISPR leads U.S. commercialization while Sirius leads Greater China. CRISPR also owes Sirius up to $300M in per-target milestones plus a shared $87.5M milestone pool and tiered royalties. Phase 1 data showed peak FXI activity reductions of >93%, sustained up to 6 months.

2025First patient dosed in CTX611 (SRSD107) Phase 2 trial

CRISPR Therapeutics and Sirius Therapeutics announce (September 22, 2025) that the first patient has been dosed in a European Phase 2 trial of SRSD107 (CTX611) for prevention of venous thromboembolism in patients undergoing total knee arthroplasty.

2025Positive in vivo cardiovascular data (CTX310)

Reports positive Phase 1 data for CTX310, an in vivo lipid-nanoparticle therapy that edits ANGPTL3 in the liver, showing deep and durable triglyceride and LDL lowering - validating CRISPR's move into one-time in vivo gene editing. Data presented at the AHA meeting and published in NEJM.

2025zugo-cel (CTX112) update and Lilly deal

Provides a broad update on zugo-cel (CTX112), its allogeneic CAR-T, with strong autoimmune and lymphoma data (90% overall response rate, 70% complete response in oncology) and a clinical-supply agreement with Eli Lilly.

2024US TDT approval and EU authorization

The FDA approves Casgevy for transfusion-dependent beta thalassemia in January 2024, and the EU grants conditional marketing authorization for both indications in February 2024, broadening global access.

2023Casgevy becomes world's first approved CRISPR therapy

The UK MHRA authorizes Casgevy in November 2023 - the first regulatory approval of any CRISPR/Cas9 gene-editing medicine - followed by US FDA approval for sickle cell disease in December 2023.

2021Amended Vertex deal ($900M upfront)

Restructures the Vertex collaboration: Vertex pays $900M upfront plus $200M at first approval, and the economics shift to a 60/40 split in Vertex's favor, with CRISPR retaining 40% of program profits and costs.

2019First patient dosed with exa-cel (CTX001)

Doses the first patient with CTX001 (later Casgevy) in transfusion-dependent beta thalassemia - one of the first company-sponsored CRISPR/Cas9 therapies to enter the clinic - with landmark results published later that year in the New England Journal of Medicine.

2016IPO on NASDAQ (CRSP)

Prices its initial public offering at $14.00 per share, raising ~$56M gross, alongside a $35M private placement from partner Bayer.

2015Vertex partnership for hemoglobinopathies

Signs a collaboration with Vertex Pharmaceuticals to develop CRISPR-based treatments for sickle cell disease and beta thalassemia - $75M upfront plus a $30M equity investment - the deal that would become Casgevy.

2014$25M Series A

Raises a $25M Series A led by Versant Ventures and formally unveils its founding team and CRISPR/Cas9 therapeutic platform.

2013Founded by CRISPR co-inventor Emmanuelle Charpentier

Incorporated in Switzerland by Emmanuelle Charpentier (co-inventor of CRISPR/Cas9 and future 2020 Nobel laureate), first CEO Rodger Novak, and Shaun Foy, incubated by Versant Ventures to translate genome editing into medicines.

Funding

Cumulative disclosed raise · dated rounds

$500M$1B$1.5B20142017202020232026$1.5B raised
RoundDateAmountInvestorsSource
Series A2014$25MVersant Ventures
Series A top-up / Series B2015$64MSR One, Celgene, NEA, Abingworth
IPO (NASDAQ: CRSP)2016$56M (+$35M Bayer placement)Public markets; Bayer
Follow-on offering2020~$450MPublic markets (6.43M shares at $70)
Registered direct offering2024$280MEcoR1 Capital and SR One (co-leads), other institutional investors
Convertible senior notes (due 2031)2026$600MDebt investors (1.73% coupon)

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