CRISPR Therapeutics

APublicFounded 2013🇨🇭Zug, Switzerland

CEO

Samarth Kulkarni

Data verified as of Jul 4, 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), and type 1 diabetes. With ~$2.44B in cash and a catalyst-rich 2026 pipeline, it pairs a historic scientific position with a still-nascent commercial ramp.

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)

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. 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

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, with analysts modeling roughly 3x growth in 2026.

2026

Advance in vivo cardiovascular programs

Report further CTX310 (ANGPTL3) Phase 1 data in H2 2026 and progress the Lp(a)-lowering programs CTX320 and next-generation CTX321 — establishing one-time in vivo gene editing for cardiovascular disease.

H2 2026

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.

H2 2026

Move new in vivo programs into the clinic

Bring next-wave in vivo editing candidates into human trials — CTX340 for refractory hypertension and CTX460 for alpha-1 antitrypsin deficiency — broadening the in vivo pipeline beyond cardiovascular targets.

2026

Recent News

View all →

Jul 5

FDA expands Casgevy gene therapy to children as young as 2

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.

Vertex Pharmaceuticals

Operations & Revenue

StatusCommercial (via partner), pre-profit

CRISPR's first product, Casgevy, is approved and commercial across 9+ 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 passed 500 in Q1 2026 and the treatment-center network exceeds 75 in the US. A ~$2.44B cash position 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

Minimal GAAP revenue (~$3.5M FY2025 grants); Casgevy sales booked by Vertex (~$116M global in 2025), with CRISPR's ~40% economics flowing through the collaboration line

Cash & Investments

~$2.44B (Mar 31, 2026)

Market Cap

~$5.8B

Casgevy List Price

$2.2M per one-time treatment

Patients Started on Casgevy

>500 cumulative (Q1 2026)

Authorized Treatment Centers

>75 in the US

Approved Jurisdictions

9+ (US, UK, EU, Canada, Switzerland, Gulf states)

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.

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

Round	Date	Amount	Investors
Series A	2014	$25M	Versant Ventures
Series A top-up / Series B	2015	$64M	SR One, Celgene, NEA, Abingworth
IPO (NASDAQ: CRSP)	2016	$56M (+$35M Bayer placement)	Public markets; Bayer
Follow-on offering	2020	~$450M	Public markets (6.43M shares at $70)
Convertible senior notes (due 2031)	2026	$600M	Debt investors (1.73% coupon)