CRISPR Therapeutics

What CRISPR Therapeutics does

CRISPR Therapeutics AG is a Swiss-headquartered, NASDAQ-listed (CRSP) clinical-stage-to-commercial biotechnology company founded in 2013 that develops transformative gene-based medicines using its proprietary CRISPR/Cas9 platform. Its foundational product CASGEVY (exagamglogene autotemcel) — co-developed and co-commercialized with Vertex Pharmaceuticals under a 60/40 U.S. profit-share arrangement — is the first CRISPR/Cas9 gene-edited therapy approved by the FDA (December 2023 for sickle cell disease, January 2024 for transfusion-dependent beta thalassemia) and by the European Commission (2024). The product is an autologous ex vivo CRISPR-edited hematopoietic stem cell therapy priced at $2.2 million per patient, distributed through 75+ authorized treatment centers globally via Vertex's specialty pharmacy and sales force.

The company's technology platform spans CRISPR/Cas9 gene editing, an allogeneic CAR-T chassis with multiplexed edits (CTX112 and related programs), the SyNTase gene correction platform (CTX460), in vivo delivery via lipid nanoparticles and AAV vectors (CTX310, CTX320), CRISPR-X next-generation editing modalities, and ex vivo hematopoietic stem cell editing. Pipeline breadth extends to 5 clinical-stage and 10 preclinical-stage programs covering hemoglobinopathies, immuno-oncology, autoimmune diseases (including CTX112/zugo-cel in lupus, scleroderma, and myositis), in vivo cardiovascular (Lp(a) and ANGPTL3 targets), and regenerative medicine (Type 1 diabetes via ViaCyte partnership).

The business model combines CASGEVY product revenue on a 60/40 U.S. profit-share with Vertex (FY2025 revenue $116 million, Q1 2026 $43 million), supplemented by collaboration revenue from milestone/royalty arrangements with partners including Vertex, Sirius Therapeutics, Nkarta, Capsida, and Bayer (Casebia Therapeutics). Customer concentration is high — effectively a single commercial product and a single dominant commercialization partner — though patient end-customer reach is broadening as authorized treatment centers expand across the U.S. and Europe. The company reported a $581.6 million net loss in FY2025 against a $2.44 billion cash position (post-March 2026 convertible raise) that funds ongoing pipeline development.

Name

CRISPR Therapeutics

Legal name

CRISPR Therapeutics AG

Website

https://crisprtx.com

Company type

Public

Founded year

2013

Operating status

Operating

Headcount range

251–500 employees

Short description

CRISPR Therapeutics AG is a Swiss-headquartered, NASDAQ-listed biotech that develops CRISPR/Cas9 gene-editing therapies, including CASGEVY — the first FDA-approved CRISPR-based therapy for sickle cell disease and beta thalassemia, co-commercialized with Vertex Pharmaceuticals across 75+ treatment centers globally.

Ownership category

akta.pro rank

Product category

Gene-Editing Therapeutics

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714), Pharmaceutical Preparation Manufacturing (325412)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN), Neurology/CNS Gene Therapies (HLAAACAH)

Keywords

• Gene editing therapies
• Cell therapy platform
• CRISPR-based medicines
• Ex vivo gene editing
• Genomic therapeutics

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure

Revenue model

1. CASGEVY Product Sales: CRISPR Therapeutics co-commercializes CASGEVY with Vertex Pharmaceuticals in the U.S. on a 60/40 profit-share basis. Vertex is the manufacturer and exclusive license holder. Revenue from product sales of the CRISPR-based gene editing therapy for sickle cell disease and beta thalassemia.
2. Collaboration Revenue: Revenue from strategic collaborations including milestone payments, royalty payments, and cost-sharing arrangements with partners such as Vertex Pharmaceuticals, Sirius Therapeutics, and others for co-development programs.
3. Grant Revenue: Revenue from research grants, including a grant from Friedreich's Ataxia Research Alliance to advance in vivo CRISPR/Cas9 gene editing therapies for HIV.

Pricing tiers

Go-to-market motion3 records

Distribution channels3 records

Marketing channels6 records

Core offering

CRISPR Therapeutics develops and commercializes CRISPR/Cas9 gene-editing therapies for serious genetic diseases. Its lead approved product, CASGEVY (exagamglogene autotemcel), is the first CRISPR-based gene-editing therapy approved by FDA, MHRA, and EC for sickle cell disease and transfusion-dependent beta thalassemia. The company also advances a diversified pipeline across hemoglobinopathies, immuno-oncology, autoimmune diseases, in vivo cardiovascular, and regenerative medicine.

Differentiator

Problem solved

Functional benefit

Brands

• CASGEVY: First-ever approved CRISPR-based gene-editing therapy for sickle cell disease and transfusion-dependent beta thalassemia, co-developed with Vertex Pharmaceuticals
• SyNTase
• CRISPR-X

Products and services

• CASGEVY (exagamglogene autotemcel) Ex vivo CRISPR/Cas9 gene-edited autologous cell therapy for patients with sickle cell disease and transfusion-dependent beta thalassemia. Administered at authorized treatment centers through Vertex Pharmaceuticals' specialty distribution network at a one-time price of $2.2 million per patient.

Quantifiable outcome

• Over 90% of trial participants achieved functional cure with CASGEVY
• +5 more outcomes

Named customers5 records

Segments5 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

Six partnerships are on record, tiered core, minor and flagship.

• Sirius TherapeuticscoreStrategic or Co-development Partner · 19 May 2025Multi-target collaboration to develop novel siRNA therapies. CRISPR and Sirius announced first patient dosed in Phase 2 trial of SRSD107 for thromboembolic disorders in Europe in September 2025. CTX611 (siRNA targeting FXI) is part of this collaboration.
• Capsida BiotherapeuticsminorStrategic or Co-development Partner · 15 June 2021Strategic collaboration to develop gene-edited therapies for amyotrophic lateral sclerosis (ALS) and Friedreich's Ataxia using Capsida's in vivo gene editing delivery platform.
• NkartaminorStrategic or Co-development Partner · 6 May 2021Global collaboration to develop gene-edited cell therapies for cancer, combining Nkarta's NK cell platform with CRISPR's gene editing capabilities.
• ViaCytecoreStrategic or Co-development Partner · 17 September 2018Strategic collaboration to develop gene-edited stem cell-derived therapy for diabetes. First patient dosed in Phase 1 clinical trial in February 2022. Collaboration includes development of immune-evasive cell replacement therapy for Type 1 diabetes.
• Vertex PharmaceuticalsflagshipStrategic or Co-development Partner · 12 December 2017Vertex and CRISPR co-develop and co-commercialize exa-cel (now CASGEVY) for sickle cell disease and beta thalassemia. Vertex is manufacturer and exclusive license holder with 60/40 U.S. profit-share. Collaboration expanded to include Duchenne's Muscular Dystrophy, Myotonic Dystrophy Type 1, and Cystic Fibrosis programs.
• Bayer (Casebia Therapeutics)coreStrategic or Co-development Partner · 6 June 2016Casebia Therapeutics (Bayer-CRISPR joint venture) focused on developing CRISPR-based therapies for eye, heart, and lung diseases using Bayer's IP and CRISPR's technology.

Scale indicators9 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Intellia Therapeutics: CRISPR/Cas9-based gene editing company developing both in vivo (LNP-delivered, liver-targeted NTLA-2001/2002 for ATTR and HAE) and ex vivo cell therapies. Closely comparable as a co-founder of the original CRISPR IP pool and a direct competitor in hemoglobinopathies, in vivo editing, and gene editing delivery.
• Editas Medicine: CRISPR-based gene editing company developing both Cas9 and Cas12a modalities for sickle cell disease, oncology, and in vivo indications. Shares foundational CRISPR IP with CRISPR Therapeutics and is the most direct competitor in hemoglobinopathies.
• Beam Therapeutics: Pioneer in base editing — a next-generation CRISPR-adjacent modality — with clinical-stage programs in sickle cell disease, alpha-1 antitrypsin deficiency, and other genetic diseases. Directly competes in the same hemoglobinopathy and rare disease gene editing markets.
• Verve Therapeutics: In vivo base editing company focused on cardiovascular disease targeting Lp(a), PCSK9, and ANGPTL3. Closely overlaps with CRSP's CTX310/CTX320 in vivo cardiovascular programs and represents direct competition in the same patient population.

Emerging players

• Prime Medicine: Developer of prime editing, a next-generation CRISPR-derived gene editing modality with applications across rare diseases, oncology, and liver diseases. Competes for similar therapeutic areas and partnership attention from major pharma.
• Caribou Biosciences: CRISPR-based allogeneic CAR T company co-founded by CRISPR co-inventor Jennifer Doudna and a co-holder of foundational CRISPR IP. Partial overlap in allogeneic cell therapy and gene editing platform.
• Tessera Therapeutics: Developer of Gene Writing and other next-generation genome engineering technologies. Emerging competitor in the broader gene editing space with potential overlap in cardiovascular and rare disease indications.

Broad incumbents

• Vertex Pharmaceuticals: CRSP's flagship co-development/co-commercialization partner for CASGEVY (60/40 U.S. profit-share) and holder of exclusive manufacturing/license rights. Both partner and broader incumbent in genetic disease therapeutics (cystic fibrosis franchise).
• Sangamo Therapeutics: Pioneer in genome editing (zinc finger nucleases) with clinical-stage programs in hemoglobinopathies and rare diseases. Operates in the same gene editing category with broader historical footprint but smaller pipeline.

Others

• Nkarta: Allogeneic NK cell therapy company collaborating with CRSP (2021) on gene-edited cell therapies for cancer. Connected through their partnership and shared focus on off-the-shelf engineered cell therapies.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat7 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/crispr-therapeutics
• Xx.com/crisprtx

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Subsidiaries2 records

Funding overview

Funding rounds7 records

Investors13 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments2 records

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about CRISPR Therapeutics