Crispr Therapeut Stock Price, News & Analysis

CRISPR Therapeutics AG (Nasdaq: CRSP) is a biopharmaceutical and gene editing company focused on developing transformative gene-based medicines for serious diseases. The company centers its work on CRISPR/Cas9 technology and related platforms to precisely edit genomic DNA and RNA. According to company disclosures, CRISPR Therapeutics has evolved from a pioneering research-stage organization into a gene editing leader, highlighted by the approval of CASGEVY, described as the world’s first CRISPR-based therapy for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia.

CRISPR Therapeutics is headquartered in Zug, Switzerland, with a wholly owned U.S. subsidiary and research and development operations based in Boston, Massachusetts and San Francisco, California. Its shares trade on Nasdaq under the symbol CRSP. The company describes a broad and diversified pipeline spanning hemoglobinopathies, oncology, regenerative medicine, cardiovascular and autoimmune conditions, and rare diseases, supported by proprietary gene-editing and delivery technologies.

Core focus: gene editing medicines

The company’s work is rooted in the CRISPR/Cas9 system, which enables targeted changes to specific DNA sequences. CRISPR Therapeutics reports that it uses this technology both in ex vivo cell therapies, where cells are edited outside the body and reinfused, and in in vivo approaches, where editing occurs directly in the body using delivery platforms such as lipid nanoparticles (LNPs). In addition, the company has added a small interfering RNA (siRNA) pillar through a collaboration with Sirius Therapeutics, broadening its toolkit beyond DNA editing.

To expand its capabilities, CRISPR Therapeutics has developed the SyNTase™ editing platform, described as a novel, proprietary gene-editing technology designed to enable precise, efficient, and scalable gene correction, including single-dose in vivo gene correction in preclinical models. The company also highlights a proprietary LNP delivery platform for liver-directed in vivo gene editing.

Hemoglobinopathies and CASGEVY

A central program for CRISPR Therapeutics is CASGEVY® (exagamglogene autotemcel [exa-cel]), a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). In this therapy, a patient’s own hematopoietic stem and progenitor cells are edited at an erythroid-specific enhancer region of the BCL11A gene, which results in production of high levels of fetal hemoglobin (HbF) in red blood cells. Company materials state that CASGEVY has been shown to reduce or eliminate vaso-occlusive crises in SCD and transfusion requirements in TDT.

CRISPR Therapeutics notes that CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies in the United States, Great Britain or the United Kingdom, the European Union, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Qatar, Canada, Switzerland, the United Arab Emirates, and Kuwait. The company and its partner Vertex Pharmaceuticals have also advanced pediatric development in patients aged 5 to 11 years through global Phase 3 studies, and Vertex is expected to lead regulatory submissions for this age group.

CASGEVY emerged from a long-standing collaboration between CRISPR Therapeutics and Vertex Pharmaceuticals, initiated in 2015 to discover and develop therapies using CRISPR/Cas9 technology. Under an amended collaboration agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY and shares program costs and profits worldwide with CRISPR Therapeutics. Vertex is described as the manufacturer and exclusive license holder of CASGEVY.

In vivo liver editing and cardiovascular/metabolic programs

CRISPR Therapeutics reports an extensive portfolio of in vivo liver-directed gene editing programs that leverage its proprietary LNP delivery platform. These programs focus on cardiovascular, cardiometabolic, and rare liver-related diseases where modulation of specific liver-expressed genes can alter disease risk or progression.

The lead in vivo program is CTX310®, an investigational CRISPR/Cas9 therapy designed to edit the ANGPTL3 gene in hepatocytes. ANGPTL3 encodes a protein that regulates triglyceride (TG) and low-density lipoprotein (LDL) levels. Company-reported Phase 1 data show dose-dependent, durable reductions in circulating ANGPTL3, TG, and LDL following a single-course intravenous infusion, and CTX310 is being advanced into Phase 1b clinical trials, with development prioritized in severe hypertriglyceridemia and mixed dyslipidemias.

Another clinical-stage in vivo candidate is CTX320™, which targets the LPA gene that encodes apolipoprotein(a), a major component of lipoprotein(a) [Lp(a)]. Elevated Lp(a) is described as a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events. CTX320 is in an ongoing Phase 1 clinical trial in patients with elevated Lp(a), and the company is also advancing a next-generation Lp(a) program, CTX321™, in preclinical development, incorporating an updated guide RNA for increased potency.

CRISPR Therapeutics is also progressing preclinical in vivo candidates such as CTX340™, targeting angiotensinogen (AGT) for refractory hypertension, and CTX450™, targeting ALAS1 for acute hepatic porphyria (AHP). These programs are reported to be in IND/CTA-enabling or preclinical stages, with preclinical data demonstrating gene editing, biomarker changes, and durability in animal models.

SyNTase™ platform and CTX460™ for alpha-1 antitrypsin deficiency

The company’s SyNTase™ editing platform is highlighted as a novel gene-editing approach designed for precise in vivo gene correction. The first investigational candidate from this platform is CTX460™, targeting the SERPINA1 gene mutation that causes alpha-1 antitrypsin deficiency (AATD). AATD is described as a genetic disorder most commonly caused by the Z allele (PiZ, E342K) in SERPINA1, leading to misfolded Z-AAT protein accumulation in the liver and insufficient functional M-AAT in circulation.

Preclinical data disclosed by CRISPR Therapeutics show that a single dose of CTX460 in animal models achieved high levels of DNA and mRNA correction in the liver, substantial increases in total serum AAT, and an M-AAT:Z-AAT ratio above 99%, with durable effects over several weeks. These findings are presented as preclinical proof-of-concept for single-dose in vivo gene correction using the SyNTase platform, and the company has stated its intention to move CTX460 into clinical trials.

Autoimmune disease and immuno-oncology: zugo-cel and cell therapies

CRISPR Therapeutics is also active in autoimmune disease and immuno-oncology through its allogeneic CAR T cell therapy programs. A key candidate is zugocaptagene geleucel (zugo-cel; formerly CTX112™), a wholly owned, allogeneic CAR T product targeting CD19. Zugo-cel is described as an off-the-shelf therapy that uses CRISPR/Cas9 for targeted gene knockout and CAR insertion to support immune evasion and T cell potency, administered after standard lymphodepletion without the need for HLA matching.

Zugo-cel is being investigated in Phase 1 basket trials for rheumatologic autoimmune diseases, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis, as well as in a separate Phase 1 trial for autoimmune hematologic disorders such as immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA). Preliminary data reported by the company describe deep B-cell depletion, shifts toward naïve B-cell repertoires, and clinical improvement in early patients, with some SLE patients achieving drug-free remission measures.

In oncology, zugo-cel is being evaluated in a Phase 1/2 trial for relapsed or refractory B-cell malignancies, including large B-cell lymphoma and other CD19-positive subtypes. Company updates describe high overall and complete response rates at the recommended Phase 2 dose, along with observed CAR T cell expansion and reported safety profiles. CRISPR Therapeutics has also entered into a collaboration and clinical supply agreement with Lilly to evaluate zugo-cel in combination with pirtobrutinib in aggressive B-cell lymphomas.

The company has previously advanced other CAR T candidates such as CTX131™, targeting CD70, and has indicated that resources have been redirected toward programs with greater perceived long-term potential. CRISPR Therapeutics also notes that its autoimmune and immuno-oncology programs are supported by a wholly owned GMP manufacturing facility in Framingham, Massachusetts, which provides end-to-end production capabilities for its cell therapy portfolio.

siRNA collaboration with Sirius Therapeutics

Beyond DNA editing, CRISPR Therapeutics has established a strategic collaboration with Sirius Therapeutics to develop small interfering RNA (siRNA) therapies for thromboembolic and other serious diseases. The lead program in this collaboration is SRSD107, also referred to as CTX611™ in some company materials, a long-acting siRNA targeting coagulation Factor XI (FXI).

SRSD107/CTX611 is designed to inhibit FXI expression and modulate the intrinsic coagulation pathway, with the goal of providing anticoagulant and antithrombotic effects while potentially minimizing bleeding risk. Phase 1 trials in Australia and China reported strong, sustained reductions in FXI levels and prolonged activated partial thromboplastin time, with effects maintained for months after dosing. A Phase 2 trial in patients undergoing total knee arthroplasty (TKA) is underway in Europe to evaluate prevention of venous thromboembolism and to inform dose selection for future pivotal studies.

Under the collaboration, CRISPR Therapeutics and Sirius share development costs and profits for SRSD107, with CRISPR Therapeutics leading commercialization in the U.S. and Sirius leading in Greater China. The agreement also provides CRISPR Therapeutics with options to license additional siRNA programs from Sirius, extending its reach into RNA-based medicines.

Regenerative medicine and diabetes

CRISPR Therapeutics reports a regenerative medicine portfolio that includes efforts in Type 1 diabetes (T1D). The company has disclosed programs such as CTX211™ and next-generation candidates like CTX213™, which use induced pluripotent stem cell (iPSC)-derived, allogeneic, gene-edited beta islet cell precursors. Early clinical data from CTX211 showed detectable C-peptide levels 12 months after implantation, and these findings have informed the design of next-generation hypoimmune cell engineering approaches represented by CTX213.

According to company updates, CTX213 has demonstrated preclinical efficacy and is progressing toward clinical development, with the goal of achieving insulin independence in T1D patients without chronic immunosuppression. These programs illustrate how CRISPR Therapeutics applies gene editing to cell replacement strategies in addition to direct gene correction.

Business model, collaborations, and capital markets activity

CRISPR Therapeutics’ business model, as reflected in its public communications and SEC filings, combines internal research and development with collaborations and capital markets financing. The company highlights strategic partnerships with organizations such as Vertex Pharmaceuticals for CASGEVY and Sirius Therapeutics for siRNA programs, as well as a collaboration with Lilly around zugo-cel in oncology.

On the capital side, CRISPR Therapeutics has used an at-the-market equity program under an Open Market Sale Agreement with Jefferies LLC to offer and sell common shares. An October 2025 Form 8-K notes that the company filed a prospectus supplement to sell additional common shares with an aggregate offering price of up to a specified amount under a previously filed automatic shelf registration statement. The company’s 8-K filings also regularly furnish quarterly financial results and business highlights, reflecting ongoing operations as a publicly traded issuer.

Geographic footprint and sector classification

CRISPR Therapeutics AG is organized in Switzerland and lists its principal location as Zug, Switzerland in SEC filings. The company’s U.S. subsidiary, CRISPR Therapeutics, Inc., operates research and development facilities in Boston, Massachusetts and San Francisco, California. Within industry classification systems, the company is described as a biopharmaceutical or gene editing company and is associated with biological product manufacturing for therapeutic use.

FAQs about CRISPR Therapeutics (CRSP) stock and business

• What does CRISPR Therapeutics do?
  CRISPR Therapeutics develops gene-based medicines using CRISPR/Cas9 and related technologies. Its programs span ex vivo cell therapies, in vivo gene editing, and siRNA-based therapies targeting serious diseases such as hemoglobinopathies, cardiovascular disease, autoimmune disorders, cancer, and rare genetic conditions.
• What is CASGEVY and how is CRISPR Therapeutics involved?
  CASGEVY (exagamglogene autotemcel [exa-cel]) is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease or transfusion-dependent beta thalassemia. CRISPR Therapeutics co-discovered and developed CASGEVY with Vertex Pharmaceuticals. Vertex leads global development, manufacturing, and commercialization, with costs and profits shared between the companies.
• Which therapeutic areas are priorities for CRISPR Therapeutics?
  Company materials identify hemoglobinopathies, oncology, regenerative medicine, cardiovascular and autoimmune diseases, and rare diseases as key areas of focus. Programs include CASGEVY for SCD and TDT, zugo-cel for autoimmune and B-cell malignancies, in vivo liver editing candidates like CTX310 and CTX320, SyNTase-based CTX460 for AATD, siRNA program SRSD107/CTX611 for thromboembolic disorders, and regenerative medicine candidates for Type 1 diabetes.
• How does CRISPR Therapeutics use in vivo gene editing?
  For in vivo applications, CRISPR Therapeutics uses a proprietary lipid nanoparticle (LNP) platform to deliver CRISPR/Cas9 or SyNTase editors to the liver. This approach is used in programs such as CTX310 for ANGPTL3, CTX320 and CTX321 for Lp(a), CTX340 for angiotensinogen, CTX450 for ALAS1, and CTX460 for SERPINA1 in AATD.
• What is the SyNTase editing platform?
  SyNTase is described by the company as a novel, proprietary gene-editing platform designed to enable precise, efficient, and scalable in vivo gene correction. Preclinical data with CTX460 in AATD models show high levels of gene and mRNA correction and increased functional protein levels, supporting SyNTase as a potential foundation for additional gene correction programs.
• What is zugo-cel and what diseases is it being studied in?
  Zugocaptagene geleucel (zugo-cel; formerly CTX112) is an allogeneic CAR T cell therapy targeting CD19. It is being evaluated in Phase 1 and Phase 1/2 trials for rheumatologic autoimmune diseases such as systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis, as well as autoimmune hematologic conditions like ITP and wAIHA, and relapsed or refractory B-cell malignancies including large B-cell lymphoma.
• How is CRISPR Therapeutics involved in siRNA therapies?
  Through a collaboration with Sirius Therapeutics, CRISPR Therapeutics is co-developing SRSD107/CTX611, a long-acting siRNA targeting Factor XI for thromboembolic and clotting-related disorders. The collaboration allows both companies to share development costs and potential profits, and provides CRISPR Therapeutics with options on additional siRNA programs.
• Where is CRISPR Therapeutics headquartered?
  CRISPR Therapeutics AG is headquartered in Zug, Switzerland. Its U.S. subsidiary operates research and development facilities in Boston, Massachusetts and San Francisco, California.
• How does CRISPR Therapeutics fund its research and development?
  Public disclosures indicate that CRISPR Therapeutics funds its activities through a combination of cash, cash equivalents, and marketable securities, collaboration arrangements, and equity offerings. For example, an October 2025 Form 8-K describes the use of an at-the-market offering program under an Open Market Sale Agreement to sell additional common shares under a shelf registration statement.
• What role do partnerships play in CRISPR Therapeutics’ strategy?
  Partnerships are a central part of the company’s approach. The collaboration with Vertex Pharmaceuticals underpins CASGEVY, while the alliance with Sirius Therapeutics extends into siRNA medicines. The company has also entered into agreements with Lilly around zugo-cel in oncology and references strategic collaborations with other biopharmaceutical partners in its public statements.