CRISPR Therapeutics
CRISPR Therapeutics is a premier gene editing company globally recognized for translating the revolutionary CRISPR/Cas9 technology into clinical-stage and approved therapeutics. The company has a robust pipeline focused on diseases with high unmet medical need, particularly in hematological disorders like Sickle Cell Disease (SCD) and transfusion-dependent beta thalassemia, as well as in oncology, regenerative medicine, and rare diseases. A cornerstone of their portfolio is the co-developed gene-editing treatment that achieved historic regulatory approval as the first-ever CRISPR-based therapy. Beyond therapeutic candidates, CRISPR Therapeutics continuously works to advance the core technology by developing high-fidelity Cas9 variants, improving guide RNA design, and utilizing advanced delivery approaches like electroporation and lipid nanoparticles. Their R&D pillars include refining gene editing tools, exploring next-generation techniques such as base and prime editing, and applying these breakthroughs to complex challenges like allogeneic CAR-T cancer therapies and direct *in vivo* editing strategies.
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Intellia Therapeutics
Intellia Therapeutics is a leading genome-editing biotechnology company dedicated to developing curative CRISPR/Cas9-based therapies across two primary approaches: *in vivo* and *ex vivo*. Their *in vivo* strategy involves delivering CRISPR components directly to target tissues within the body, exemplified by the first-ever systemic *in vivo* CRISPR therapy tested in humans for transthyretin amyloidosis (ATTR). Their most advanced program targets hereditary angioedema (HAE), aiming for a permanent reduction in painful swelling attacks. The *ex vivo* approach focuses on modifying cells outside the body, such as hematopoietic stem cells or T cells, before reinfusion for treating diseases like Acute Myeloid Leukemia. Intellia’s commitment to unlocking the full potential of CRISPR technology for a broad range of genetic disorders, coupled with its advanced clinical-stage pipeline, positions it as a key driver in the development and validation of genome editing as a transformative medicine.
Editas Medicine
Editas Medicine is one of the pioneering companies in the gene editing space, recognized for its early intellectual property foundation and unwavering focus on developing transformative genomic medicines for serious diseases. The company’s core strategy centers on utilizing CRISPR/Cas9 technology for precise gene editing to repair disease-causing genes in patients with genetically determined disorders. Their pipeline includes both *in vivo* and *ex vivo* programs, leveraging the power of CRISPR to address genetic mutations with the goal of providing durable and potentially curative therapeutic solutions. By employing a direct, targeted approach to genetic sequences, Editas is working to correct broken genes, improve patient outcomes, and address high unmet needs in areas like ocular and hematologic diseases. This innovative focus on engineering lasting solutions solidifies their role as a leader in translating foundational CRISPR science into clinical reality.
Caribou Biosciences
Caribou Biosciences positions itself as a next-generation CRISPR company, primarily focused on developing allogeneic (off-the-shelf) cell therapies for life-threatening diseases. Their core technological distinction is the proprietary chRDNA (CRISPR hybrid RNA–DNA) platform. This innovative platform is designed to overcome limitations of traditional CRISPR methods by supporting more complex genomic editing while simultaneously preserving genomic integrity. The chRDNA system aims to reduce unwanted off-target effects and chromosomal damage, which is critical for enhancing the safety, efficacy, and manufacturability of cell therapies. By leveraging this advanced platform, Caribou is accelerating the development of allogeneic CAR-T and natural killer (NK) cell therapies for oncology, affirming their role as a key innovator in applying CRISPR to create safer and more effective therapeutic products.
Verve Therapeutics
Verve Therapeutics is revolutionizing the treatment paradigm for cardiovascular disease by moving away from chronic, long-term medication management toward single-course gene-editing therapies. Their mission is to permanently lower the risk of heart disease by editing specific genes in the liver that control blood lipid levels, such as PCSK9 and ANGPTL3. Verve leverages the precision of CRISPR-based technology to achieve this permanent modification with a one-time treatment. This novel application of gene editing represents a major technological leap, offering the potential to provide a curative solution for what is currently one of the world’s most prevalent health crises. Their strategy to deploy CRISPR to treat common diseases positions them at the forefront of using genetic modification to fundamentally change population health outcomes.
Eligo Bioscience
Eligo Bioscience is a unique and focused player in the CRISPR landscape, specializing in the precision gene editing of the microbiome to treat diseases driven by bacterial genes. They employ their proprietary Gene Editing of the Microbiome (GEM) platform, which ingeniously utilizes engineered bacteriophages to deliver CRISPR-Cas systems directly to specific bacterial populations within the gut. This technology allows for the precise and targeted elimination of pathogenic bacteria or the correction of harmful bacterial genes without causing widespread disruption to the overall balance of the healthy microbiome. Their approach, which includes Sequence-Specific Antimicrobials (SSAM) as a modality, enables them to address various diseases associated with microbiome dysbiosis, including antibiotic-resistant infections and chronic conditions. Eligo’s in-vivo targeting of bacterial genes positions them as a pioneer in developing highly selective and transformative microbial gene therapies.
Thermo Fisher Scientific
Thermo Fisher Scientific is a globally renowned leader that acts as a critical enabler of the entire CRISPR research and development ecosystem. The company significantly contributes to the field by offering a comprehensive suite of reagents, delivery systems, and custom gene editing solutions, making it an indispensable resource for both academic and industrial scientists. Their expansive platform supports everything from early discovery research to therapeutic development and commercialization, providing high-quality Cas enzymes, guide RNAs, and delivery technologies. Thermo Fisher’s robust supply chain and commitment to integrating with evolving gene editing techniques ensure that researchers have access to the necessary infrastructure and tools to refine CRISPR specificity, test efficacy, and accelerate the development of new genomic medicines worldwide.
Danaher Corporation
Danaher Corporation, a diversified global science and technology conglomerate, is a major force in the CRISPR market, primarily through its life sciences and diagnostics operating companies. They supply essential life sciences instrumentation and specialty tools that are crucial for enabling CRISPR-driven workflows. This includes advanced diagnostic platforms, high-quality reagents, and integrated, automated systems necessary for complex laboratory processes such as genetic screening, sample preparation, and quality control. By strategically investing and focusing on seamless integration of these components, Danaher provides the technological backbone for next-generation precision medicine and genomic analysis, ensuring that researchers and clinicians have the reliable and high-throughput systems required to successfully implement and scale CRISPR-based research and clinical applications.
Illumina, Inc.
Illumina, Inc. is a technological powerhouse whose sequencing platforms are foundational to the validation and success of the CRISPR industry. While not a therapeutic developer, the company provides the critical infrastructure necessary for verifying CRISPR edits with unparalleled accuracy. Their Next-Generation Sequencing (NGS) platforms are widely used by researchers to perform comprehensive genetic screenings, confirm on-target editing, and—crucially—identify and quantify any unwanted off-target effects. This capability is indispensable for the R&D, quality control, and regulatory submission processes for all gene-editing therapies. By empowering users to track gene editing outcomes and discover new genome targets, Illumina plays an essential, enabling role in accelerating scientific breakthroughs in academic, clinical, and agri-genomics CRISPR applications globally.
Graphite Bio
Graphite Bio is a gene editing company dedicated to developing therapies by leveraging the cell’s natural DNA repair processes to precisely rewrite genes. The company builds upon foundational CRISPR technology, but their focus is on homology-directed repair (HDR) as the preferred mechanism to achieve targeted gene insertion, correction, or replacement. This approach is designed to provide highly precise and stable genetic outcomes, aiming to cure diseases by correcting the underlying genetic defect. Their pipeline targets serious diseases, with a flagship program focusing on blood disorders like sickle cell disease and beta-thalassemia. By concentrating on this high-precision method of gene repair, Graphite Bio is advancing the field toward truly curative genetic medicines, minimizing the reliance on less predictable DNA repair pathways.
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