Top 50 Crispr Therapy

CRISPR Therapeutics leverages a proprietary CRISPR/Cas9 platform to create ex vivo edited cell therapies and in vivo lipid‑nanoparticle delivery of gene‑editing components for diseases such as hemoglobinopathies, cancer, autoimmune and metabolic disorders. The company develops autologous and allogeneic cell products, including edited hematopoietic stem cells and CAR‑T cells, as well as liver‑targeted therapies, and generates revenue through sales of approved treatments, licensing agreements, and platform subscriptions.

The startup develops optimized CRISPR guide RNA and edited cells for the cell biology and gene therapy research markets, offering products such as CRISPR reagents and gene knockout kits. Their solutions enable researchers to perform genome engineering more efficiently and reliably, facilitating advancements in genetic research and therapeutic applications.

This Cambridge-based startup develops a synthetic biology platform that utilizes CRISPR technology to engineer induced pluripotent stem cells into functional chassis cells for cell therapies. Their approach aims to enhance treatment options for serious diseases, including autoimmunity and cancer, by enabling the production of genetically engineered therapeutic constructs.

Mammoth Biosciences develops CRISPR-Cas-based gene editing therapeutics and diagnostics to enable precise in vivo applications for serious diseases and molecular detection. Their technology addresses the challenges of gene delivery and expands testing capabilities, providing permanent solutions for unmet medical needs.

Scribe Therapeutics engineers CRISPR-based therapeutics to modify genetic predispositions, specifically targeting cardiometabolic diseases. By creating safe and effective gene-editing solutions, the company aims to empower individuals to take control of their health outcomes rather than being limited by inherited conditions.

SNIPR Biome is pioneering CRISPR-based microbial gene therapy to precisely target and eliminate harmful bacteria associated with difficult-to-treat conditions, including inflammatory bowel disease and multi-drug resistant infections. The company aims to transform the management of microbial diseases by advancing its microbiome drugs into human clinical trials.

GenKOre specializes in gene editing technologies, particularly CRISPR, to develop targeted gene therapies for genetic disorders such as Huntington's disease. The company addresses the limitations of traditional treatments by utilizing patient-specific cells to minimize immune rejection and reduce treatment costs.

Carver Biosciences is developing CRISPR-Cas13-based antiviral therapies that target and degrade the RNA of respiratory viruses, including influenza A and SARS-CoV-2. This platform technology addresses the lack of broad-spectrum antiviral treatments, enabling rapid response to viral outbreaks and pandemics.

CorriXR Therapeutics utilizes a CRISPR/Cas gene editing platform to disable drug resistance genes in tumor cells, specifically targeting squamous cell carcinoma of the head and neck. Their lipid nanoparticle delivery system minimizes off-target effects, enhancing the efficacy of cancer treatments.

Modalis is a biotech company that utilizes CRISPR technology to develop targeted therapeutics for genetic disorders, specifically focusing on conditions like congenital muscular dystrophy. Their approach aims to provide precise gene editing solutions to address the underlying genetic causes of these diseases.

GenomixThera develops personalized cancer therapies that utilize CRISPR-Cas9 gene editing combined with quantum-guided nanoparticle delivery systems to target genomic alterations in tumors. Their approach aims to enhance treatment efficacy by tailoring therapies to individual tumor profiles and improving immune response while addressing the limitations of traditional cancer treatments.

Kamau Therapeutics is a clinical-stage company developing next-generation gene correction therapies using high-efficiency targeted gene integration. The company focuses on creating a new class of medicines designed to cure a range of serious and life-threatening diseases. Their work centers on delivering definitive, curative genetic treatments for patients with genetic disorders.

The startup has developed a mediated genome editing platform that utilizes a CRISPR-based gene editing system, CasPlus, combined with engineered lipid nanoparticle carriers for targeted delivery to critical organs. This technology aims to correct genetic mutations responsible for debilitating diseases, offering potential curative therapies for affected patients.

KSQ Therapeutics utilizes its CRISPRomics® platform to identify and target the genome's most promising therapeutic candidates for curing human diseases. The company is advancing CRISPR/Cas9-engineered Tumor Infiltrating Lymphocytes (eTIL®) programs to develop effective treatments for cancer.

BD Gene Therapeutics utilizes CRISPR technology to precisely edit genetic sequences linked to hereditary diseases. The platform aims to provide targeted therapeutic interventions that can potentially reverse the effects of genetic disorders in patients.

GenEdit utilizes the NanoGalaxy® delivery platform to enhance the safe and efficient delivery of CRISPR-Cas9 gene editing therapies for genetic diseases. This technology addresses the challenge of limited access to effective genetic medicines, enabling broader patient treatment options.

Integra Therapeutics develops gene writing tools that combine CRISPR-Cas precision with engineered piggybac transposase for efficient delivery of both small and large DNA payloads. The technology addresses limitations in gene therapy related to gene size, precision, and stability, aiming to cure genetic and oncological diseases with high unmet medical needs.

Mosaic Therapeutics develops targeted drug combinations for cancer treatment by utilizing genome-scale CRISPR screening and advanced computational models to identify effective therapies based on the molecular characteristics of tumors. The company addresses the challenge of cancer's complexity and heterogeneity, aiming to improve patient outcomes through precision medicine in biomarker-defined settings.

Genesist utilizes CRISPR-based gene therapy to enhance the resistance of stromal cells to cancer, effectively preventing tumor metastasis. By empowering healthy cells surrounding tumors, the technology aims to inhibit cancer invasion and spread.

The startup utilizes a modified CRISPR system to direct stem cells along specific developmental pathways through precise genetic instruction sets, mimicking embryonic development. This technology enables the scalable production of diverse stem cell types for therapeutic applications across various medical conditions.

Addy Gene specializes in CRISPR-Cas gene editing technology, offering services such as gene knockout, point mutation, and cell line construction to enhance genetic modification processes. The company improves the efficiency and precision of gene editing, enabling advancements in biotechnology and therapeutic applications for research institutions and enterprises.

Orphion Therapeutics develops gene therapies utilizing targeted gene editing techniques to address rare genetic disorders caused by specific genetic mutations. Their approach aims to restore normal gene function, potentially improving patient outcomes and quality of life for those affected by these conditions.

This startup develops platform solutions for cell therapy and regenerative medicine, focusing on induced pluripotent stem cell (iPSC) technologies. Their offerings include rapid iPSC reprogramming, a CRISPR-Cas12b gene-editing platform, and bioinformatics-guided differentiation techniques to support upstream and downstream R&D processes for iPSCs and their derivatives.

Aurora Therapeutics develops a scalable platform for personalized gene editing to address rare genetic diseases. The company leverages CRISPR technology to rapidly tailor treatments for specific genetic mutations that current therapies cannot reach. This approach aims to transform personalized gene editing from isolated successes into a broad therapeutic solution for millions of patients.

This company develops genomic technology focused on improving the safety and characterization of gene editing tools for cell and gene therapies. Their INDUCE-seq platform enables developers to accurately detect on- and off-target edits associated with gene editing systems like CRISPR. This technology supports the standardization and acceleration of safer gene-edited therapeutic development.

This company develops cell and gene therapies specifically targeting rare pediatric diseases. They utilize proprietary CRISPR 3.0 technology for precise gene editing to correct genetic mutations in patient cells. Their pipeline includes ex vivo cell therapies that have received Orphan Drug Designation from both the EMA and FDA for severe genetic disorders.

Nerai develops custom CRISPR‑based genome editors using its MORPHEME platform, which combines automated directed evolution, high‑throughput functional screening, and machine learning to achieve high specificity and safety. The company applies these bespoke editors to rare inherited diseases, focusing on liver and retinal targets, and advances multiple in‑vivo programs such as Citrullinemia type 1 and phenylketonuria. By delivering one‑time curative gene‑editing therapies, Nerai aims to expand the treatable genetic space beyond the limits of conventional genome editing.

GeneToBe develops targeted gene delivery systems using viral vectors to introduce corrective genes into patients' cells. This technology aims to treat genetic disorders by directly addressing the underlying genetic mutations responsible for inherited diseases.

Wugen produces off‑the‑shelf allogeneic CAR‑T cell therapies using CRISPR/TALEN editing to delete T‑cell receptor and HLA loci, creating a universal, cryopreservable product that can be shipped to treatment centers on demand. The platform supports a pipeline of CD7‑targeted candidates for T‑cell acute lymphoblastic leukemia, T‑cell non‑Hodgkin lymphoma, acute myeloid leukemia and selected autoimmune diseases, and includes an inducible safety switch for rapid cell ablation. By eliminating patient‑specific manufacturing, the approach reduces time‑to‑treatment and manufacturing costs while expanding access to cellular immunotherapy.

This company develops therapeutic cures for diseases with unmet needs using proprietary LgRNA-guided gene editing technology. They focus on scalable production of ultra-pure ligated single molecule guide RNAs (LgRNA) which offer enhanced efficacy and safety profiles. The platform is being applied to develop LgRNA-conjugate-directed CRISPR polymerase editing (STAR) therapeutics for conditions like neurodegenerative disorders and cancers.

Signia Therapeutics is a French startup focused on developing targeted therapies for genetic disorders using advanced gene editing techniques. The company aims to provide precise treatment options that address the underlying genetic causes of these conditions, improving patient outcomes and quality of life.

SeQure Dx provides analytical tools that evaluate on-target specificity and off-target risks in CRISPR gene editing therapies, ensuring safety throughout the development process. By delivering precise safety data, the company addresses concerns about unintended genomic alterations that could impact patient outcomes and regulatory compliance.

Hemu Gene develops gene therapy technologies using lentivirus and CRISPR/Cas platforms to treat hemoglobin disorders, specifically targeting transfusion-dependent beta-thalassemia. Their lead product, HGI-001, has shown promise in clinical trials, enabling patients to achieve long-term independence from blood transfusions.

EPIGENIC develops next-generation gene modulation therapies by regulating the epigenetic genome for various human diseases. The company utilizes an Epigenetic Modulator platform combining dead Cas protein and epi-enzymes to precisely control gene expression without altering DNA structure. This technology offers high efficiency, long-lasting durability, and favorable safety profiles through non-viral LNP delivery.

Twelve Bio develops CRISPR-Cas12a diagnostic technology that enables precise detection and modification of DNA sequences. This technology addresses the need for accurate genetic diagnostics and targeted gene editing to treat genetic disorders and diseases.

ORIG3N is a biotechnology company focused on developing gene therapies for rare genetically inherited diseases, utilizing CRISPR and other genomic editing technologies. Their approach aims to provide targeted treatments that address the underlying genetic causes of these conditions, improving patient outcomes and quality of life.

The startup develops immunotherapy cancer treatments utilizing CRISPR and mRNA technologies to create a universal cancer vaccine and conventional immunotherapy options. This approach enables researchers and institutions to enhance their cancer vaccine portfolios, addressing the need for more effective cancer therapies.

Immitra Bio is developing a CRISPR-Cas-based gene therapy platform that utilizes Hematopoietic Stem Cells and their derivatives, such as red blood cells, to produce therapeutic proteins in vivo. This technology aims to provide a versatile and easily reprogrammable solution for the production of biotherapeutics, addressing the need for efficient protein manufacturing in gene therapy applications.

Function Oncology utilizes a CRISPR-powered functional genomics platform to assess gene function in cancer patients, moving beyond traditional gene sequencing methods. This approach enables the identification of personalized therapeutic opportunities, enhancing the development of targeted cancer treatments.

This clinical-stage biopharmaceutical company develops allogeneic cell therapies for serious diseases using proprietary genome-editing technology. Their chRDNA platform enables precise, complex genome editing for creating "off-the-shelf" CAR-T cell therapies. The company focuses on armoring these cell therapies through strategies like immune cloaking to enhance anti-disease activity.

The startup has developed a biotechnology platform that utilizes precision gene editing techniques to create targeted gene therapies for severe and life-threatening diseases. This platform enables healthcare providers to improve patient outcomes by delivering tailored treatments for various genetic disorders.

Brink Therapeutics develops next-generation recombinase enzymes designed for ultra-precise and efficient gene editing. These enzymes aim to enable large-scale production of cell and gene therapies for treating cancer and genetic disorders.

Genevide is a biotech company focused on gene research and development, utilizing CRISPR technology for precise gene editing. The company aims to enhance genetic therapies for genetic disorders, improving treatment efficacy and patient outcomes.

The startup operates a gene-editing application platform that utilizes CRISPR technology to engineer animals, plants, and microorganisms for the production of safe, non-allergic biomaterials. These biomaterials are designed for clinical, aesthetic, and regenerative medicine applications, providing healthcare professionals with customizable solutions for tissue repair and regeneration.

Amber Bio develops novel gene editing modalities capable of performing multi-kilobase edits to address previously undruggable patient populations. The company is creating a first-of-its-kind RNA writing platform designed to treat thousands of diverse mutations with a single therapeutic product. This approach moves beyond developing separate therapies for individual mutations in genetic medicine.

The startup has developed a CRISPR-based platform for molecular diagnostics that enables multiplex detection of DNA and RNA biomarkers with the simplicity of Lateral Flow Assays and the precision of PCR. This technology enhances patient care by providing healthcare professionals with reliable diagnostic tools that facilitate informed healthcare decisions.

The company utilizes CRISPR technology and artificial intelligence algorithms to develop precision anti-cancer drugs through its Platform C and Platform H pipelines. This approach provides medical professionals with targeted treatment options to improve patient outcomes in cancer care.

CorrectSequence Therapeutics utilizes base editing technology to develop targeted therapies for severe genetic diseases, specifically focusing on conditions like hemoglobinopathy. Their approach enables precise modifications to DNA sequences, offering potential breakthroughs in treatment where traditional methods fall short.

EdiGene develops genome editing technologies, including the LEAPER platform for in vivo RNA base editing and the MAGIC platform for allogeneic cell therapies, to address genetic diseases and cancer. Their high-throughput genome editing screening capabilities enable precise therapeutic interventions, enhancing treatment efficacy and patient outcomes.

CLOVERNA is a biopharmaceutical company utilizing a CRISPR-based RNA-targeted drug discovery platform to identify drug target RNA motif sequences in non-coding regions of the genome. This technology addresses the challenge of unexplored non-coding RNA, which has been overlooked in drug development despite its potential role in various diseases.