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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

DEM Bio is developing a proprietary immunotherapeutics platform that utilizes inter-cellular CRISPR screening to identify novel 'don't eat me' and 'eat me' signals on cancer cells and macrophages. This technology aims to enhance the innate immune response by activating macrophages and myeloid effector cells to effectively eliminate tumors.

Broken String Biosciences develops the INDUCE-seq® platform, a technology for characterizing genome breaks in cell and gene therapies. This platform addresses the challenge of off-target effects in CRISPR-Cas9 gene editing, enhancing the safety and efficacy of these transformative treatments.

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.

Prime Medicine utilizes Prime Editing, a precise gene editing technology that enables the targeted replacement of faulty DNA sequences without causing double-strand breaks. This approach aims to directly correct genetic mutations responsible for debilitating diseases, potentially offering long-lasting therapeutic benefits.

The company develops genome editing-based therapies for genetic disorders, oncology, and organ transplants. Their technology enables precise genetic recombination to create off-the-shelf treatments for previously incurable diseases.

MEDIC develops a cancer drug target discovery platform that integrates CRISPR functional genomics with patient tumor-like cancer models. This platform identifies potential targets for cancer drug development, aiming to accelerate the discovery of new cancer treatments.

Develops proprietary genomic editors that target the root causes of genetic diseases by selecting optimal editing tools based on disease-specific pathology. This approach enables the discovery and development of potentially curative genomic medicines, with a focus on liver and central nervous system disorders.

MEDiC utilizes a proprietary CRISPR functional genomics platform combined with 3D tumor models to identify cancer biomarkers and novel drug targets with high precision. This approach addresses the challenge of missed potential biomarkers in current drug development, enhancing the accuracy of patient stratification for oncology treatments.

The startup develops genetic medicines utilizing genome editing and engineering techniques to create pharmaceutical and biological preparations for human therapeutics. This approach enables researchers to effectively target and modify genetic disorders, improving treatment outcomes for patients.

Kytopen develops a non-viral gene delivery platform, Flowfect®, that utilizes continuous fluid flow and electric fields to efficiently deliver mRNA, DNA, and CRISPR Cas9 RNP to billions of cells in minutes. This technology addresses the challenges of cell therapy manufacturing by enabling scalable and customizable transfection processes from discovery to large-scale production.

The startup develops a healthcare technology platform that reprograms recombinases for precise gene editing, enabling the restoration of health in patients with severe conditions. Its technology provides clients with disease-modifying product candidates, enhancing the therapeutic potential of gene editing.

BioSourcing utilizes a proprietary platform that employs CRISPR-Cas9 gene editing and reproductive technologies to produce monoclonal antibodies in the milk of specially bred goats, significantly lowering production costs and carbon footprint. This approach addresses the global shortage and high costs of biopharmaceuticals, making essential treatments more accessible and sustainable for underserved populations.

Beam Therapeutics develops precision genetic medicines using base editing technology to enable targeted modifications of DNA sequences. This approach addresses the limitations of traditional gene editing methods, offering potential treatments for genetic disorders with improved accuracy and reduced off-target effects.

Alia Therapeutics is developing next-generation gene-editing therapies that utilize precise genome editing techniques to target and correct mutations responsible for rare genetic diseases. By focusing on the efficacy and durability of these treatments, the company aims to provide long-lasting cures that address the root causes of genetic disorders.

This startup provides bioinformatics solutions for drug discovery and development, focusing on personalized and precision therapies. Their services include CRISPR/siRNA selection, customized reference genomes, target discovery, and mechanism of action identification, helping healthcare firms accelerate drug development for cancers and rare diseases.

Editpep develops a class of peptides designed to deliver CRISPR enzymes effectively, enhancing the precision of gene editing applications. This technology addresses the challenge of efficient and targeted delivery in CRISPR therapies, improving the potential for therapeutic outcomes.

Trobix Bio develops precision human microbiome therapeutics using its TBX technology platform, which combines CRISPR, engineered phages, and synthetic biology to deliver targeted DNA cargo to specific gut bacteria. This approach enables the in vivo production of biotherapeutics for metabolic inflammatory and cancer diseases, addressing the challenges of microbiome dysbiosis and transient bacterial colonization.

MyFloraDNA operates a DNA plant laboratory that utilizes CRISPR technology for precise gene editing to develop allergy-free peanuts, targeting specific allergenic compounds without altering the peanuts' natural qualities. This approach aims to significantly reduce allergy-related health incidents for millions of peanut allergy sufferers.

InnoSkel is a biotechnology company developing targeted gene therapies for rare skeletal diseases, utilizing advanced CRISPR technology to correct genetic mutations. Their approach aims to improve patient outcomes by addressing the underlying genetic causes of these conditions, which currently lack effective treatment options.

The startup has developed a patented, non-permanent gene therapy utilizing zinc-finger and CRISPR-based epigenome regulation to target chronic pain without the risk of addiction. This approach addresses the opioid epidemic by providing a specific and long-lasting pain management solution that allows patients to maintain normal sensory function.

GenEditBio develops precision gene editing technologies to create targeted in vivo gene therapies for genetic diseases. Their platform utilizes advanced gene editing tools to address a wide range of genetic mutations, offering safe and effective treatment options.

Intellia Therapeutics develops in vivo CRISPR‑Cas9 gene‑editing medicines that directly correct disease‑causing mutations in patients with severe monogenic disorders. Its platform integrates guide‑RNA design, lipid nanoparticle/AAV delivery, safety screening, and GMP manufacturing to advance candidates from target discovery through clinical trials, aiming for single‑dose curative treatments.

Synvect utilizes CRISPR gene knockout technology to produce sterilized mosquito eggs that develop into non-biting males, effectively reducing disease-carrying mosquito populations. This biological solution addresses the global health crisis caused by mosquito-borne diseases, which result in approximately 700 million cases and 1 million deaths annually.

Algen Biotechnologies utilizes a proprietary CRISPR gene-modulation system and advanced AI models to decode complex gene networks and identify disease-driving RNA signaling pathways. This approach accelerates therapeutic drug discovery by providing high-confidence insights into the biological mechanisms underlying various diseases.

Acrigen Biosciences develops precision gene editing systems utilizing proprietary αCas and μCas CRISPR-Cas nucleases paired with engineered anti-CRISPR proteins to enhance the accuracy and safety of genetic therapies. The company aims to address genetic disorders by providing targeted and efficient gene editing solutions for human therapeutics.

CrisprBits utilizes CRISPR gene-editing technology to develop affordable diagnostics and therapeutics for infectious and inherited diseases, as well as cancer. The company focuses on creating next-generation CAR-T cells and disease tissue models from edited induced pluripotent stem cells, addressing critical healthcare needs in India.

InEdita Bio has developed a proprietary genome editing platform utilizing CRISPR technology to create crops with enhanced resistance to pests and diseases, while improving nutrient acquisition efficiency. This technology reduces the reliance on chemical fertilizers and pesticides, addressing the challenges of food security and environmental sustainability in agriculture.

The startup develops a microfluidics chip that enables non-viral transfection for CRISPR screening at single donor resolution, requiring 100 times less input material. This technology significantly reduces the time and cost of drug discovery workflows by enhancing lab automation through miniaturization and parallelization.

C4U Corporation specializes in the research and development of gene editing technologies, focusing on CRISPR-based methods to precisely modify genetic sequences. The company aims to address genetic disorders and enhance agricultural traits, providing targeted solutions for health and food security challenges.

HuidaGene Biotechnology Co., Ltd develops CRISPR-based gene-editing tools and therapies targeting genetic disorders in ophthalmology, neurology, and neuromuscular diseases. Their technology aims to repair genetic mutations responsible for life-threatening conditions, such as Duchenne Muscular Dystrophy and inherited retinal dystrophies.

Reforgene Medicine utilizes gene editing and genomic technology to develop curative therapies for genetic diseases, specifically targeting conditions like thalassemia and Usher syndrome. Their approach aims to provide lifelong cures through a single administration of gene editing treatments, addressing the significant unmet medical needs of patients suffering from these hereditary disorders.

Cellogen Therapeutics develops third and fourth generation CAR T-cell therapies and gene editing techniques, including CRISPR, to treat hematological malignancies and genetic blood disorders. The company aims to provide long-lasting cures by targeting the root causes of diseases, significantly improving patient outcomes and reducing treatment costs.

The startup develops targeted therapeutics that utilize gene editing techniques to treat cancer, focusing on genetic alterations that conventional therapies cannot reach. This approach enables patients to receive personalized treatment options that improve their health outcomes and quality of life.

GrittGene Therapeutics is focused on developing a cure for Myotonic Dystrophy Type 2 using CRISPR technology and artificial intelligence for precise gene editing and tissue-specific delivery. By leveraging a multidisciplinary approach and a wide range of expertise, the company aims to provide effective treatments that go beyond merely alleviating symptoms.

The startup provides customized genetic modeling services using CRISPR-facilitated gene targeting in embryonic stem cells to create specific mutations for research. This technology enables clients to efficiently develop knockout mice and complex genetic modifications for studying gene function and disease mechanisms.

Stat Labs develops novel CRISPR-based gene editing tools for therapeutic applications. Their technology aims to precisely modify DNA sequences to correct genetic mutations and treat a range of diseases.

Neoclease engineers highly precise and efficient gene-editing nucleases using a generative AI platform. Their technology enables targeted therapeutic interventions for genetic disorders and complex diseases, including novel approaches for difficult-to-access tissues.

MyoPax develops proprietary regenerative stem cell therapies utilizing patented methods for isolating and cultivating primary human satellite cells, alongside CRISPR/Cas gene-editing technologies for precise gene repair. The company targets muscle function loss due to disorders such as muscular dystrophies and trauma, aiming to restore muscle health and improve patients' quality of life.