Evonetix Ltd is a biotechnology company based in Little Chesterford, United Kingdom, established in 2015. The company focuses on developing innovative technology for the parallel synthesis of DNA on silicon arrays, thereby advancing the field of synthetic biology. By employing novel techniques, Evonetix aims to synthesize DNA with higher accuracy and scale, which enhances accessibility for researchers. This platform enables scientists to utilize synthetic biology on a previously unattainable scale, fundamentally changing how DNA is accessed, produced, and utilized for gene synthesis. The engineering team at Evonetix boasts extensive experience in creating high-integrity automated systems, bringing together a diverse range of expertise from various scientific and engineering disciplines to support its mission.
Myome helps identify genes that cause disease and act based on leading science. The data is secure, portable, and reusable throughout life.
Mirvie is a biotech company that creates precise, actionable, and non-invasive tests for maternal-fetal health. The company delivers insights to moms and families to make pregnancy safe. It was founded in 2018 by Maneesh Jain and is headquartered in South San Francisco, California, United States.
DNAnexus, Inc. specializes in cloud-based genome informatics and data management tools tailored for enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company provides a platform that facilitates the analysis of raw sequencing data, integrates genomic data with clinical information, and enhances collaboration among researchers. Key offerings include the MOSAIC Microbiome platform for microbiome research, DNAnexus Apollo for clinico-genomic data exploration, DNAnexus Titan for data analysis, and DNAnexus Portals, which create secure online workspaces for collaborative projects. Additionally, the Clinico-Genomic Data Solution aims to enhance disease-specific datasets through partnerships with healthcare providers, improving screening and treatment pathways for complex diseases. DNAnexus serves a diverse clientele, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.
TenSixteen Bio is a biotechnology company based in San Francisco that specializes in developing innovative therapeutics for various age-related disorders. The company focuses on leveraging somatic mosaicism and clonal hematopoiesis in its approach to precision medicine, aiming to address complex diseases such as cancer and cardiovascular conditions. By utilizing advanced DNA analysis, TenSixteen Bio seeks to discover and develop new treatments that can enhance the capabilities of pharmaceutical companies in managing these diseases. Co-founded by Foresite Labs, TenSixteen Bio is dedicated to transforming the future of healthcare through its unique therapeutic strategies.
Maze Therapeutics is a biotechnology company based in South San Francisco, California, that specializes in the development of genetic modifier therapeutics. Founded in 2017 and formerly known as Modulus Therapeutics, the company is dedicated to translating genetic insights into innovative medicines. Maze Therapeutics utilizes a comprehensive approach that integrates large-scale human genetics data, functional genomics, and various drug discovery methodologies. This approach allows the company to identify modifier genes that offer protection against diseases, enhancing the understanding of target biology and informing drug development strategies. By leveraging these insights, Maze Therapeutics aims to facilitate the creation of novel therapies that can benefit patients.
Mammoth Biosciences, Inc. is a biotechnology company based in South San Francisco, California, founded in 2017. It specializes in developing advanced CRISPR solutions, including its proprietary DETECTR technology, which allows for the detection of specific nucleic acids in samples, aiding in diagnostics for various conditions such as bacterial infections, cancer, and viral infections. The company focuses on democratizing disease detection through accessible point-of-care tests that can simultaneously identify multiple health issues. Additionally, Mammoth offers CRISPR-Cas systems for genome editing, targeting therapeutic applications in immuno-oncology, autoimmune diseases, and liver diseases. Its innovative solutions are utilized across diverse sectors, including healthcare, agriculture, environmental monitoring, and biodefense. Co-founded by CRISPR pioneer Jennifer Doudna, Mammoth Biosciences is supported by notable institutional and individual investors, reflecting its commitment to harnessing CRISPR technology for significant advancements in life sciences.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
23andMe, Inc. is a consumer genetics company that provides DNA analysis services through home-based saliva collection kits, enabling users to explore their ancestry, inherited traits, and health insights. Founded in 2006 and headquartered in Sunnyvale, California, the company offers various services, including Health + Ancestry and Ancestry + Traits, which provide insights into a person's genetic background and over 30 trait reports. 23andMe aims to help individuals understand the implications of their genetic information while also catering to researchers by offering categorized and searchable data. The company was co-founded by Linda Avey and Anne Wojcicki and has positioned itself as a significant player in the growing biotechnology and healthcare sectors.
Interline Therapeutics is a drug discovery platform based in South San Francisco, California, founded in 2020 by Zach Sweeney. The company specializes in mapping and correcting dysfunctional protein communities to elucidate the molecular mechanisms of disease. By utilizing advancements in genomics, proteomics, structural biology, and computational chemistry, Interline aims to develop a precision medicine approach that focuses on three key areas: genomics, communities, and modulators. This innovative methodology enables the identification of new medicines targeting genetically validated signaling pathways and ensures that drug candidates effectively address dysfunctional disease networks. Through collaborations with leading academic institutions, Interline Therapeutics strives to enhance drug discovery and improve therapeutic outcomes.
Inscripta, Inc. is a gene editing technology company focused on developing advanced CRISPR enzymes, specifically MADzymes, for precision gene editing. Founded in 2015 and headquartered in Boulder, Colorado, with additional offices in Pleasanton and San Diego, Inscripta aims to revolutionize genome engineering through its innovative benchtop platform. This platform integrates an instrument, consumables, software, and assays to facilitate fully automated and scalable editing of single cells, enabling researchers to conduct massively parallel experiments with unprecedented efficiency. By offering tools that simplify the editing process and making its MAD7 CRISPR nuclease available for research, Inscripta seeks to empower scientists in overcoming existing technical and licensing barriers, fostering a new era of biological discovery.
Interline Therapeutics is a drug discovery platform based in South San Francisco, California, founded in 2020 by Zach Sweeney. The company specializes in mapping and correcting dysfunctional protein communities to elucidate the molecular mechanisms of disease. By utilizing advancements in genomics, proteomics, structural biology, and computational chemistry, Interline aims to develop a precision medicine approach that focuses on three key areas: genomics, communities, and modulators. This innovative methodology enables the identification of new medicines targeting genetically validated signaling pathways and ensures that drug candidates effectively address dysfunctional disease networks. Through collaborations with leading academic institutions, Interline Therapeutics strives to enhance drug discovery and improve therapeutic outcomes.
Genomics PLC is focused on advancing the use of genomic data to transform healthcare. The company develops algorithms and software solutions that facilitate the analysis of large genomic and phenotypic datasets, aimed at understanding human biology and improving disease diagnosis and treatment. Its offerings serve a diverse range of clients, including governments, healthcare providers, pharmaceutical companies, and research laboratories. By providing tools that enhance the accuracy of genetic analysis, Genomics PLC helps de-risk the drug development process and enables clinicians to better identify individuals at risk for specific diseases. Founded in 2014 and based in Oxford, United Kingdom, the company aims to set standards in genomic data application, ultimately benefiting organizations and patients alike through innovative healthcare solutions.
Gemini Therapeutics, Inc. is a precision medicine company established in 2015 and based in Cambridge, Massachusetts. The company specializes in developing therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini's approach focuses on addressing genetically-defined conditions by creating therapeutic candidates that target specific molecular abnormalities prevalent in patients with significant clinical needs. Their diverse pipeline features a range of innovative treatment modalities, including monoclonal antibodies, recombinant proteins, and gene therapies. The company has secured funding from prominent life science investors and collaborates with academic institutions globally to advance its mission of delivering potentially first-in-class therapeutics.
Mammoth Biosciences, Inc. is a biotechnology company based in South San Francisco, California, founded in 2017. It specializes in developing advanced CRISPR solutions, including its proprietary DETECTR technology, which allows for the detection of specific nucleic acids in samples, aiding in diagnostics for various conditions such as bacterial infections, cancer, and viral infections. The company focuses on democratizing disease detection through accessible point-of-care tests that can simultaneously identify multiple health issues. Additionally, Mammoth offers CRISPR-Cas systems for genome editing, targeting therapeutic applications in immuno-oncology, autoimmune diseases, and liver diseases. Its innovative solutions are utilized across diverse sectors, including healthcare, agriculture, environmental monitoring, and biodefense. Co-founded by CRISPR pioneer Jennifer Doudna, Mammoth Biosciences is supported by notable institutional and individual investors, reflecting its commitment to harnessing CRISPR technology for significant advancements in life sciences.
Sestina Bio, LLC is a biotechnology company founded in 2020 and based in Delaware. Formerly known as F-SynBio, the company focuses on developing a technology-centric research and development platform aimed at addressing significant global challenges. Sestina Bio seeks to provide innovative solutions to sustainably feed, clothe, and maintain the health of the growing world population. The company integrates cutting-edge advancements in synthetic biology, data sciences, and bio-analytical systems to achieve its mission.
DNAnexus, Inc. specializes in cloud-based genome informatics and data management tools tailored for enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company provides a platform that facilitates the analysis of raw sequencing data, integrates genomic data with clinical information, and enhances collaboration among researchers. Key offerings include the MOSAIC Microbiome platform for microbiome research, DNAnexus Apollo for clinico-genomic data exploration, DNAnexus Titan for data analysis, and DNAnexus Portals, which create secure online workspaces for collaborative projects. Additionally, the Clinico-Genomic Data Solution aims to enhance disease-specific datasets through partnerships with healthcare providers, improving screening and treatment pathways for complex diseases. DNAnexus serves a diverse clientele, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.
Evonetix Ltd is a biotechnology company based in Little Chesterford, United Kingdom, established in 2015. The company focuses on developing innovative technology for the parallel synthesis of DNA on silicon arrays, thereby advancing the field of synthetic biology. By employing novel techniques, Evonetix aims to synthesize DNA with higher accuracy and scale, which enhances accessibility for researchers. This platform enables scientists to utilize synthetic biology on a previously unattainable scale, fundamentally changing how DNA is accessed, produced, and utilized for gene synthesis. The engineering team at Evonetix boasts extensive experience in creating high-integrity automated systems, bringing together a diverse range of expertise from various scientific and engineering disciplines to support its mission.
Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing gene therapies aimed at treating both rare and prevalent diseases. Established in 2016, the company focuses on conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs an innovative non-viral platform that includes a proprietary high-capacity DNA construct known as closed-ended DNA (ceDNA), a cell-targeted lipid nanoparticle delivery system (ctLNP), and a scalable, capsid-free manufacturing process. This approach allows for the targeted delivery of genetic materials, including large and multiple genes across various tissues, and enables redosable treatments tailored to individual patient needs. By enhancing access to therapies for rare diseases and addressing more common conditions, Generation Bio aims to provide durable and sustainable treatment options for patients throughout their lives.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
Kinnate Biopharma develops precision oncology therapeutics to treat patients with genomically-defined cancers. Its mission is to expand the reach of targeted therapeutics by developing products for underserved populations.
The company utilizes its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine. Its programs include candidates in preclinical development for cancers that are driven by specific oncogenic alterations in either the BRAF kinase gene or in the FGFR2 and FGFR3 kinase genes.
GenapSys, Inc. develops innovative DNA sequencing technologies for applied genomic testing and medical sequencing. The company's primary product, the GenapSys Sequencer, utilizes electrical-based detection for single nucleotide incorporations, facilitating sequencing in various laboratory settings and automating clonal amplification. GenapSys offers solutions for a wide range of applications, including research, gene editing, drug development, agriculture, forensics, and food testing. Additionally, the company seeks to collaborate with local health agencies and researchers to enhance access to sequencing insights, particularly for controlling and mitigating outbreaks. The compact and portable nature of the sequencer allows it to operate from standard power sources, making it suitable for deployment in hospitals, airports, and public transportation hubs to provide rapid results for virus samples. Founded in 2010, GenapSys is headquartered in Redwood City, California.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
Inscripta, Inc. is a gene editing technology company focused on developing advanced CRISPR enzymes, specifically MADzymes, for precision gene editing. Founded in 2015 and headquartered in Boulder, Colorado, with additional offices in Pleasanton and San Diego, Inscripta aims to revolutionize genome engineering through its innovative benchtop platform. This platform integrates an instrument, consumables, software, and assays to facilitate fully automated and scalable editing of single cells, enabling researchers to conduct massively parallel experiments with unprecedented efficiency. By offering tools that simplify the editing process and making its MAD7 CRISPR nuclease available for research, Inscripta seeks to empower scientists in overcoming existing technical and licensing barriers, fostering a new era of biological discovery.
Inscripta, Inc. is a gene editing technology company focused on developing advanced CRISPR enzymes, specifically MADzymes, for precision gene editing. Founded in 2015 and headquartered in Boulder, Colorado, with additional offices in Pleasanton and San Diego, Inscripta aims to revolutionize genome engineering through its innovative benchtop platform. This platform integrates an instrument, consumables, software, and assays to facilitate fully automated and scalable editing of single cells, enabling researchers to conduct massively parallel experiments with unprecedented efficiency. By offering tools that simplify the editing process and making its MAD7 CRISPR nuclease available for research, Inscripta seeks to empower scientists in overcoming existing technical and licensing barriers, fostering a new era of biological discovery.
Maze Therapeutics is a biotechnology company based in South San Francisco, California, that specializes in the development of genetic modifier therapeutics. Founded in 2017 and formerly known as Modulus Therapeutics, the company is dedicated to translating genetic insights into innovative medicines. Maze Therapeutics utilizes a comprehensive approach that integrates large-scale human genetics data, functional genomics, and various drug discovery methodologies. This approach allows the company to identify modifier genes that offer protection against diseases, enhancing the understanding of target biology and informing drug development strategies. By leveraging these insights, Maze Therapeutics aims to facilitate the creation of novel therapies that can benefit patients.
DNAnexus, Inc. specializes in cloud-based genome informatics and data management tools tailored for enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company provides a platform that facilitates the analysis of raw sequencing data, integrates genomic data with clinical information, and enhances collaboration among researchers. Key offerings include the MOSAIC Microbiome platform for microbiome research, DNAnexus Apollo for clinico-genomic data exploration, DNAnexus Titan for data analysis, and DNAnexus Portals, which create secure online workspaces for collaborative projects. Additionally, the Clinico-Genomic Data Solution aims to enhance disease-specific datasets through partnerships with healthcare providers, improving screening and treatment pathways for complex diseases. DNAnexus serves a diverse clientele, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.
Inscripta, Inc. is a gene editing technology company focused on developing advanced CRISPR enzymes, specifically MADzymes, for precision gene editing. Founded in 2015 and headquartered in Boulder, Colorado, with additional offices in Pleasanton and San Diego, Inscripta aims to revolutionize genome engineering through its innovative benchtop platform. This platform integrates an instrument, consumables, software, and assays to facilitate fully automated and scalable editing of single cells, enabling researchers to conduct massively parallel experiments with unprecedented efficiency. By offering tools that simplify the editing process and making its MAD7 CRISPR nuclease available for research, Inscripta seeks to empower scientists in overcoming existing technical and licensing barriers, fostering a new era of biological discovery.
Genomics PLC is focused on advancing the use of genomic data to transform healthcare. The company develops algorithms and software solutions that facilitate the analysis of large genomic and phenotypic datasets, aimed at understanding human biology and improving disease diagnosis and treatment. Its offerings serve a diverse range of clients, including governments, healthcare providers, pharmaceutical companies, and research laboratories. By providing tools that enhance the accuracy of genetic analysis, Genomics PLC helps de-risk the drug development process and enables clinicians to better identify individuals at risk for specific diseases. Founded in 2014 and based in Oxford, United Kingdom, the company aims to set standards in genomic data application, ultimately benefiting organizations and patients alike through innovative healthcare solutions.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
Kinnate Biopharma develops precision oncology therapeutics to treat patients with genomically-defined cancers. Its mission is to expand the reach of targeted therapeutics by developing products for underserved populations.
The company utilizes its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine. Its programs include candidates in preclinical development for cancers that are driven by specific oncogenic alterations in either the BRAF kinase gene or in the FGFR2 and FGFR3 kinase genes.
Inscripta, Inc. is a gene editing technology company focused on developing advanced CRISPR enzymes, specifically MADzymes, for precision gene editing. Founded in 2015 and headquartered in Boulder, Colorado, with additional offices in Pleasanton and San Diego, Inscripta aims to revolutionize genome engineering through its innovative benchtop platform. This platform integrates an instrument, consumables, software, and assays to facilitate fully automated and scalable editing of single cells, enabling researchers to conduct massively parallel experiments with unprecedented efficiency. By offering tools that simplify the editing process and making its MAD7 CRISPR nuclease available for research, Inscripta seeks to empower scientists in overcoming existing technical and licensing barriers, fostering a new era of biological discovery.
Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing gene therapies aimed at treating both rare and prevalent diseases. Established in 2016, the company focuses on conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs an innovative non-viral platform that includes a proprietary high-capacity DNA construct known as closed-ended DNA (ceDNA), a cell-targeted lipid nanoparticle delivery system (ctLNP), and a scalable, capsid-free manufacturing process. This approach allows for the targeted delivery of genetic materials, including large and multiple genes across various tissues, and enables redosable treatments tailored to individual patient needs. By enhancing access to therapies for rare diseases and addressing more common conditions, Generation Bio aims to provide durable and sustainable treatment options for patients throughout their lives.
DNAnexus, Inc. specializes in cloud-based genome informatics and data management tools tailored for enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company provides a platform that facilitates the analysis of raw sequencing data, integrates genomic data with clinical information, and enhances collaboration among researchers. Key offerings include the MOSAIC Microbiome platform for microbiome research, DNAnexus Apollo for clinico-genomic data exploration, DNAnexus Titan for data analysis, and DNAnexus Portals, which create secure online workspaces for collaborative projects. Additionally, the Clinico-Genomic Data Solution aims to enhance disease-specific datasets through partnerships with healthcare providers, improving screening and treatment pathways for complex diseases. DNAnexus serves a diverse clientele, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
Inscripta, Inc. is a gene editing technology company focused on developing advanced CRISPR enzymes, specifically MADzymes, for precision gene editing. Founded in 2015 and headquartered in Boulder, Colorado, with additional offices in Pleasanton and San Diego, Inscripta aims to revolutionize genome engineering through its innovative benchtop platform. This platform integrates an instrument, consumables, software, and assays to facilitate fully automated and scalable editing of single cells, enabling researchers to conduct massively parallel experiments with unprecedented efficiency. By offering tools that simplify the editing process and making its MAD7 CRISPR nuclease available for research, Inscripta seeks to empower scientists in overcoming existing technical and licensing barriers, fostering a new era of biological discovery.
10X Genomics is a life science technology company that specializes in developing and selling instruments, consumables, and software for analyzing biological systems. Headquartered in Pleasanton, California, the company offers a range of products, including chromium instruments, microfluidic chips, reagents, and other consumables. Its advanced single-cell solutions allow researchers to measure gene expression, immune profiling, epigenetics, and cellular heterogeneity, providing insights into the physical organization of DNA and gene activity on a cell-by-cell basis. Additionally, the Visium spatial gene expression solution enables the measurement of spatial gene expression patterns across tissue samples. 10X Genomics serves a diverse clientele, including academic institutions, government agencies, and biopharmaceutical companies, and has a presence in North America, Europe, the Middle East, Africa, China, and the Asia Pacific. The company was founded in 2012 and rebranded from 10X Technologies, Inc. in November 2014.
Twist Bioscience Corporation, a synthetic biology company, manufactures and sells synthetic DNA-based products. The company’s DNA synthesis platform enables the manufacturing of synthetic DNA by writing DNA on a silicon chip. It offers synthetic DNA-based products, including synthetic genes, tools for sample preparation, antibody libraries for drug discovery and development, and DNA as a digital data storage medium. The company has a collaboration agreement with ImmunoPrecise Antibodies Ltd. Twist Bioscience Corporation was founded in 2013 and is headquartered in South San Francisco, California.
Twist Bioscience Corporation, a synthetic biology company, manufactures and sells synthetic DNA-based products. The company’s DNA synthesis platform enables the manufacturing of synthetic DNA by writing DNA on a silicon chip. It offers synthetic DNA-based products, including synthetic genes, tools for sample preparation, antibody libraries for drug discovery and development, and DNA as a digital data storage medium. The company has a collaboration agreement with ImmunoPrecise Antibodies Ltd. Twist Bioscience Corporation was founded in 2013 and is headquartered in South San Francisco, California.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
Based in Dallas, Texas, AveXis is a clinic-ready, synthetic biology platform company. AveXis has, at its core, a desire to establish unique industry and research alliances which will bring innovative treatments to people with unmet medical needs. Our work in spinal muscular atrophy (SMA), a rare/orphan disease, is our first focus.
Intellia Therapeutics is a genome editing company based in Cambridge, Massachusetts, focused on developing curative gene-editing treatments using the CRISPR/Cas9 technology. The company aims to address severe and life-threatening genetic diseases through both in vivo and ex vivo therapeutic approaches. Its pipeline includes programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia is developing engineered cell therapies for oncological and autoimmune diseases. The company has established collaborations with various entities, including leading pharmaceutical firms and research institutions, to enhance its research and development efforts. Founded in 2014, Intellia Therapeutics holds a comprehensive intellectual property platform for the therapeutic application of CRISPR technology, positioning it at the forefront of gene editing innovation.
WaVe Life Sciences Ltd. is a clinical stage genetic medicine company focused on developing novel stereopure oligonucleotides through its proprietary PRISM platform. The company aims to address genetic defects by either reducing the expression of harmful proteins or converting dysfunctional mutant proteins into functional ones. It primarily concentrates on neurological disorders affecting the central and neuromuscular systems. WaVe Life Sciences has established research and collaboration agreements with major pharmaceutical companies to advance its oligonucleotide therapeutics. Founded in 2012, the company is headquartered in Singapore and maintains research facilities in Boston and Japan. The organization was established by leading scientists in the field of chemistry and life sciences, underscoring its commitment to innovative approaches in nucleic acid therapeutics.
Twist Bioscience Corporation, a synthetic biology company, manufactures and sells synthetic DNA-based products. The company’s DNA synthesis platform enables the manufacturing of synthetic DNA by writing DNA on a silicon chip. It offers synthetic DNA-based products, including synthetic genes, tools for sample preparation, antibody libraries for drug discovery and development, and DNA as a digital data storage medium. The company has a collaboration agreement with ImmunoPrecise Antibodies Ltd. Twist Bioscience Corporation was founded in 2013 and is headquartered in South San Francisco, California.
REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing gene therapy product candidates that utilize its proprietary NAV Technology Platform, which is based on adeno-associated virus (AAV) gene delivery. This platform aims to address genetic defects and enable cells to produce therapeutic proteins or antibodies to combat various diseases. The company's lead candidate, RGX-314, is currently undergoing Phase I/IIa clinical trials for wet age-related macular degeneration. Other notable product candidates include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia, all in various stages of clinical trials. Additionally, REGENXBIO licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has partnered with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.
10X Genomics is a life science technology company that specializes in developing and selling instruments, consumables, and software for analyzing biological systems. Headquartered in Pleasanton, California, the company offers a range of products, including chromium instruments, microfluidic chips, reagents, and other consumables. Its advanced single-cell solutions allow researchers to measure gene expression, immune profiling, epigenetics, and cellular heterogeneity, providing insights into the physical organization of DNA and gene activity on a cell-by-cell basis. Additionally, the Visium spatial gene expression solution enables the measurement of spatial gene expression patterns across tissue samples. 10X Genomics serves a diverse clientele, including academic institutions, government agencies, and biopharmaceutical companies, and has a presence in North America, Europe, the Middle East, Africa, China, and the Asia Pacific. The company was founded in 2012 and rebranded from 10X Technologies, Inc. in November 2014.
Sequenta is a biotech company focused on the discovery and development of clinical diagnostics that leverage a novel platform for understanding immune system status. The advancements in DNA sequencing technology have enabled Sequenta to create innovative methods for measuring the immune cell receptor genes, which exhibit significant variability and play a crucial role in numerous health conditions. This capability positions the company to inform a wide range of clinical decisions through a single, powerful assay. Founded in 2008 by entrepreneurs with previous experience in the biotech sector, Sequenta aims to enhance the understanding of the immune system and improve diagnostic approaches in healthcare.