Developer of genetic medicines intended to treat cancer and other immune diseases. The company's medicines provide elegant cutting-edge in vivo gene delivery technology that uses potent lentiviral vector-like particles to precisely deliver in vivo genetic cargo to the desired target tissue, enabling physicians to get a pipeline of drugs for solid tumors and hematologic cancers and other immune diseases.
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.
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.
Encoded Therapeutics, Inc. is a biotechnology company focused on developing precision gene therapies for severe genetic disorders. The company's innovative platform identifies sequences within the human genome that regulate gene expression through advanced screening and computational methods. Its therapy pipeline targets a variety of genetic and acquired disorders across multiple disease pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and headquartered in South San Francisco, California, Encoded Therapeutics aims to enhance treatment options available to medical practitioners, ultimately improving patient outcomes and quality of life.
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.
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.
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.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
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.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
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.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
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.
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.
Aeglea BioTherapeutics is a clinical-stage biotechnology company focused on developing human enzyme therapeutics for rare genetic and cancer diseases. Its lead product candidate, pegzilarginase, is a recombinant human Arginase 1 enzyme currently undergoing Phase III trials to assess its safety and efficacy in treating Arginase 1 deficiency. The company's preclinical pipeline includes several innovative candidates: ACN00177 for homocystinuria, AEB5100 which degrades plasma cystine and cysteine, AEB2109 targeting the degradation of methionine, and AEB3103. Aeglea BioTherapeutics aims to exploit the amino acid dependencies of tumors by creating novel, human-derived enzymes that selectively target these vulnerabilities, differentiating the metabolic needs of tumor cells from those of normal tissues. Founded in 2013 and headquartered in Austin, Texas, the company was formerly known as Aeglea BioTherapeutics Holdings, LLC before changing its name in 2015.
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.
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.
Ariosa Diagnostics, based in San Jose, California, is a molecular diagnostics company specializing in prenatal testing for fetal health. The company offers blood tests that assess the risk of common fetal chromosome conditions, providing crucial information for expectant mothers. Utilizing proprietary technology, Ariosa Diagnostics enables targeted analysis of DNA, facilitating accurate and reliable assessments of fetal health risks.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
Pathwork Diagnostics, located in Redwood City, California, specializes in molecular diagnostics for oncology. The company is known for its Pathwork Tissue of Origin Test, which was the first microarray-based gene expression test to receive FDA clearance. This innovative test assists in identifying challenging tumors, including poorly differentiated, undifferentiated, and metastatic cancers. The test leverages genomic information from a tumor to enhance diagnostic precision and can be applied to various specimen types, including frozen tissue and formalin-fixed, paraffin-embedded samples. Through its advanced diagnostic solutions, Pathwork Diagnostics aims to improve clinical outcomes in cancer care.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
Ariosa Diagnostics, based in San Jose, California, is a molecular diagnostics company specializing in prenatal testing for fetal health. The company offers blood tests that assess the risk of common fetal chromosome conditions, providing crucial information for expectant mothers. Utilizing proprietary technology, Ariosa Diagnostics enables targeted analysis of DNA, facilitating accurate and reliable assessments of fetal health risks.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
Ariosa Diagnostics, based in San Jose, California, is a molecular diagnostics company specializing in prenatal testing for fetal health. The company offers blood tests that assess the risk of common fetal chromosome conditions, providing crucial information for expectant mothers. Utilizing proprietary technology, Ariosa Diagnostics enables targeted analysis of DNA, facilitating accurate and reliable assessments of fetal health risks.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
Pathwork Diagnostics, located in Redwood City, California, specializes in molecular diagnostics for oncology. The company is known for its Pathwork Tissue of Origin Test, which was the first microarray-based gene expression test to receive FDA clearance. This innovative test assists in identifying challenging tumors, including poorly differentiated, undifferentiated, and metastatic cancers. The test leverages genomic information from a tumor to enhance diagnostic precision and can be applied to various specimen types, including frozen tissue and formalin-fixed, paraffin-embedded samples. Through its advanced diagnostic solutions, Pathwork Diagnostics aims to improve clinical outcomes in cancer care.
Illumina is a leading company in the field of genomics, specializing in tools and services for analyzing genetic material with applications in life sciences and clinical laboratories. Founded in 1998 in San Diego, California, by David R. Walt, Satnam Alag, Larry Bock, and John Stuelpnagel, Illumina primarily generates its revenue from sequencing instruments, consumables, and associated services, accounting for over 90% of its sales. The company’s high-throughput technology facilitates whole genome sequencing in humans and other organisms, while its lower throughput tools are designed for specific applications such as viral and cancer tumor screening. Additionally, Illumina offers microarrays for cost-effective genetic screening, which are used mainly in consumer and agricultural contexts. Through initiatives like Illumina Accelerator, the company fosters innovation by supporting startups with mentorship and access to essential resources, thereby contributing to advancements in personalized medicine and improving human health.
Geron Corporation is a late-stage clinical biopharmaceutical company based in Menlo Park, California, specializing in the development and commercialization of therapeutics for hematologic myeloid malignancies. The company's primary focus is on imetelstat, a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. Imetelstat aims to inhibit the uncontrolled growth of malignant progenitor cells in these cancers, which helps reduce the production of dysfunctional blood cells and facilitates the recovery of normal blood cell production. Founded in 1990, Geron is dedicated to advancing innovative treatments for patients with serious blood disorders.
Vical's core technology grew out of a chance discovery and provided the cornerstones for two new biopharmaceutical industry sectors: DNA vaccines and non-viral gene therapy.
Vical's management systematically leveraged that technology into a broad portfolio of independent and partnered product development programs. The first products for animal health are on the market. The first human products are approaching.
Genetics Institute is a U.S. based biotechnology research and development company. The company developed drugs such as Neumega and Recombinate used in the prevention of severe reductions in the number of blood clotting cells. The company was founded in 1980 by Thomas Maniatis and Mark Ptashne, and started off by operating out of Ptashne’s home. Genetics Institute was acquired by Wyeth in 1992 and operates as part of Wyeth’s research division.