hC Bioscience is an innovator and pioneer of protein editing using context agnostic engineered tRNA. Their First in Class approach for protein editing advances beyond genome editing to direct modification of the proteome.
Generate Biomedicines, Inc. specializes in developing a generative biology platform aimed at inventing novel therapeutic proteins, including antibodies, peptides, enzymes, and other related compounds. Utilizing advanced machine learning techniques, the platform analyzes the genetic code that determines protein function, allowing for the creation of new protein sequences optimized for therapeutic applications. This innovative approach enables the company to generate proteins that interact specifically and effectively with targeted therapeutic needs, facilitating the development of new drugs across various protein modalities. Founded in 2018 and located in Cambridge, Massachusetts, Generate Biomedicines was previously known as Generate Biologics, Inc. until its name change in March 2020.
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.
Jaguar Gene Therapy is a clinical-stage biotechnology company focused on advancing gene therapy solutions for patients with severe genetic diseases. The company aims to address significant unmet medical needs by developing therapies that target nonworking or absent genes, introducing healthy copies of these genes to restore their function. By accelerating the process of bringing innovative gene therapies from research to clinical application, Jaguar Gene Therapy seeks to improve patient outcomes and enhance the overall standard of care for those affected by genetic disorders.
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.
Excision BioTherapeutics Inc. is a life science company based in Philadelphia, Pennsylvania, that specializes in the development of gene editing therapeutics targeting life-threatening diseases caused by neurotropic viruses. Founded in 2015, the company employs CRISPR-based technology to create innovative treatments that aim to eradicate or disrupt viral genes in human patients. Excision is committed to advancing these therapeutics into safe and effective medicines, addressing significant medical needs and improving the quality of life for individuals affected by viral infections.
Encodia, Inc. engages in proteomics research and creates scalable and parallelized approaches to protein analysis. The company was founded in 2015 and is based in San Diego, California.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
Omniome, Inc. is a biotechnology company based in San Diego, California, established in 2013. It specializes in developing a proprietary DNA sequencing platform that aims to deliver high accuracy in sequencing results. The company employs its Sequencing By Binding technology, which enhances nucleotide and DNA matching by utilizing the natural matching capabilities of polymerase. This innovative approach not only provides precise and rapid results but also offers a cost-effective solution, which has the potential to significantly advance cancer diagnostics. Omniome is backed by prominent life sciences venture investors and is focused on becoming a trusted leader in the field of clinical sequencing.
EGenesis Inc. is a biotechnology company based in Cambridge, Massachusetts, with an additional office in New York. Founded in 2015, the company specializes in gene editing and genome engineering, focusing on the development of human-compatible organs, tissues, and cells for transplantation. EGenesis employs advanced gene editing techniques to create transplantable cells and solid organs, specifically targeting therapeutic applications in kidney and islet cell transplantation. By leveraging its innovative platform, the company aims to transform the field of organ transplantation, providing safe and effective solutions for patients with life-threatening diseases globally.
Prime Medicine Inc is a biotechnology company committed to delivering genetic therapies to address diseases by deploying gene editing technology, Prime Editing. The Prime Editing technology is a next-generation technology that can search and replace to restore normal genetic function in the genome and can treat a wide spectrum of diseases with high unmet medical needs and efficient and broad gene editing technology.
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.
LunaPBC, Inc. operates a community-owned platform focused on health and DNA research. The company offers LunaDNA, a database that facilitates the sharing of health and genetic data for medical research. This platform supports health research initiatives by both non-profit and for-profit organizations, contributing to medical discoveries. LunaPBC operates as a Public Benefit Corporation, emphasizing community welfare by allowing individuals to share their health data, which in turn drives research and innovation. Additionally, members of the platform have the opportunity to share in the earnings derived from medical advancements, promoting a sense of shared ownership and benefit within the community. Founded in 2017 and based in San Diego, California, LunaPBC aims to advance genomic and medical research while prioritizing the interests of its contributors.
LunaDNA is a community-owned platform that facilitates the sharing of health and DNA data for medical research. Founded by the creators of Illumina, a leader in DNA sequencing, LunaDNA addresses the issue of siloed genomic data by providing individuals with a compelling incentive to share their health information. Participants are rewarded with shares in the database, allowing them to benefit from the profits generated by medical research and discoveries. This model not only empowers users to contribute their health records, genomic data, and personal health experiences but also fosters a sense of community ownership. As researchers pay to access the data for their studies, the proceeds are distributed back to the community, akin to dividends. By prioritizing privacy and trust, LunaDNA aims to increase participation in genomic research while facilitating advancements in healthcare that can ultimately save lives.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. This technology allows for targeted alterations of single bases in the genome without creating double-stranded breaks in DNA, representing a potential advancement in genetic therapies. The company's pipeline includes programs aimed at treating serious diseases such as sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, and pediatric acute myeloid leukemia, as well as conditions like alpha-1 antitrypsin deficiency and certain central nervous system disorders. Beam Therapeutics is committed to providing life-long cures through its various approaches, which include gene correction, gene modification, gene activation, gene silencing, and multiplex editing. Founded in 2017, Beam continues to build collaborations to enhance its research and development efforts.
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.
KSQ Therapeutics is pioneering High-Confidence Drug Development: a fundamentally new approach to R&D where the majority of the de-risking work is done prior to the generation of therapeutic drug candidates. Powered by our proprietary CRISPRomics™ drug discovery engine, their team has elucidated the function that each human gene plays in multiple diseases, providing a unique and more comprehensive understanding of disease biology. The quality of these insights enables their scientists to rapidly identify and validate high-confidence, patient-tailored, novel drug targets and then focus their collective efforts on the development of medicines with the greatest potential to impact the lives of patients. KSQ has initiated a pipeline of preclinical discovery programs and was founded in 2015.
Omniome, Inc. is a biotechnology company based in San Diego, California, established in 2013. It specializes in developing a proprietary DNA sequencing platform that aims to deliver high accuracy in sequencing results. The company employs its Sequencing By Binding technology, which enhances nucleotide and DNA matching by utilizing the natural matching capabilities of polymerase. This innovative approach not only provides precise and rapid results but also offers a cost-effective solution, which has the potential to significantly advance cancer diagnostics. Omniome is backed by prominent life sciences venture investors and is focused on becoming a trusted leader in the field of clinical sequencing.
Qihan Biotech is a biomedical company specializing in genetic technology with a focus on xenotransplantation. The company aims to address the critical shortage of organ donors by utilizing advanced gene editing techniques to develop safe and effective cells, tissues, and organs for human transplantation. Their mission is to eliminate the waiting period associated with organ transplants, providing patients with access to life-saving organs. By harnessing cutting-edge genetic editing technology, Qihan Biotech seeks to create viable organs that can be transplanted into humans, thereby contributing to a solution for the global organ donor crisis.
Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. This technology allows for targeted alterations of single bases in the genome without creating double-stranded breaks in DNA, representing a potential advancement in genetic therapies. The company's pipeline includes programs aimed at treating serious diseases such as sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, and pediatric acute myeloid leukemia, as well as conditions like alpha-1 antitrypsin deficiency and certain central nervous system disorders. Beam Therapeutics is committed to providing life-long cures through its various approaches, which include gene correction, gene modification, gene activation, gene silencing, and multiplex editing. Founded in 2017, Beam continues to build collaborations to enhance its research and development efforts.
LunaDNA is a community-owned platform that facilitates the sharing of health and DNA data for medical research. Founded by the creators of Illumina, a leader in DNA sequencing, LunaDNA addresses the issue of siloed genomic data by providing individuals with a compelling incentive to share their health information. Participants are rewarded with shares in the database, allowing them to benefit from the profits generated by medical research and discoveries. This model not only empowers users to contribute their health records, genomic data, and personal health experiences but also fosters a sense of community ownership. As researchers pay to access the data for their studies, the proceeds are distributed back to the community, akin to dividends. By prioritizing privacy and trust, LunaDNA aims to increase participation in genomic research while facilitating advancements in healthcare that can ultimately save lives.
Biopalette is a operator of a platform intended to offer gene-editing techniques. Bio Palette plans to develop our business in three fields, classified based on the type of cells targeted for genome editing.
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
AnchorDx is a company focused on advancing precision medicine through its development of next-generation sequencing techniques, particularly utilizing molecular liquid biopsies. This approach offers non-invasive access to genetic information, enabling patients to gain insights into their physiological conditions and prepare for potential future health issues. With a core team boasting over 20 years of experience in cancer genomics, genetics, and bioinformatics, AnchorDx aims to establish itself as a leading global provider of precision medical solutions. The company collaborates with various middle and downstream enterprises, fostering a comprehensive industrial chain that spans from academic research to clinical product transformation. Additionally, AnchorDx has formed partnerships with numerous large-scale third-party inspection agencies, hospitals, and research institutes, contributing to the development of independent intellectual property rights and competitive clinical testing products that deliver accurate and comprehensive services to patients, doctors, and medical institutions.
KSQ Therapeutics is pioneering High-Confidence Drug Development: a fundamentally new approach to R&D where the majority of the de-risking work is done prior to the generation of therapeutic drug candidates. Powered by our proprietary CRISPRomics™ drug discovery engine, their team has elucidated the function that each human gene plays in multiple diseases, providing a unique and more comprehensive understanding of disease biology. The quality of these insights enables their scientists to rapidly identify and validate high-confidence, patient-tailored, novel drug targets and then focus their collective efforts on the development of medicines with the greatest potential to impact the lives of patients. KSQ has initiated a pipeline of preclinical discovery programs and was founded in 2015.
Biopalette is a operator of a platform intended to offer gene-editing techniques. Bio Palette plans to develop our business in three fields, classified based on the type of cells targeted for genome editing.
EGenesis Inc. is a biotechnology company based in Cambridge, Massachusetts, with an additional office in New York. Founded in 2015, the company specializes in gene editing and genome engineering, focusing on the development of human-compatible organs, tissues, and cells for transplantation. EGenesis employs advanced gene editing techniques to create transplantable cells and solid organs, specifically targeting therapeutic applications in kidney and islet cell transplantation. By leveraging its innovative platform, the company aims to transform the field of organ transplantation, providing safe and effective solutions for patients with life-threatening diseases globally.
Boragen Inc. is a biotechnology company based in Durham, North Carolina, that specializes in designing and developing multifunctional small molecule boron solutions for applications in crop protection, animal health, and human health. Founded in 2015, the company focuses on creating next-generation fungicides through its synthetic chemistry platforms, which aim to promote sustainable farming practices. Boragen's product pipeline includes BAG8, a boron-based multisite fungicide, and BN2266, which targets mRNA processing as a crop protection fungicide. Additionally, the company is developing a dual-function nematicide-fungicide designed for root protection and endo-parasiticides that address resistance challenges. Through its innovative approach, Boragen seeks to enhance agricultural efficiency while minimizing chemical usage.
ApoGen Biotechnologies, Inc. is focused on the development of new class of therapeutics targeting drivers of cancer genomic mutation.As the arsenal of available cancer therapies has grown and evolved from cytotoxic chemotherapy to targeted therapy to immunotherapy, one fact has unfortunately remained constant nearly all cancer drug therapies eventually fail due to the development of drug resistance. Mutation of cellular DNA is central to the formation of cancer, and chronic mutation of the cancer genome is a primary cause of cancer evolution and drug resistance, resulting in ineffective therapy, cancer recurrence and metastasis, and decreased overall survival.
Genomics Medicine Ireland is a life sciences company focused on conducting extensive research studies throughout Ireland to explore the connections between the human genome, health, and disease. By analyzing genetic information, the company aims to deepen the understanding of how genetics influences various health outcomes and disease susceptibility. Through its research initiatives, Genomics Medicine Ireland seeks to contribute valuable insights that could inform future medical practices and enhance healthcare delivery.
Homology Medicines, Inc. is a genetic medicines company based in Bedford, Massachusetts, focused on improving the lives of patients with rare genetic diseases. The company employs a proprietary platform that utilizes human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies in vivo, either through gene therapy or nuclease-free gene editing. This innovative approach allows for targeted treatment of various disease-relevant tissues, including the liver and central nervous system, through a single injection. Homology's lead product candidate, HMI-102, is currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, while HMI-103 targets pediatric PKU patients, and HMI-202 is being developed for metachromatic leukodystrophy. Founded in 2015, Homology Medicines aims to address significant unmet medical needs by targeting the underlying causes of genetic disorders.
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
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.
Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.
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.
NextCODE Health LLC is a clinical diagnostics company specializing in sequence-based genomics. It offers a platform that assists healthcare professionals in obtaining clinical insights for rapid disease diagnosis. The company provides a range of services, including data analysis, genome interpretation, data mining, and sequencing services, both clinical and non-clinical. Founded on technology developed at deCODE genetics over 16 years, NextCODE focuses on integrating genome sequence data into patient care. Based in Cambridge, Massachusetts, the company was acquired by WuXi PharmaTech in 2015 and underwent a rebranding to Genuity Science in 2020. Through its innovative solutions, NextCODE aims to enhance the speed and accuracy of identifying genetic causes of diseases.
Genuity Science is a contract genomics and data-sourcing organization based in Boston, Massachusetts, with additional offices in Dublin, Ireland, and Reykjavik, Iceland. The company collaborates with global biopharma firms to provide comprehensive discovery services that enhance precision health and improve patient outcomes. Its offerings encompass population-scale, disease-specific data sourcing, high-quality sequencing, and advanced statistical analysis, along with software tools for managing large datasets and artificial intelligence applications. Genuity operates state-of-the-art CAP/CLIA genomics laboratories in Woburn, Massachusetts, and Dublin, emphasizing a strong commitment to data stewardship and governance. The company focuses on exploring biological data insights, storage, interpretation, and scalable analytics, particularly in the central nervous system, cardiometabolic, and inflammatory disease sectors.
Boreal Genomics Inc. is a biotechnology company that focuses on the development and commercialization of technologies for blood-based detection and monitoring of circulating tumor DNA. Founded in 2007 and headquartered in Los Altos, California, with an additional office in Vancouver, Canada, the company offers products like OnTarget, which allows for the sensitive detection and quantification of tumor mutations in plasma or tissue. Boreal's platform is designed to enable healthcare professionals to perform accurate genomic profiling and monitor tumor evolution from cell-free DNA in plasma, thereby improving cancer patient care. The OnTarget assay, available as a research-use service, reveals tumor mutations that may be undetectable by other methods, assisting in therapy selection, enhancing clinical trial efficiency, and expediting drug development. Additionally, Boreal provides DNA enrichment and library construction solutions tailored for high accuracy sequencing in liquid biopsy applications.
Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.
Bluebird Bio is a clinical-stage biotechnology company focused on developing transformative gene therapies for severe genetic diseases and cancer. The company is engaged in researching and commercializing therapies that aim to genetically modify patients' cells to correct the underlying genetic causes of diseases. Key programs include LentiGlobin for β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing CAR T cell therapies, such as bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with Bristol-Myers Squibb and Regeneron Pharmaceuticals to advance gene therapies in oncology, and works with various partners to develop innovative solutions in cancer treatment and in vivo genome editing for genetic disorders. Founded in 1992 and headquartered in Cambridge, Massachusetts, bluebird bio generates revenue through collaboration arrangements, research fees, license fees, and grants.
Agios Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of therapies targeting cellular metabolism, particularly in cancer and rare genetic disorders. The company offers TIBSOVO, an oral targeted inhibitor approved for treating relapsed or refractory acute myeloid leukemia (AML) and newly diagnosed AML, as well as IDHIFA, which targets AML patients with specific genetic mutations. Agios is advancing multiple clinical programs, including TIBSOVO for frontline AML and cholangiocarcinoma, and developing mitapivat for pyruvate kinase deficiency and thalassemia. Other investigational compounds include vorasidenib for solid tumors and AG-270 for specific cancer types. Agios employs a precision medicine approach, focusing on genetically or biomarker-defined patient populations to enhance the potential for successful early clinical proof of concept and accelerated approvals. The company was originally established in 2007 under the name Cancer Metabolism Therapeutics before rebranding to Agios Pharmaceuticals in 2008.
Bluebird Bio is a clinical-stage biotechnology company focused on developing transformative gene therapies for severe genetic diseases and cancer. The company is engaged in researching and commercializing therapies that aim to genetically modify patients' cells to correct the underlying genetic causes of diseases. Key programs include LentiGlobin for β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing CAR T cell therapies, such as bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with Bristol-Myers Squibb and Regeneron Pharmaceuticals to advance gene therapies in oncology, and works with various partners to develop innovative solutions in cancer treatment and in vivo genome editing for genetic disorders. Founded in 1992 and headquartered in Cambridge, Massachusetts, bluebird bio generates revenue through collaboration arrangements, research fees, license fees, and grants.
Elixir Pharmaceuticals is a pharmaceutical company focused on the discovery, development and commercialization of novel pharmaceuticals for the treatment of metabolic diseases such as diabetes and obesity. The Company's scientific founders identified that interactions between specific genes and enzymes can slow the aging process, and they are developing compounds that stimulate these interactions and will be used to treat a range of diseases of aging, including metabolic disease.
Agios Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of therapies targeting cellular metabolism, particularly in cancer and rare genetic disorders. The company offers TIBSOVO, an oral targeted inhibitor approved for treating relapsed or refractory acute myeloid leukemia (AML) and newly diagnosed AML, as well as IDHIFA, which targets AML patients with specific genetic mutations. Agios is advancing multiple clinical programs, including TIBSOVO for frontline AML and cholangiocarcinoma, and developing mitapivat for pyruvate kinase deficiency and thalassemia. Other investigational compounds include vorasidenib for solid tumors and AG-270 for specific cancer types. Agios employs a precision medicine approach, focusing on genetically or biomarker-defined patient populations to enhance the potential for successful early clinical proof of concept and accelerated approvals. The company was originally established in 2007 under the name Cancer Metabolism Therapeutics before rebranding to Agios Pharmaceuticals in 2008.
PhaseRx is a biopharmaceutical company based in Seattle that focuses on the development of innovative therapies for inherited enzyme deficiencies in the liver. Utilizing its proprietary Hybrid messenger RNA technology platform, PhaseRx aims to provide intracellular enzyme replacement therapy (i-ERT) by enabling the synthesis of essential enzymes directly within cells. The company is developing a portfolio of products targeting specific urea cycle disorders, including ornithine transcarbamylase deficiency, argininosuccinate lyase deficiency, and argininosuccinate synthetase deficiency. Founded by a team with expertise from the University of Washington and Fred Hutchinson Cancer Research Center, PhaseRx leverages novel polymer technology for the delivery of therapeutic agents, including siRNA and other macromolecular therapeutics.
Elixir Pharmaceuticals is a pharmaceutical company focused on the discovery, development and commercialization of novel pharmaceuticals for the treatment of metabolic diseases such as diabetes and obesity. The Company's scientific founders identified that interactions between specific genes and enzymes can slow the aging process, and they are developing compounds that stimulate these interactions and will be used to treat a range of diseases of aging, including metabolic disease.
Elixir Pharmaceuticals is a pharmaceutical company focused on the discovery, development and commercialization of novel pharmaceuticals for the treatment of metabolic diseases such as diabetes and obesity. The Company's scientific founders identified that interactions between specific genes and enzymes can slow the aging process, and they are developing compounds that stimulate these interactions and will be used to treat a range of diseases of aging, including metabolic disease.
Alnylam Pharmaceuticals is a biopharmaceutical company specializing in the discovery, development, and commercialization of RNA interference (RNAi) therapeutics. Its innovative pipeline targets a range of serious conditions, including genetic disorders, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system and ocular diseases. The company has successfully brought several RNAi therapeutics to market, including ONPATTRO for hereditary transthyretin-mediated amyloidosis and GIVLAARI for acute hepatic porphyria. Additionally, Alnylam is advancing other investigational therapies, such as givosiran for acute hepatic porphyria and lumasiran for primary hyperoxaluria type 1. The company collaborates with various partners, including Sanofi Genzyme and Vir Biotechnology, to enhance its research and development efforts. Founded in 2002 and headquartered in Cambridge, Massachusetts, Alnylam Pharmaceuticals aims to transform treatment approaches for patients with rare and debilitating diseases through its RNAi technology.
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.
GenVec, Inc. is a clinical-stage biopharmaceutical company based in Gaithersburg, Maryland, specializing in the development of therapeutics and vaccines through its proprietary AdenoVerse gene delivery platform. The company's lead product candidate, CGF166, is currently undergoing Phase I/II clinical trials aimed at treating hearing loss and balance disorders. Additionally, GenVec is working on GV2311, a vaccine for respiratory syncytial virus, and GV2207, targeting herpes simplex virus. The company collaborates with prominent organizations, including Novartis and Merial, to advance its product pipeline, which encompasses various infectious diseases and animal health concerns. Notably, GenVec is also engaged with the National Institute of Allergy and Infectious Diseases and the Laboratory of Malaria Immunology and Vaccinology to develop malaria vaccine candidates. Founded in 1992, GenVec has established a diverse array of partnerships to support its mission of addressing significant health challenges through innovative gene-based medicine.