Developer of life sciences technology intended to develop highly effective and differentiated treatments for cystic fibrosis. The company is developing first-in-class small molecules that are focused on a unique target with the potential to fully normalize CFTR function for the first time for the vast majority of people living with cystic fibrosis, enabling patients with best-in-class efficacy and reach and long-term benefit for people with cystic fibrosis.
Arkuda Therapeutics is a biotechnology company leveraging new insights into progranulin and lysosomal biology to develop medicines to change the trajectory of neurodegenerative disease. Arkuda’s lead compounds aim to correct progranulin deficiency and lysosomal dysfunction in GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the GRN gene.
Vedere Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on developing gene therapy products aimed at restoring functional vision for patients who have experienced vision loss due to Inherited Retinal Degenerations (IRDs) and other causes of vision impairment, both genetic and non-genetic. Unlike many ocular gene therapies that primarily slow the progression of vision loss, Vedere Bio seeks to restore lost vision, regardless of the patient's genetic background or stage of their condition. As of September 2020, Vedere Bio operates as a subsidiary of Novartis AG.
Korro Bio Inc. specializes in the development of nucleic acid-based therapeutics aimed at targeting specific sequences in genetic code, with a focus on treating rare diseases. Founded in 2018 and located in Cambridge, Massachusetts, the company utilizes a proprietary platform that enables efficient and selective RNA editing by leveraging natural processes found in multi-cellular organisms. This innovative approach allows Korro Bio to edit messenger RNA and recode specific codons, facilitating targeted modifications in protein structure and function across various tissues.
Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It develops various programs for myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapies. The company was founded in 2017 and is based in Waltham, Massachusetts.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
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.
Chroma Medicine is a genomic medicine company focused on epigenetic editing to transform the treatment of genetically driven diseases. By leveraging epigenetics, which regulates gene expression naturally, Chroma Medicine aims to develop innovative therapies that provide precise control over gene activity. The company utilizes programmable epigenetic editors that combine DNA binding domains with epigenetic effector domains, allowing for targeted manipulation of genes and chromatin structure. This approach promises to create a new class of therapeutics that offers unparalleled control over gene expression, potentially leading to more effective treatments for a range of genetic disorders.
Arkuda Therapeutics is a biotechnology company leveraging new insights into progranulin and lysosomal biology to develop medicines to change the trajectory of neurodegenerative disease. Arkuda’s lead compounds aim to correct progranulin deficiency and lysosomal dysfunction in GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the GRN gene.
Vedere Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on developing gene therapy products aimed at restoring functional vision for patients who have experienced vision loss due to Inherited Retinal Degenerations (IRDs) and other causes of vision impairment, both genetic and non-genetic. Unlike many ocular gene therapies that primarily slow the progression of vision loss, Vedere Bio seeks to restore lost vision, regardless of the patient's genetic background or stage of their condition. As of September 2020, Vedere Bio operates as a subsidiary of Novartis AG.
Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It develops various programs for myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapies. The company was founded in 2017 and is based in Waltham, Massachusetts.
Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It develops various programs for myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapies. The company was founded in 2017 and is based in Waltham, Massachusetts.
Avrobio is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing ex vivo lentiviral gene therapies aimed at treating rare diseases with a single-dose approach. The company utilizes hematopoietic stem cells collected from patients, which are modified using a lentiviral vector to insert functional copies of defective genes associated with specific diseases. Avrobio's lead product candidate, AVR-RD-01, is currently in Phase II clinical trials for Fabry disease. Additionally, the company's pipeline includes AVR-RD-02, which has completed preclinical trials for type 1 Gaucher disease; AVR-RD-03, in preclinical development for Pompe disease; and AVR-RD-04, which has also completed preclinical studies for cystinosis. Avrobio has established a research collaboration with Magenta Therapeutics to explore targeted antibody-drug conjugates as a conditioning regimen for its lentiviral gene therapies. Founded in 2015, Avrobio aims to leverage advancements in cell and gene therapies to provide potential cures for serious and debilitating conditions.
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.
Korro Bio Inc. specializes in the development of nucleic acid-based therapeutics aimed at targeting specific sequences in genetic code, with a focus on treating rare diseases. Founded in 2018 and located in Cambridge, Massachusetts, the company utilizes a proprietary platform that enables efficient and selective RNA editing by leveraging natural processes found in multi-cellular organisms. This innovative approach allows Korro Bio to edit messenger RNA and recode specific codons, facilitating targeted modifications in protein structure and function across various tissues.
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.
Translate Bio is a clinical-stage biotechnology company based in Lexington, Massachusetts, that focuses on developing messenger RNA (mRNA) therapeutics aimed at treating diseases linked to protein or gene dysfunction. Since its founding in 2011, the company has been working on innovative treatments, including MRT5005, which is currently undergoing Phase I/II clinical trials for cystic fibrosis, and MRT5201, intended for the treatment of ornithine transcarbamylase deficiency. Translate Bio aims to create transformative medicines that address unmet medical needs in rare diseases.
Avrobio is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing ex vivo lentiviral gene therapies aimed at treating rare diseases with a single-dose approach. The company utilizes hematopoietic stem cells collected from patients, which are modified using a lentiviral vector to insert functional copies of defective genes associated with specific diseases. Avrobio's lead product candidate, AVR-RD-01, is currently in Phase II clinical trials for Fabry disease. Additionally, the company's pipeline includes AVR-RD-02, which has completed preclinical trials for type 1 Gaucher disease; AVR-RD-03, in preclinical development for Pompe disease; and AVR-RD-04, which has also completed preclinical studies for cystinosis. Avrobio has established a research collaboration with Magenta Therapeutics to explore targeted antibody-drug conjugates as a conditioning regimen for its lentiviral gene therapies. Founded in 2015, Avrobio aims to leverage advancements in cell and gene therapies to provide potential cures for serious and debilitating conditions.
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.
Avrobio is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing ex vivo lentiviral gene therapies aimed at treating rare diseases with a single-dose approach. The company utilizes hematopoietic stem cells collected from patients, which are modified using a lentiviral vector to insert functional copies of defective genes associated with specific diseases. Avrobio's lead product candidate, AVR-RD-01, is currently in Phase II clinical trials for Fabry disease. Additionally, the company's pipeline includes AVR-RD-02, which has completed preclinical trials for type 1 Gaucher disease; AVR-RD-03, in preclinical development for Pompe disease; and AVR-RD-04, which has also completed preclinical studies for cystinosis. Avrobio has established a research collaboration with Magenta Therapeutics to explore targeted antibody-drug conjugates as a conditioning regimen for its lentiviral gene therapies. Founded in 2015, Avrobio aims to leverage advancements in cell and gene therapies to provide potential cures for serious and debilitating conditions.
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
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.
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
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.
Cogent Biosciences Inc a biotechnology company focused on developing precision therapies for genetically defined diseases. It designs rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Its program CGT9486, is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. In the vast majority of cases, KIT D816V is responsible for driving Systemic Mastocytosis (SM), a serious disease caused by the unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancermore
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
Translate Bio is a clinical-stage biotechnology company based in Lexington, Massachusetts, that focuses on developing messenger RNA (mRNA) therapeutics aimed at treating diseases linked to protein or gene dysfunction. Since its founding in 2011, the company has been working on innovative treatments, including MRT5005, which is currently undergoing Phase I/II clinical trials for cystic fibrosis, and MRT5201, intended for the treatment of ornithine transcarbamylase deficiency. Translate Bio aims to create transformative medicines that address unmet medical needs in rare diseases.
Molecular Biometrics, Inc. is a metabolomics company based in Norwood, Massachusetts, focused on developing non-invasive methodologies for assessing biological functions in health and disease, as well as for drug discovery and development. Established in 2005, the company leverages innovative metabolomic technologies to create molecular gastronomy kits. Its applications span various fields, including reproductive health, neurodegenerative diseases such as Alzheimer's and Parkinson's, maternal-fetal medicine, pulmonary metabolism and edema, and lactate metabolism. By applying its proprietary technology, Molecular Biometrics aims to enhance the understanding and treatment of complex health issues.
Helicos BioSciences Corporation is a life sciences company that specializes in advanced genetic analysis technology. It has developed the True Single Molecule Sequencing (tSMS) technology, which allows for the rapid analysis of large amounts of genetic material by directly sequencing individual molecules of DNA and RNA. This innovative approach enables precise sequencing and offers significant advantages in research, drug discovery, and clinical diagnostics. The Helicos Genetic Analysis Platform includes the HeliScope Single Molecule Sequencer, which performs a series of biochemical reactions on single DNA or RNA molecules, and the HeliScope Analysis Engine, an image analysis computer system that processes the sequencing data. The platform also utilizes specialized reagents and disposable supplies essential for the sequencing process. Through its unique technology, Helicos aims to enhance the understanding of genetic information and improve applications in various fields of life sciences.
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.
ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.
Phylogix Inc. is a biotechnology company focused on tissue protection and oncology. The company develops lectin-based drugs focused on tissue protection, oncology, and immunology. It also involves in the development of FRIL, an agent to protect patients from the toxic side-effects of chemotherapy. Phylogix Inc. is based in Scarborough, Maine.
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.
Variagenics
Funding Round in 2000
Variagenics develops and commercializes proprietary gene variance detection technology to streamline drug development.
Develogen is a biology-driven drug discovery company that is engaged in the development of therapies for diabetes and obesity. The company was founded in 1995 and is based in Georgia, United States.
DeCODE Genetics is a biopharmaceutical company based in Reykjavik, Iceland, established in 1996. It specializes in analyzing the human genome to identify genetic variants associated with common diseases. The company conducts population studies to uncover key genetic risk factors for various health conditions, including cardiovascular disease and cancer. DeCODE Genetics develops diagnostic tests and technologies aimed at improving the treatment, diagnosis, and prevention of these diseases. By leveraging its expertise in chemistry and structural biology, the company supports the healthcare sector in offering DNA-based reference laboratory tests and consumer genome analysis services, which help assess individual disease risk.