ReCode Therapeutics is a development-stage biopharmaceutical company focused on the correction of the nonsense mutations that account for 1/3 of all inherited diseases, including approximately 10% of cystic fibrosis. They have combined selected tRNAs with their unique and diverse proprietary delivery platforms to create and develop first-in-class suppressor tRNA-nanoparticle therapeutics for correcting nonsense mutations. ReCode’s diverse pipeline includes lead programs for primary ciliary dyskinesia (PCD) and nonsense mutations in cystic fibrosis (CF). The company’s proprietary non-viral lipid nanoparticle platform enables the delivery of a variety of payloads, and precise organ targeting of nucleic acid and protein payloads via both systemic and local delivery. ReCode is leveraging its nanoparticle and nucleic acid technologies across multiple modalities, including mRNA-mediated protein replacement therapies, first-in-class tRNA NanoCorrectors for diseases caused by nonsense mutations, and gene editing. The company was founded in 2015 and is based in Dallas, Texas.
OMass Technologies Ltd engages in drug discovery by applying mass spectrometry to the characterization of intact protein assemblies. The company offers biotherapeutics, membrane proteins, drug binding, and purifications guiding services. In addition, OMass Technologies Ltd offers structural mass spectrometry (MS) platforms, often referred to as native mass spectrometry, for characterizing complex protein assemblies. The company assists biotechnology and pharmaceutical companies in tackling drug targets and biotherapeutics. OMass Technologies Ltd was founded in 2016 and is based in Begbroke, United Kingdom.
Nucleai Ltd. is a company that specializes in AI-powered pathology technology aimed at enhancing cancer detection and treatment. Founded in 2017 and headquartered in Tel Aviv-Yafo, Israel, Nucleai's platform analyzes tissue samples to provide insights into cancer biology, which aids in biomarker discovery and improves clinical trial outcomes. The company serves hospitals and laboratories in the United States and Israel, collaborating with leading pharmaceutical firms to license its research platform for internal applications and participate in biomarker discovery projects. By leveraging unique tissue datasets, Nucleai seeks to transform the precision oncology landscape, ultimately improving patient care.
Click Therapeutics, Inc. specializes in the engineering, validation, development, and commercialization of digital therapeutic solutions aimed at addressing unmet medical needs through cognitive and neurobehavioral modification. Founded in 2012 and based in New York, the company offers innovative digital therapeutics for a range of conditions, including smoking cessation, major depressive disorder, schizophrenia, insomnia, and chronic pain. Its Digital Therapeutics™ are designed to function independently or alongside traditional biomedical treatments, utilizing the Clickometrics® adaptive data science platform to personalize user experiences and enhance engagement. Notably, Click's smoking cessation program has gained widespread access through various payers and providers, while its primary prescription program is advancing into a significant phase III clinical trial for major depressive disorder in adults.
ReCode Therapeutics is a development-stage biopharmaceutical company focused on the correction of the nonsense mutations that account for 1/3 of all inherited diseases, including approximately 10% of cystic fibrosis. They have combined selected tRNAs with their unique and diverse proprietary delivery platforms to create and develop first-in-class suppressor tRNA-nanoparticle therapeutics for correcting nonsense mutations. ReCode’s diverse pipeline includes lead programs for primary ciliary dyskinesia (PCD) and nonsense mutations in cystic fibrosis (CF). The company’s proprietary non-viral lipid nanoparticle platform enables the delivery of a variety of payloads, and precise organ targeting of nucleic acid and protein payloads via both systemic and local delivery. ReCode is leveraging its nanoparticle and nucleic acid technologies across multiple modalities, including mRNA-mediated protein replacement therapies, first-in-class tRNA NanoCorrectors for diseases caused by nonsense mutations, and gene editing. The company was founded in 2015 and is based in Dallas, Texas.
Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases.
ROME Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapies for cancer and autoimmune diseases. Established in 2019, ROME Therapeutics is pioneering the exploration of the repeatome, which consists of extensive regions of genetic material previously considered "junk DNA." The company has identified several drug targets and is actively advancing multiple discovery programs. To facilitate its research, ROME has assembled a team of experts across various disciplines, including oncology, immunology, virology, and machine learning, to harness this uncharted area of biology for therapeutic advancements.
Operator of a biopharmaceutical company intended to discover and develop therapies that slow or stop the progression of neurodegenerative diseases. The company engages in identifying new medicines to preserve cognition and other brain functions and enhance resilience to neurodegenerative diseases including Alzheimer's, Frontotemporal Dementia, and Parkinson's, enabling people suffering from neurodegenerative diseases to access suitable therapeutics for fast recovery.
Glycomine, Inc. is a biotechnology company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.
Veralox Therapeutics specializes in the development of small molecule therapeutics aimed at addressing the underlying causes of thrombosis and type one diabetes. By leveraging a deep understanding of the molecular mechanisms associated with these conditions, the company seeks to create innovative treatment options that can lead to improved health outcomes for patients. Their focus includes therapies specifically targeting heparin-induced thrombocytopenia and thrombosis, with the goal of providing patients with effective solutions for complete recovery from diabetes and associated complications.
Therini Bio is dedicated to the research and development of novel therapeutics for the treatment of neurological diseases. The company's approach is based on novel findings by the Akassoglou lab at Gladstone/UCSF that implicate fibrin as a key driver of neuroinflammation.
Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. It specializes in the development of vaccines using computationally designed virus-like particles (VLPs). The company's primary focus is on creating safe and effective vaccines to address significant unmet medical needs in the realm of infectious diseases, particularly life-threatening respiratory illnesses. One of its key vaccine candidates is IVX-121, aimed at protecting older adults from respiratory syncytial virus (RSV) disease. Icosavax leverages exclusive technology licensed from the Institute for Protein Design at the University of Washington to advance its innovative vaccine solutions.
MediSafe Project LTD. provides MediSafe, a cloud-synced mobile medication management solution that helps families in preventing emergencies caused by over-or under-dosing medications. It offers patient compliance and businesses solutions. The company was founded in 2012 and is based in Haifa, Israel.
DiCE Therapeutics, founded in 2013 and based in Menlo Park, California, specializes in developing a platform for the discovery of novel small molecules aimed at targets that have previously been difficult to address in the pharmaceutical sector. The company utilizes innovative technology that extends directed evolution, allowing for the direct translation of DNA-encoded information into organic compounds, thereby streamlining the traditionally labor-intensive processes of medicinal chemistry. This platform not only serves the pharmaceutical industry but also has applications in agriculture and materials sciences. The founding team includes notable figures such as Stanford Professor Pehr Harbury, who developed the core technology, and other experienced professionals from various sectors, contributing to the company's mission of transforming drug discovery and development.
Abcuro, Inc. is focused on developing immunotherapies aimed at treating autoimmunity and cancer. Incorporated in 2015 and based in Newton, Massachusetts, the company specializes in creating therapeutic antibodies that target novel aspects of the immune system. By employing advanced bioinformatics and analyzing transcription data from human diseases, Abcuro identifies new therapeutic targets and validates them through ex-vivo studies in diseased tissues. This innovative approach aims to enhance treatment strategies and improve patient outcomes in the fields of autoimmunity and oncology.
Minervax is pursuing the development of a vaccine against Group B streptococcus (GBS) infections by utilizing a particular fusion protein, which has proved to elicit good protective immunity. A vaccine including this fusion protein will likely elicit protective immunity against many clinically relevant GBS strains.
Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief.
Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.
Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief.
Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.
Lava Therapeutics B.V. is a biotechnology company based in Utrecht, the Netherlands, focused on developing innovative immune oncology biopharmaceuticals. Founded in 2016, the company specializes in a bispecific antibody platform designed to engage gamma-delta T cells, which play a crucial role in the immune response against cancer. Lava Therapeutics aims to create next-generation bispecific antibodies that not only activate and recruit the immune system but also enhance its ability to identify and eliminate tumor cells. The company's approach seeks to provide effective, safe, and cost-efficient treatments for both hematological and solid cancers, empowering healthcare professionals to better harness the body's immune capabilities in the fight against cancer.
Escient Pharmaceuticals, Inc., a biotechnology company, develops and manufactures G protein-coupled receptor (GPCR)-targeted drugs for treating neuro-immuno-inflammatory and autoreactive diseases. The company focuses on unleashing the therapeutic potential of specific orphan GPCRs, including the novel family of Mas-Related G-Protein Receptors (Mrgprs) for neuro-immuno-inflammatory and autoreactive diseases. Escient Pharmaceuticals, Inc. was formerly known as Mas Therapeutics, Inc. The company was founded in 2017 and is based in San Diego, California.
Science 37, Inc. is a technology-driven clinical trial company focused on transforming clinical research through patient-centric models. It has developed NORA (Network Oriented Research Assistant), a cloud-based mobile research platform that facilitates communication between researchers and patients via videos, photographs, and surveys, enabling end-to-end networked clinical trial services. The company also offers the Science 37 Metasite, which enhances access to diverse patient populations for clinical studies, and the Science 37 Platform for decentralized clinical trials. By utilizing its virtual model, Science 37 has demonstrated faster enrollment and higher patient retention, reaching a broader and more representative patient demographic than traditional site-based research. The company serves a wide range of clients, including large pharmaceutical firms, biotechnology companies, universities, and startups. Since its incorporation in 2014, Science 37 has established itself as a leader in virtual clinical trials, leveraging an extensive network of telemedicine investigators and home-health nurses to conduct more decentralized interventional trials than any other organization.
NodThera Limited is a biotechnology company focused on researching and developing novel inhibitors of the NLRP3 inflammasome, which play a critical role in inflammatory and neuroinflammatory diseases. Established in 2016 and headquartered in Little Chesterford, United Kingdom, NodThera operates a laboratory in Seattle, Washington, and a corporate office in Lexington, Massachusetts. The company aims to create therapies targeting a range of conditions including arthritis, atherosclerosis, Alzheimer’s disease, diabetes, and certain cancers. By developing potent and selective NLRP3 inflammasome inhibitors, NodThera seeks to reduce pro-inflammatory cytokines, enabling more effective treatment options for patients suffering from chronic inflammation-related diseases.
Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief.
Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.
i2O Therapeutics, Inc. is a biotechnology company based in Saratoga, California, founded in 2019. The company specializes in developing oral formulations for therapies that are traditionally administered via injections. Utilizing its innovative ionic liquid technology, i2O Therapeutics aims to enhance the delivery of biologics by protecting the drug cargo and improving its absorption through the epithelial lining when taken orally. This approach focuses on creating next-generation oral therapies, particularly for peptides and proteins, which are typically challenging to deliver in pill form. The company's work addresses the need for safer and more convenient alternatives to injection-based treatments, targeting metabolic and inflammatory diseases.
Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. It specializes in the development of vaccines using computationally designed virus-like particles (VLPs). The company's primary focus is on creating safe and effective vaccines to address significant unmet medical needs in the realm of infectious diseases, particularly life-threatening respiratory illnesses. One of its key vaccine candidates is IVX-121, aimed at protecting older adults from respiratory syncytial virus (RSV) disease. Icosavax leverages exclusive technology licensed from the Institute for Protein Design at the University of Washington to advance its innovative vaccine solutions.
Veralox Therapeutics specializes in the development of small molecule therapeutics aimed at addressing the underlying causes of thrombosis and type one diabetes. By leveraging a deep understanding of the molecular mechanisms associated with these conditions, the company seeks to create innovative treatment options that can lead to improved health outcomes for patients. Their focus includes therapies specifically targeting heparin-induced thrombocytopenia and thrombosis, with the goal of providing patients with effective solutions for complete recovery from diabetes and associated complications.
Omada Health, Inc. is a digital healthcare company that creates online programs aimed at managing chronic conditions. Founded in 2011 and headquartered in San Francisco, with an additional office in Atlanta, Omada Health helps employers and health plans identify individuals at risk for preventable chronic diseases such as prediabetes, diabetes, hypertension, and heart disease. The company offers personalized programs that adapt to the needs of participants, including services for diabetes prevention and management, hypertension, behavioral health, and musculoskeletal issues. By integrating professional health coaching, connected devices, real-time data, and personalized feedback, Omada Health strives to empower individuals to engage with their health and achieve sustainable lifestyle changes. It is recognized as the largest provider of the National Diabetes Prevention Program by the CDC.
Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.
Aetion, Inc. develops a science and analytics platform to assess real-world evidence (RWE) from a range of sources. The company offers Aetion Evidence Platform, a platform that generates RWE needed by healthcare decision makers to engage in value-based care. Its solution allows payers, providers, medical device makers, biopharma customers, and academic institutions to analyze data from various clinical and financial interactions in healthcare, including claims, electronic health records, registries, and clinical trials. The company’s platform is also used in database analytics and comparative effectiveness courses taken by epidemiologists, outcomes researchers, health economists, and biostatisticians. It serves customers worldwide. Aetion, Inc. has a strategic collaboration with McKesson. The company was founded in 2012 and is based in New York, New York.
Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, established in 2015. It specializes in targeted protein degradation, developing innovative small molecule therapeutics that selectively degrade proteins associated with various diseases by utilizing the body's natural protein degradation mechanisms. The company is advancing several therapeutic programs, including the IRAK4 program aimed at treating immunology-inflammation diseases such as hidradenitis suppurativa, atopic dermatitis, and rheumatoid arthritis. Additionally, it is developing the IRAKIMiD program for MYD88-mutated diffuse large B cell lymphoma and the STAT3 program for hematologic malignancies and solid tumors, as well as autoimmune diseases. Through its proprietary predictive modeling platform, Kymera Therapeutics seeks to accelerate drug discovery and address previously untreatable conditions.
Click Therapeutics, Inc. specializes in the engineering, validation, development, and commercialization of digital therapeutic solutions aimed at addressing unmet medical needs through cognitive and neurobehavioral modification. Founded in 2012 and based in New York, the company offers innovative digital therapeutics for a range of conditions, including smoking cessation, major depressive disorder, schizophrenia, insomnia, and chronic pain. Its Digital Therapeutics™ are designed to function independently or alongside traditional biomedical treatments, utilizing the Clickometrics® adaptive data science platform to personalize user experiences and enhance engagement. Notably, Click's smoking cessation program has gained widespread access through various payers and providers, while its primary prescription program is advancing into a significant phase III clinical trial for major depressive disorder in adults.
Evidation Health develops a health data analytics platform that collects and analyzes continuous behavior data alongside healthcare information to enhance health outcomes. Founded in 2012 and headquartered in San Mateo, California, the company utilizes raw, high-frequency data from various sources such as sensors, devices, and video to generate insights into health and disease. Its products include a Data Platform, which transforms everyday behavior data into actionable health measures, and Studies, a service for conducting real-world research that incorporates behavioral data to support clinical and commercial teams. Evidation Health connects digital health companies with healthcare providers and payers, facilitating a better understanding of how everyday behaviors influence health. The company's offerings also encompass health outcomes research, technology assessment, and predictive analytics services.
Imbria Pharmaceuticals, Inc. is a Boston-based pharmaceutical company founded in 2018, dedicated to developing innovative treatments that enhance or restore cellular energy production. Focused on addressing life-altering cardiometabolic disorders, Imbria's therapies aim to improve outcomes for patients suffering from conditions such as non-obstructive hypertrophic cardiomyopathy, stable angina, and heart failure with preserved ejection fraction. The company operates at the clinical stage, leveraging a deep scientific understanding of cellular metabolism and mitochondrial function to create novel medicines. Founded and led by a team of doctors, Imbria is committed to translating scientific insights into effective therapies that address significant unmet medical needs, prioritizing the well-being of patients and their communities.
Common Sensing, Inc. is a Cambridge, Massachusetts-based company that specializes in developing data-driven hardware and software solutions for individuals using injectable medications, particularly in diabetes care. Founded in 2012, the company is known for its flagship product, the Gocap, a smart cap that transforms conventional injector pens into smart injectors. This device allows for the logging of insulin doses, monitoring of temperature, and provides reminders and alerts, facilitating better management of insulin usage. Additionally, the Gocap app enhances patient care by allowing users to track the effects of insulin alongside glucose and meal entries, creating a comprehensive snapshot of their health. By enabling patients to share their insulin logs with healthcare providers and family members, Common Sensing aims to improve access to care, reduce healthcare costs, and ultimately enhance health outcomes for those managing diabetes.
Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases.
Curisium Inc. operates as a healthcare technology and services company that develops blockchain based platform. It facilitates patient-centric contracting for payers, providers, and life science companies. Curisium Inc. was formerly known as Holosense Inc. The company was founded in 2016 and is headquartered in Manhattan Beach, California. As of July 20, 2020, Curisium Inc. operates as a subsidiary of HealthVerity, Inc.
Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.
Neurovia, Inc. is a pharmaceutical company based in Cambridge, Massachusetts, founded in 2013. The company focuses on developing therapies for neurological disorders, specifically targeting unmet medical needs associated with X-linked adrenoleukodystrophy (X-ALD). Neurovia's primary objective is to provide a treatment that can halt the progression of neurological deficits caused by this rare genetic disease. The company has developed a selective thyromimetic agent aimed at improving the quality of life for patients suffering from X-ALD.
Omada Health, Inc. is a digital healthcare company that creates online programs aimed at managing chronic conditions. Founded in 2011 and headquartered in San Francisco, with an additional office in Atlanta, Omada Health helps employers and health plans identify individuals at risk for preventable chronic diseases such as prediabetes, diabetes, hypertension, and heart disease. The company offers personalized programs that adapt to the needs of participants, including services for diabetes prevention and management, hypertension, behavioral health, and musculoskeletal issues. By integrating professional health coaching, connected devices, real-time data, and personalized feedback, Omada Health strives to empower individuals to engage with their health and achieve sustainable lifestyle changes. It is recognized as the largest provider of the National Diabetes Prevention Program by the CDC.
Evidation Health develops a health data analytics platform that collects and analyzes continuous behavior data alongside healthcare information to enhance health outcomes. Founded in 2012 and headquartered in San Mateo, California, the company utilizes raw, high-frequency data from various sources such as sensors, devices, and video to generate insights into health and disease. Its products include a Data Platform, which transforms everyday behavior data into actionable health measures, and Studies, a service for conducting real-world research that incorporates behavioral data to support clinical and commercial teams. Evidation Health connects digital health companies with healthcare providers and payers, facilitating a better understanding of how everyday behaviors influence health. The company's offerings also encompass health outcomes research, technology assessment, and predictive analytics services.
Science 37, Inc. is a technology-driven clinical trial company focused on transforming clinical research through patient-centric models. It has developed NORA (Network Oriented Research Assistant), a cloud-based mobile research platform that facilitates communication between researchers and patients via videos, photographs, and surveys, enabling end-to-end networked clinical trial services. The company also offers the Science 37 Metasite, which enhances access to diverse patient populations for clinical studies, and the Science 37 Platform for decentralized clinical trials. By utilizing its virtual model, Science 37 has demonstrated faster enrollment and higher patient retention, reaching a broader and more representative patient demographic than traditional site-based research. The company serves a wide range of clients, including large pharmaceutical firms, biotechnology companies, universities, and startups. Since its incorporation in 2014, Science 37 has established itself as a leader in virtual clinical trials, leveraging an extensive network of telemedicine investigators and home-health nurses to conduct more decentralized interventional trials than any other organization.
Science 37, Inc. is a technology-driven clinical trial company focused on transforming clinical research through patient-centric models. It has developed NORA (Network Oriented Research Assistant), a cloud-based mobile research platform that facilitates communication between researchers and patients via videos, photographs, and surveys, enabling end-to-end networked clinical trial services. The company also offers the Science 37 Metasite, which enhances access to diverse patient populations for clinical studies, and the Science 37 Platform for decentralized clinical trials. By utilizing its virtual model, Science 37 has demonstrated faster enrollment and higher patient retention, reaching a broader and more representative patient demographic than traditional site-based research. The company serves a wide range of clients, including large pharmaceutical firms, biotechnology companies, universities, and startups. Since its incorporation in 2014, Science 37 has established itself as a leader in virtual clinical trials, leveraging an extensive network of telemedicine investigators and home-health nurses to conduct more decentralized interventional trials than any other organization.
Immunexcite, Inc., a biopharmaceutical company, develops monoclonal antibodies for cancer. The company develops monoclonal antibodies by employing its mAbXcite, an immunotherapy platform technology that targets cancer cells for destruction by engaging neutrophils. Immunexcite, Inc. was founded in 2007 and is based in Lexington, Massachusetts.
Yumanity Therapeutics is a biotechnology company dedicated to transforming drug discovery for neurodegenerative diseases linked to protein misfolding. Founded in December 2014 by protein folding expert Susan Lindquist and biotech leader Tony Coles, Yumanity aims to develop innovative, disease-modifying therapies to address critical unmet medical needs in conditions such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). The company leverages proprietary platforms to identify potential therapeutic targets and has made progress in advancing a new chemical lead series specifically for Parkinson’s disease, while also exploring additional compounds for Alzheimer's disease and ALS.
Navitor Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing small molecule drugs that target mTORC1, a key regulator of cellular metabolism and renewal, to treat age-related diseases. Utilizing a proprietary platform, Navitor's therapies can selectively inhibit or activate mTORC1, which plays a crucial role in cellular processes. One of its notable products, NV-5138, is an orally bioavailable small molecule designed to activate mTORC1, particularly addressing its suppression in the brain associated with depression. Additionally, the company's N-Valog program focuses on selective inhibition of mTORC1 for chronic kidney disease, with implications for other age-related conditions. Founded in 2009 and headquartered in Cambridge, Massachusetts, Navitor Pharmaceuticals aims to unlock the therapeutic potential of the mTOR pathway by modulating nutrient-sensing proteins, thereby addressing various metabolic, neurodegenerative, autoimmune, and musculoskeletal diseases.
Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.
Selecta Biosciences is a clinical-stage biopharmaceutical company focused on developing nanoparticle immunomodulatory drugs aimed at treating and preventing human diseases. The company’s proprietary platform integrates advancements in immunobiology and nanotechnology to create targeted immunomodulatory nanoparticles. Its lead product, SEL-212, has completed Phase II clinical trials for chronic refractory gout. Additionally, Selecta is advancing gene therapy candidates, including SEL-302 and SEL-313, which are in preclinical development for rare metabolic disorders. The company has established collaborations and licensing agreements with various organizations, including Spark Therapeutics and the Massachusetts Institute of Technology. Founded in 2007 and headquartered in Watertown, Massachusetts, Selecta Biosciences continues to innovate in the biopharmaceutical field.
Ovid Therapeutics Inc. is a biopharmaceutical company based in New York, focused on developing innovative therapies for patients with neurological disorders, particularly rare diseases. Founded in 2014, the company is advancing a promising pipeline of drug candidates aimed at treating conditions such as angelman syndrome and fragile X syndrome. Its lead candidate, OV101, is in Phase III clinical trials for angelman syndrome and has completed Phase II trials for fragile X syndrome. Additionally, Ovid is developing OV935, currently in Phase II trials for cyclin-dependent kinase-like 5 deficiency disorder and Dravet syndrome. The company is also researching OV329 for infantile spasms and OV881 as a microRNA gene therapy for angelman syndrome. Ovid Therapeutics has established collaborations and licensing agreements with various pharmaceutical and research organizations to enhance its development efforts. The company's commitment to addressing unmet medical needs is underpinned by strong intellectual property and a focus on safe, effective treatments for patients facing challenging neurological conditions.
Unum Therapeutics is a biopharmaceutical company focused on developing immunotherapy products for cancer treatment, utilizing proprietary T-cell engineering technology. The company’s lead programs, based on its Antibody-Coupled T-cell Receptor (ACTR) technology, are in Phase I clinical trials. These include ACTR707 and ACTR087, both being tested in combination with rituximab for relapsed or refractory CD20+ non-Hodgkin lymphoma, as well as ACTR087 combined with SEA-BCMA for relapsed multiple myeloma. Additionally, ACTR707 is being evaluated with trastuzumab for HER2+ solid tumors. Unum Therapeutics is also exploring BOXR1030, a pre-clinical candidate targeting glypican-3 to enhance T cell functionality in the solid tumor microenvironment. Founded in 2014 and headquartered in Cambridge, Massachusetts, Unum aims to activate the body’s immune system to effectively combat cancer through its innovative therapies.
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
Lysosomal Therapeutics, Inc. is focused on discovering and developing small-molecule therapies aimed at treating severe neurological diseases, including Gaucher-related neurodegeneration and Parkinson’s disease. Founded in 2011 and based in Cambridge, Massachusetts, the company utilizes a research strategy that capitalizes on the established connection between lysosomal disorders and neurodegenerative conditions. This approach supports the development of innovative treatment options for patients affected by movement, cognition, and overall health issues related to these diseases. By leveraging clinically validated insights, Lysosomal Therapeutics aims to create effective therapies to address the unmet medical needs in the field of neurodegeneration.
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
Unum Therapeutics is a biopharmaceutical company focused on developing immunotherapy products for cancer treatment, utilizing proprietary T-cell engineering technology. The company’s lead programs, based on its Antibody-Coupled T-cell Receptor (ACTR) technology, are in Phase I clinical trials. These include ACTR707 and ACTR087, both being tested in combination with rituximab for relapsed or refractory CD20+ non-Hodgkin lymphoma, as well as ACTR087 combined with SEA-BCMA for relapsed multiple myeloma. Additionally, ACTR707 is being evaluated with trastuzumab for HER2+ solid tumors. Unum Therapeutics is also exploring BOXR1030, a pre-clinical candidate targeting glypican-3 to enhance T cell functionality in the solid tumor microenvironment. Founded in 2014 and headquartered in Cambridge, Massachusetts, Unum aims to activate the body’s immune system to effectively combat cancer through its innovative therapies.
Lysosomal Therapeutics, Inc. is focused on discovering and developing small-molecule therapies aimed at treating severe neurological diseases, including Gaucher-related neurodegeneration and Parkinson’s disease. Founded in 2011 and based in Cambridge, Massachusetts, the company utilizes a research strategy that capitalizes on the established connection between lysosomal disorders and neurodegenerative conditions. This approach supports the development of innovative treatment options for patients affected by movement, cognition, and overall health issues related to these diseases. By leveraging clinically validated insights, Lysosomal Therapeutics aims to create effective therapies to address the unmet medical needs in the field of neurodegeneration.
Immune Design Corp. (IDC) uses breakthrough technology to develop safe and effective vaccines and therapeutics, with emphasis on the prevention and treatment of infectious disease. This goal is being achieved through dedicated research aimed towards a greater understanding of the immune system, and by developing a new generation of immune-based products for the pharmaceutical industry. IDC is passionate about its mission to alleviate human suffering and pursues this goal with high energy and an unsurpassed standard of excellence
KAHR Medical Ltd. is an immuno-oncology drug development company based in Jerusalem, Israel, founded in 2005. The company focuses on creating fusion protein pharmaceuticals aimed at treating cancer and autoimmune diseases. KAHR develops innovative drugs such as DSP105 and DSP106, which facilitate T-cell mediated tumor destruction, and DSP107, which targets CD47 tumors to activate phagocytes while delivering an immune costimulatory signal. Utilizing a unique platform of multifunctional immuno-recruitment proteins, KAHR's technology aims to enhance immune system recognition of cancer cells, thereby addressing the challenge of cancer evasion. This approach represents a significant advancement in protein-based pharmaceuticals, expanding treatment options for patients facing these serious health conditions.
Edimer Pharmaceuticals: Working toward improving the health and quality of life of families living with XLHED. Edimer Pharmaceuticals is dedicated to developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED). XLHED is a rare, orphan disease that causes a range of symptoms including lack of sweat glands, poor temperature control, respiratory problems, and hair and tooth malformations. Edimer is led by a team of seasoned biotechnology industry veterans with deep experience in drug development complemented by a strong network of world-class clinical and scientific advisers.
Lumena Pharmaceuticals focuses on developing oral therapeutics for rare liver diseases, aiming to enhance liver function and alleviate disease symptoms. The company's clinical-stage product candidates are designed to selectively target a transporter in the intestine, minimizing systemic absorption and reducing the risk of toxicities. Lumena's lead candidate, LUM001, has been thoroughly evaluated in over 12 clinical studies involving more than 1,400 subjects, positioning it for rapid advancement in clinical trials for both pediatric and adult patients with various forms of cholestatic liver disease. In addition to its primary objective of creating novel treatments for rare liver conditions, Lumena's innovative therapeutic approach also holds potential for addressing metabolic diseases affecting the liver, which represent a significant and growing health concern among both children and adults.
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.
GlycoMimetics, Inc. is a clinical-stage biotechnology company dedicated to the discovery and development of innovative glycomimetic drugs aimed at addressing significant unmet medical needs associated with diseases influenced by carbohydrate biology. The company’s lead drug candidate, rivipansel, is a pan-selectin antagonist that has completed Phase III clinical trials for treating vaso-occlusive crises in sickle cell disease. Additionally, GlycoMimetics is developing uproleselan, an E-selectin inhibitor that is being tested in combination with chemotherapy for acute myeloid leukemia (AML) and in a Phase III trial for relapsed/refractory AML. The company also offers GMI-1359, which targets E-selectin and a chemokine receptor, having completed Phase I clinical trials, and is involved in developing other candidates, including GMI-1687 and Galectin-3. GlycoMimetics has established research collaborations, including an agreement with the National Cancer Institute and a partnership with Apollomics for the commercialization of uproleselan and GMI-1687. Founded in 2003, the company is headquartered in Rockville, Maryland.
Fate Therapeutics is using the fundamental biological mechanisms that guide cell fate to develop stem cell therapeutics.
The company has brought together the foremost scientists from the nation's research hotbeds (Boston, San Francisco, San Diego, and Seattle) who have demonstrated the potential to create and modulate stem cells to restore health.
The backstory is that while others were working towards therapies based on transplanting stem cell-derived cells into patients, Randall Moon (HHMI and the University of Washington) and Alex Rives (Arch Venture Capitol) envisioned a different approach to develop regenerative therapies. The concept, based on data from hematopoietic progenitor cells, was that one could modulate the properties of stem cells in vivo using small molecules or biologics. They presented this idea to Arch Venture Capitol in Seattle, which launched the company, soon to be joined by other investors.
Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.
TcLand Expression is a fully integrated company displaying high expertise in all stages of biomarker development: R&D, bioinformatics & biostatistics, clinical & regulatory , market access and production. TcLand Expression is a pioneer and European leader in personalized medicine in immunology (transplantation and auto-immune disorders) with a rich pipeline of biomarkers in development.
Sirtris Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of proprietary, orally available small molecule drugs aimed at treating diseases related to aging. The company's research primarily focuses on metabolic disorders, including Type 2 Diabetes. By leveraging innovative approaches in drug development, Sirtris seeks to address the growing healthcare challenges associated with age-related diseases.