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. develops AI-powered pathology platform that is used for satellite image analysis to detect cancer, prostate, breast, and gastrointestinal related diseases. Its platform leverages unique tissue datasets to produce insights into cancer biology, increasing clinical trial efficacy, and improving patient care. It serves hospitals and labs in in the United States and Israel. The company was founded in 2017 and is headquartered in Tel Aviv-Yafo, Israel.
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.
Glycomine, Inc. is a biotechnology company based in San Carlos, California, established in 2014. The company specializes in developing nanomedicines aimed at treating rare genetic disorders, particularly those related to protein and lipid glycosylation. These disorders often lead to significant health challenges for affected individuals, and Glycomine focuses on creating therapeutics for diseases that currently lack FDA-approved treatment options. With approximately 7,000 rare disorders identified worldwide, and a majority of them without viable therapies, Glycomine is positioned to address critical unmet medical needs in this niche area of healthcare.
Veralox Therapeutics develops small molecule therapeutics that treat the underlying pathologies of thrombosis and type one diabetes. Based on an understanding of the molecular mechanisms of these diseases, these efforts will lead to new treatment paradigms and better outcomes for patients.
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 is focused on developing safe and effective vaccines against infectious diseases that address important unmet medical needs and reduce healthcare costs. tHEY were founded on breakthrough computationally-designed virus-like particle technology, exclusively licensed for a variety of infectious disease indications from the Institute for Protein Design at the University of Washington.
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’s mission is to develop a new class of immune-modulatory bio-therapeutics for treating both autoimmunity and cancer. They leverage the systematic target validation and clinical insights gained by its founders to identify new targets. Their intensive use of bioinformatics to interrogate the transcriptome of human disease together with ex-vivo validation in diseased tissue has uncovered new approaches to target key compartments of the immune system.
The company was incorporated in 2015 and is based in Newton, Massachusetts.
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.
Lava Therapeutics is a developer of a bispecific antibody platform used to engage gamma-delta T cells for the treatment of hematological and solid cancers. Its platform creates and develops next-generation γδ T cell engaging bispecific antibodies for the treatment of cancer as well as develop potent, safe, and cost-effective biopharmaceuticals that arm the immune system to recognize and destroy tumor cells that enable doctors to safely channel the immune system's response towards tumors.
Lava Therapeutics was established in 2016 and is headquartered in Utrecht, The Netherlands.
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 is making the promise of virtual trials the new reality. By engaging with patients from the comfort of their own home, the company provides access to patients who can never be reached by traditional site-based research models. Its virtual model has proven to enroll faster, retain patients at a higher rate, and reach a more representative patient population. Science 37 has conducted more virtual (or decentralized), interventional trials than any other company, using an expansive, in-house network of telemedicine investigators and home-health nurses. The company's research is powered by the industry’s most comprehensive, fully integrated clinical trial platform purpose-built to conduct virtual trials.
NodThera Limited is a biotechnology company based in Little Chesterford, United Kingdom, with additional facilities in Seattle, Washington, and Lexington, Massachusetts. Founded in 2016, the company specializes in researching and developing novel inhibitors of the NLRP3 inflammasome, a multi-protein complex involved in the body's immune response. NodThera aims to address a variety of inflammatory and neuroinflammatory diseases, including arthritis, atherosclerosis, Alzheimer’s disease, diabetes, and certain cancers. Through its innovative approach to inflammasome research, NodThera seeks to contribute to the advancement of therapeutic options for these conditions.
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 is focused on developing safe and effective vaccines against infectious diseases that address important unmet medical needs and reduce healthcare costs. tHEY were founded on breakthrough computationally-designed virus-like particle technology, exclusively licensed for a variety of infectious disease indications from the Institute for Protein Design at the University of Washington.
Veralox Therapeutics develops small molecule therapeutics that treat the underlying pathologies of thrombosis and type one diabetes. Based on an understanding of the molecular mechanisms of these diseases, these efforts will lead to new treatment paradigms and better outcomes for patients.
Omada Health, Inc. is a digital healthcare company that develops online programs aimed at managing chronic conditions. Founded in 2011 and headquartered in San Francisco, with an additional office in Atlanta, the company collaborates with employers and health plans to identify individuals at risk for preventable chronic conditions such as prediabetes, diabetes, hypertension, and heart disease. Omada's programs are personalized to address the evolving needs of participants, offering support in areas like diabetes prevention, type 2 diabetes management, and behavioral health. The company integrates professional health coaching, connected health devices, real-time data, and personalized feedback to achieve clinically significant outcomes. Omada Health is recognized as the largest provider of the National Diabetes Prevention Program by the Centers for Disease Control and Prevention (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 is a provider of a health data analytics platform designed to collect and analyze continuous behavior data and healthcare information so as to create better health outcomes. Its platform turns raw, high-frequency everyday behavior data from sensors, devices, speech, video, and other sources into new knowledge about health and disease, that enable individuals and innovative companies to understand and influence the everyday behaviors.
It was founded in 2012 and is headquartered in San Mateo, California.
Imbria Pharmaceuticals’ mission is to translate our understanding of cellular metabolism into innovative medicines that improve the lives of patients with limited or inadequate treatment options. The company was founded by doctors and launched by an all doctor executive team motivated by the promise of bringing new therapies to people in need. We are propelled by a deep scientific interest in cellular metabolism and the mitochondria, and are guided by both values and a code of business conduct built upon our commitment to always put the patients, families and communities we serve at the forefront of all that we do.
Common Sensing, Inc. is a Cambridge, Massachusetts-based company that develops and manufactures innovative hardware and software solutions for individuals using injectable medications, particularly in diabetes management. The company's flagship product, Gocap, is a smart cap designed to transform standard injector pens into smart injectors, enabling enhanced connectivity and data tracking. Gocap features include dose logging, temperature monitoring, reminders, and alerts, along with a companion app that provides insights into insulin effects, glucose levels, and meal entries. By integrating these technologies, Common Sensing aims to improve patient education and communication while offering valuable decision support for healthcare providers. Founded in 2012, the company combines expertise from a team of skilled designers and engineers to advance healthcare solutions.
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 committed to addressing unmet medical needs in neurological diseases with the primary goal to arrest the onset of devastating neurological deficits associated with X-ALD.
Omada Health, Inc. is a digital healthcare company that develops online programs aimed at managing chronic conditions. Founded in 2011 and headquartered in San Francisco, with an additional office in Atlanta, the company collaborates with employers and health plans to identify individuals at risk for preventable chronic conditions such as prediabetes, diabetes, hypertension, and heart disease. Omada's programs are personalized to address the evolving needs of participants, offering support in areas like diabetes prevention, type 2 diabetes management, and behavioral health. The company integrates professional health coaching, connected health devices, real-time data, and personalized feedback to achieve clinically significant outcomes. Omada Health is recognized as the largest provider of the National Diabetes Prevention Program by the Centers for Disease Control and Prevention (CDC).
Evidation Health is a provider of a health data analytics platform designed to collect and analyze continuous behavior data and healthcare information so as to create better health outcomes. Its platform turns raw, high-frequency everyday behavior data from sensors, devices, speech, video, and other sources into new knowledge about health and disease, that enable individuals and innovative companies to understand and influence the everyday behaviors.
It was founded in 2012 and is headquartered in San Mateo, California.
Science 37 is making the promise of virtual trials the new reality. By engaging with patients from the comfort of their own home, the company provides access to patients who can never be reached by traditional site-based research models. Its virtual model has proven to enroll faster, retain patients at a higher rate, and reach a more representative patient population. Science 37 has conducted more virtual (or decentralized), interventional trials than any other company, using an expansive, in-house network of telemedicine investigators and home-health nurses. The company's research is powered by the industry’s most comprehensive, fully integrated clinical trial platform purpose-built to conduct virtual trials.
Science 37 is making the promise of virtual trials the new reality. By engaging with patients from the comfort of their own home, the company provides access to patients who can never be reached by traditional site-based research models. Its virtual model has proven to enroll faster, retain patients at a higher rate, and reach a more representative patient population. Science 37 has conducted more virtual (or decentralized), interventional trials than any other company, using an expansive, in-house network of telemedicine investigators and home-health nurses. The company's research is powered by the industry’s most comprehensive, fully integrated clinical trial platform purpose-built to conduct virtual trials.
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 is a developer of novel medicines designed to target specific nutrient-sensing proteins that selectively regulate mTORC1 activity to treat diseases. The company's medicines selectively modulate the activity of mTORC1, a master regulator of cell growth and metabolism, and unlock the therapeutic potential of the mTOR pathway by targeting the amino acid and other nutrient sensors that selectively regulate the activity of mTORC1, enabling healthcare practitioners to address a wide range of metabolic, neurodegenerative, autoimmune and musculoskeletal diseases.
It was founded in 2009 and headquartered in Cambridge, Massachusetts.
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.
Proteostasis Therapeutics is discovering and developing novel small molecule therapeutics designed to control the body's protein homeostasis, or Proteostasis Network. The Proteostasis Network maintains the body's natural balance of proteins to protect us from numerous diseases. These novel therapies, or Proteostasis Regulators, are designed to treat multiple genetic and degenerative disorders associated with deficiencies of the Proteostasis Network, such as emphysema, type II diabetes, Alzheimer's Disease and Huntington's Disease.
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.
Lysosomal Therapeutics, Inc. is a biotechnology company focused on discovering and developing small-molecule therapies for severe neurological diseases. Established in 2011 and based in Cambridge, Massachusetts, the company utilizes its expertise in lysosome-based genetic disorders to create innovative treatment options. It specifically targets conditions such as Gaucher-related neurodegeneration and Parkinson’s disease, along with other synucleinopathies. Through its research and development efforts, Lysosomal Therapeutics aims to provide new solutions for patients suffering from these 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.
Lysosomal Therapeutics, Inc. is a biotechnology company focused on discovering and developing small-molecule therapies for severe neurological diseases. Established in 2011 and based in Cambridge, Massachusetts, the company utilizes its expertise in lysosome-based genetic disorders to create innovative treatment options. It specifically targets conditions such as Gaucher-related neurodegeneration and Parkinson’s disease, along with other synucleinopathies. Through its research and development efforts, Lysosomal Therapeutics aims to provide new solutions for patients suffering from these challenging neurological conditions.
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., an immuno-oncology drug development company, develops and manufactures fusion protein pharmaceuticals for the treatment of cancer and autoimmune diseases. It offers DSP105 and DSP 106 that are used for T-cell mediated tumor destruction; and DSP 107 that targets CD47 tumors activating phagocytes and delivering an immune costimulatory signal. The company was founded in 2005 and is based in Jerusalem, Israel.
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., a biopharmaceutical company, engages in the design and development of small molecule therapeutics to mimic the functions of bioactive carbohydrates. Its products include GMI-1070, a synthetic glycomimetic molecule designed to inhibit selectin types, such as E, P, and L-selectin; and GMI-1051, a small molecular weight compound for the treatment or prevention of infections caused by pseudomonas aeruginosa. The company was founded in 2003 and is based in Gaithersburg, 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 is discovering and developing novel small molecule therapeutics designed to control the body's protein homeostasis, or Proteostasis Network. The Proteostasis Network maintains the body's natural balance of proteins to protect us from numerous diseases. These novel therapies, or Proteostasis Regulators, are designed to treat multiple genetic and degenerative disorders associated with deficiencies of the Proteostasis Network, such as emphysema, type II diabetes, Alzheimer's Disease and Huntington's Disease.
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.