Nimbus Therapeutics is a biotechnology company based in Cambridge, Massachusetts, that specializes in discovering and developing innovative small molecule therapeutics for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company employs a computational technology-driven approach to advance its diverse pipeline of drug candidates into clinical development. Its Nimbus Apollo program includes the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase, which is being investigated for treating non-alcoholic steatohepatitis and hepatocellular carcinoma, among other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, and the company aims to leverage its strategic partnerships to enhance its research and development efforts. Founded in 2009, Nimbus Therapeutics continues to focus on creating highly selective and potent medicines that address unmet medical needs.
MiroBio Ltd is a biotechnology company based in Oxford, United Kingdom, focused on developing antibody modulators for immune cell receptors aimed at treating autoimmune diseases. Founded in 2018, MiroBio leverages pioneering research from the University of Oxford to understand how immune cells communicate and are activated. This foundational knowledge allows the company to create innovative antibodies that stimulate specific immune cell signals, harnessing the body's natural control mechanisms to restore balance within the immune system. By activating these natural pathways, MiroBio's therapeutics aim to provide significant benefits to patients suffering from various autoimmune conditions, enabling clinicians to effectively manage overactive immune responses associated with these diseases.
Dren Bio is a biotechnology company with a portfolio of attractive projects focused on powerful protein engineering technologies for the depletion of cells, protein aggregates, and other disease-causing agents. It is a holding company business model aimed at creating value with multiple projects as stand-alone assets.
Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.
Simcha Therapeutics is a biopharmaceutical company focused on developing engineered cytokine immunotherapies for cancer treatment. Based in New Haven, Connecticut, the company employs directed evolution to create novel cytokines aimed at enhancing the immune system's ability to combat diseases. Its lead program, ST-067, is a designer IL-18 cytokine that has demonstrated significant antitumor activity in animal studies, both as a standalone treatment and in combination with anti-PD-1 checkpoint inhibitors. Founded in 2018, Simcha Therapeutics is dedicated to advancing innovative therapies that empower patients to effectively fight cancer.
Nplex Biosciences is a biotechnology company based in the United States that specializes in proteomic technology. The company has developed a platform that utilizes DNA nanotechnology to understand, detect, and treat diseases. Nplex offers a range of instruments and automation tools for measuring multiple proteins from blood samples, providing cost-effective solutions for protein data analysis. Its technology integrates seamlessly with industry-standard cytometers, enabling high-throughput analysis and the development of custom protein panels. Nplex aims to enhance existing instruments and workflows while facilitating automation in proteomic research and applications.
Odyssey Therapeutics is a biotechnology company pioneering the efficient development of next-generation immunomodulators and oncology medicines. Odyssey is making a transformational impact on the field of drug discovery and is accelerating the path to clinical development to drive the creation of more effective precision medicines.
HotSpot Therapeutics is a developer of a therapy platform designed to discover medicines and provide proper treatment. The company's platform helps to identify hotspots and locate pathway treatments for cancer, metabolic and rare diseases, leverages a database of hotspot structures, harnessing the power of data science in its multidisciplinary approach to drug discovery enabling patients to get treatment against several diseases.
ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in utilizing a proprietary platform that leverages endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in mRNA transcripts. This innovative approach aims to restore the production of functional proteins, addressing various genetic mutations that contribute to diseases. ADARx is developing a pipeline of therapeutics designed to treat a range of conditions, including genetic, cardiometabolic, complement-mediated, and central nervous system diseases, offering potential solutions for previously incurable ailments.
ARS Pharmaceuticals, Inc., founded in 2015 and based in San Diego, California, is a biopharmaceutical company specializing in the development of innovative treatments for severe allergic reactions, including anaphylaxis. The company's flagship product, neffy, is a novel intranasal epinephrine spray that utilizes a proprietary absorption technology known as Intravail. This unique formulation allows for effective, injection-like absorption of epinephrine at a low dose, making it a convenient and reliable option for patients at risk of life-threatening allergic reactions to foods, medications, and insect stings. With its focus on enhancing accessibility and ease of use, ARS Pharmaceuticals aims to improve emergency responses for individuals experiencing severe allergic reactions.
Effector Therapeutics is a biotechnology company based in San Diego, California, that specializes in the development of small molecule drugs aimed at treating cancer and other serious diseases. Founded in 2012, the company focuses on creating selective translation regulators that target the regulation of protein synthesis, a critical process in tumor growth and survival. By selectively blocking dysregulated translation, Effector Therapeutics aims to inhibit cancer cell proliferation while providing a potent and targeted therapeutic approach. This innovative strategy positions the company to potentially deliver new and effective treatments to patients facing challenging health conditions.
Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.
Arcellx is a privately held development-stage company founded in 2015 and located in Montgomery County, Maryland. The Arcellx team is devoted to providing patients with superior immune cell therapies through scientific innovation, accelerated development, and responsible patient care. Although our initial clinical focus is cancer therapy, we are committed to extending our Antigen- Receptor Complex T cell (ARC-T) therapies across a broad spectrum of human disease.
Design Therapeutics, Inc. is a clinical-stage biotechnology company based in Solana Beach, California, focused on developing innovative therapies for degenerative disorders resulting from nucleotide repeat expansions. Founded in 2017, the company is advancing its lead program aimed at treating Friedreich's ataxia, while also exploring treatment options for other conditions such as Fragile X syndrome and myotonic dystrophy. Design Therapeutics is known for its GeneTACTM molecules, a new class of small-molecule gene-targeted therapies designed to address the underlying causes of diseases linked to inherited nucleotide repeat expansion mutations. The company's ongoing research efforts seek to develop disease-modifying treatments for serious conditions driven by these genetic factors.
F-star Therapeutics, Inc., a clinical-stage biopharmaceutical company develops tetravalent bispecific antibodies for cancer therapy. Its medicines used in immuno-oncology treatments. The company plans for the initiation of a Phase I, open label, first-in-human clinical study of FS222, a bispecific antibody targeting CD137 (4-1BB) and PD-L1. The company is based in Cambridge, the United Kingdom.
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.
Dren Bio is a biotechnology company with a portfolio of attractive projects focused on powerful protein engineering technologies for the depletion of cells, protein aggregates, and other disease-causing agents. It is a holding company business model aimed at creating value with multiple projects as stand-alone assets.
NovellusDx, Ltd. is a biotechnology company based in Jerusalem, Israel, established in 2011 by Haim Gil-ad and Yoram Altschuler. The company specializes in developing targeted therapies for cancer patients by focusing on tumor-specific driver mutations. NovellusDx utilizes innovative testing methods to evaluate the effectiveness of targeted therapy drugs on emulated tumors, allowing for the quantification of therapeutic effects. The company also monitors the oncogenic activity of relevant genes and proteins in patients, assessing changes before and after drug administration. Additionally, NovellusDx develops assays to detect the misregulated translocation of mutated signaling proteins to the nucleus, measuring the impact of drug candidates on specific tumors, particularly concerning their ability to inhibit the translocation of up-regulated signaling proteins.
Palleon Pharmaceuticals Inc., a biotechnology company, focuses on developing drugs that target glycan-mediated immune regulation to treat cancer and inflammatory diseases. It offers therapeutics to treat cancer and inflammatory diseases. The company also develops drugs for inflammatory diseases, such as autoimmunity and fibrosis. Palleon Pharmaceuticals Inc. was formerly known as Palleon Pharma Inc. and changed its name to Palleon Pharmaceuticals Inc. in February 2017. The company was founded in 2015 and is based in Waltham, Massachusetts.
Tranquis Therapeutics, Inc., a biotechnology company, engages in developing immuno-therapeutics for neurodegenerative and aging-related diseases. Its TQS-168 is a therapeutic candidate that targets underlying myeloid immune cell dysfunction that is linked to a variety of nervous system disorders. Its solution is used to treat amyotrophic lateral sclerosis, frontotemporal dementia, Parkinson’s disease, and age-related cognitive impairment. The company was incorporated in 2016 and is based in San Mateo, California.
MISSION Therapeutics Ltd. is a biotechnology company based in Cambridge, United Kingdom, focused on discovering and developing small-molecule drugs that selectively target deubiquitylating enzymes (DUBs). The company's pipeline includes promising candidates such as USP30, which is aimed at treating Parkinson's disease and cancer, and USP7, which is recognized as a significant target in oncology. In addition to these, MISSION Therapeutics has various DUB programs that address conditions in oncology, neurodegeneration, rare mitochondrial diseases, kidney disease, and inflammation. Founded in 2011, the company is actively engaged in advancing its innovative drug development platform, which capitalizes on the ubiquitin pathway to enhance protein homeostasis and offers potential therapeutic options in several challenging medical areas.
HotSpot Therapeutics is a developer of a therapy platform designed to discover medicines and provide proper treatment. The company's platform helps to identify hotspots and locate pathway treatments for cancer, metabolic and rare diseases, leverages a database of hotspot structures, harnessing the power of data science in its multidisciplinary approach to drug discovery enabling patients to get treatment against several diseases.
Pandion Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapeutics for autoimmune diseases. The company's lead product candidate, PT101, is an engineered variant of interleukin-2 currently undergoing Phase 1a clinical trials to address various autoimmune and inflammatory conditions. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and two bifunctional molecules, PT001 and PT002, designed to facilitate tissue-selective immunomodulation in the gastrointestinal tract. PT001 combines a PD-1 agonist with a tether module that binds to mucosal vascular addressing cell adhesion molecule (MAdCAM), while PT002 merges an IL-2 mutein effector with a MAdCAM-binding module. The company has also established a collaboration with Astellas Pharma to develop locally acting immunomodulators for pancreatic autoimmune diseases. Headquartered in Watertown, Massachusetts, Pandion Therapeutics aims to create effective and safe treatments to improve patient outcomes in the field of autoimmune disorders.
Design Therapeutics, Inc. is a clinical-stage biotechnology company based in Solana Beach, California, focused on developing innovative therapies for degenerative disorders resulting from nucleotide repeat expansions. Founded in 2017, the company is advancing its lead program aimed at treating Friedreich's ataxia, while also exploring treatment options for other conditions such as Fragile X syndrome and myotonic dystrophy. Design Therapeutics is known for its GeneTACTM molecules, a new class of small-molecule gene-targeted therapies designed to address the underlying causes of diseases linked to inherited nucleotide repeat expansion mutations. The company's ongoing research efforts seek to develop disease-modifying treatments for serious conditions driven by these genetic factors.
MiroBio Ltd is a biotechnology company based in Oxford, United Kingdom, focused on developing antibody modulators for immune cell receptors aimed at treating autoimmune diseases. Founded in 2018, MiroBio leverages pioneering research from the University of Oxford to understand how immune cells communicate and are activated. This foundational knowledge allows the company to create innovative antibodies that stimulate specific immune cell signals, harnessing the body's natural control mechanisms to restore balance within the immune system. By activating these natural pathways, MiroBio's therapeutics aim to provide significant benefits to patients suffering from various autoimmune conditions, enabling clinicians to effectively manage overactive immune responses associated with these diseases.
Arcellx is a privately held development-stage company founded in 2015 and located in Montgomery County, Maryland. The Arcellx team is devoted to providing patients with superior immune cell therapies through scientific innovation, accelerated development, and responsible patient care. Although our initial clinical focus is cancer therapy, we are committed to extending our Antigen- Receptor Complex T cell (ARC-T) therapies across a broad spectrum of human disease.
Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for cancer treatment. Founded in 2015 and headquartered in South San Francisco, California, Nkarta aims to enhance the efficacy and tolerability of cell therapies, addressing both hematologic and solid tumor malignancies. The company's approach utilizes chimeric antigen receptors on NK cells, enabling them to recognize and attack tumor cells more effectively. Nkarta's lead product candidates include NKX101, designed to harness innate NK cell biology for cancer detection and destruction, and NKX019, targeting the CD19 antigen to treat various B cell malignancies. By leveraging its NK expansion platform and proprietary cell engineering technologies, Nkarta seeks to provide a more potent and accessible treatment option, capitalizing on the natural ability of NK cells to identify and eliminate abnormal cells while minimizing side effects typically associated with other therapies.
Turning Point Therapeutics, Inc. is a clinical-stage structure-based drug design company for the discovery and development of precision medicines for cancer and other diseases. We are focusing on the design of novel chemical identities for established oncogene drivers with secondary resistant mutations, newly identified disease-driven targets, and potential targets regulating tumor microenvironment and tumor immunity.
Gotham Therapeutics is a biotechnology company based in New York that focuses on developing a novel class of drugs aimed at targeting epitranscriptomics machinery. Established in 2017, the company seeks to create new treatment options for patients afflicted with various conditions, including cancers, autoimmune disorders, and neurodegenerative diseases. By altering the activity of proteins that modify messenger ribonucleic acid (mRNA), Gotham Therapeutics aims to advance therapeutic strategies and improve patient outcomes in these challenging medical areas.
Morphic Therapeutic is a biotechnology company focused on developing oral small-molecule integrin therapeutics for various medical conditions, including autoimmune diseases, cardiovascular issues, metabolic disorders, fibrosis, and cancer. Its lead candidates in clinical development include MORF-720, an avß6 specific integrin inhibitor targeting idiopathic pulmonary fibrosis and primary sclerosing cholangitis, and MORF-057, an a4ß7 specific integrin inhibitor for inflammatory bowel disease. The company is also exploring additional integrin inhibitors for similar applications. Founded in 2015, Morphic Therapeutic has established collaborations with various organizations, including Engitix Ltd, Schrödinger, LLC, AbbVie Biotechnology Ltd, and Janssen Pharmaceuticals, to advance its research and development efforts. The company is headquartered in Waltham, Massachusetts, and leverages scientific discoveries made by its founder Tim Springer to innovate in the field of integrin therapies.
Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines which are referred to as Bicycles. The Bicycles are synthetic short peptides constrained to form two loops which stabilize their structural geometry. Its initial internal programs are focused on oncology indications with high unmet medical need. The company's product candidate, BT1718, is a Bicycle Toxin Conjugate, or BTC.
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.
Nimbus Therapeutics is a biotechnology company based in Cambridge, Massachusetts, that specializes in discovering and developing innovative small molecule therapeutics for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company employs a computational technology-driven approach to advance its diverse pipeline of drug candidates into clinical development. Its Nimbus Apollo program includes the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase, which is being investigated for treating non-alcoholic steatohepatitis and hepatocellular carcinoma, among other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, and the company aims to leverage its strategic partnerships to enhance its research and development efforts. Founded in 2009, Nimbus Therapeutics continues to focus on creating highly selective and potent medicines that address unmet medical needs.
Propeller Health is a digital health platform focused on improving the management of chronic respiratory diseases such as asthma and COPD. The company provides a mobile application and sensors that attach to inhalers, enabling users to track their medication usage, triggers, and symptoms. This data is stored securely in a cloud-based infrastructure and facilitates personalized feedback and education for users. The platform also allows users to set medication reminders and alerts, as well as share their health data with physicians and family members. Propeller Health collaborates with various healthcare organizations and has received multiple awards for innovation in the field. Founded by David Van Sickle, Greg Tracy, and Mark Gehring, the company operates from Wisconsin, United States.
Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.
Zikani Therapeutics, Inc. develops and commercializes therapeutics for patients with limited treatment options. It offers TURBO-ZM, a platform to develop novel ribosome modulating agents (RMAs) for the treatment of cystic fibrosis, familial adenomatous polyposis, APC mutant colon cancer, recessive dystrophic epidermolysis bullosa, rare cancers, and other rare genetic diseases. Zikani Therapeutics, Inc. was formerly known as Macrolide Pharmaceuticals, Inc. and changed its name to Zikani Therapeutics, Inc. in February 2015. Zikani Therapeutics, Inc. was incorporated in 2014 and is based in Watertown, Massachusetts.
Pandion Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapeutics for autoimmune diseases. The company's lead product candidate, PT101, is an engineered variant of interleukin-2 currently undergoing Phase 1a clinical trials to address various autoimmune and inflammatory conditions. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and two bifunctional molecules, PT001 and PT002, designed to facilitate tissue-selective immunomodulation in the gastrointestinal tract. PT001 combines a PD-1 agonist with a tether module that binds to mucosal vascular addressing cell adhesion molecule (MAdCAM), while PT002 merges an IL-2 mutein effector with a MAdCAM-binding module. The company has also established a collaboration with Astellas Pharma to develop locally acting immunomodulators for pancreatic autoimmune diseases. Headquartered in Watertown, Massachusetts, Pandion Therapeutics aims to create effective and safe treatments to improve patient outcomes in the field of autoimmune disorders.
Atox Bio Inc. is a biotechnology company established in 2003, based in Ness Ziona, Israel, with an additional office in North Carolina. The company specializes in developing immunomodulators aimed at treating critically ill patients suffering from acute and life-threatening conditions due to severe infections and inflammation. Atox Bio focuses on novel modulators of the immune response that target interconnected pathways of both the adaptive and innate immune systems, which are crucial in addressing infectious diseases, inflammatory diseases, and ischemia/reperfusion injuries.
Palleon Pharmaceuticals Inc., a biotechnology company, focuses on developing drugs that target glycan-mediated immune regulation to treat cancer and inflammatory diseases. It offers therapeutics to treat cancer and inflammatory diseases. The company also develops drugs for inflammatory diseases, such as autoimmunity and fibrosis. Palleon Pharmaceuticals Inc. was formerly known as Palleon Pharma Inc. and changed its name to Palleon Pharmaceuticals Inc. in February 2017. The company was founded in 2015 and is based in Waltham, Massachusetts.
Effector Therapeutics is a biotechnology company based in San Diego, California, that specializes in the development of small molecule drugs aimed at treating cancer and other serious diseases. Founded in 2012, the company focuses on creating selective translation regulators that target the regulation of protein synthesis, a critical process in tumor growth and survival. By selectively blocking dysregulated translation, Effector Therapeutics aims to inhibit cancer cell proliferation while providing a potent and targeted therapeutic approach. This innovative strategy positions the company to potentially deliver new and effective treatments to patients facing challenging health conditions.
Turning Point Therapeutics, Inc. is a clinical-stage structure-based drug design company for the discovery and development of precision medicines for cancer and other diseases. We are focusing on the design of novel chemical identities for established oncogene drivers with secondary resistant mutations, newly identified disease-driven targets, and potential targets regulating tumor microenvironment and tumor immunity.
Progyny, Inc. is a benefits management company that focuses on fertility and family building solutions for employers in the United States. The company offers a comprehensive fertility benefits solution that includes personalized concierge-style support, a selective network of high-quality fertility specialists, and innovative treatment plans known as Smart Cycles. In addition to standard fertility services, Progyny provides integrated pharmacy benefits through Progyny Rx, which ensures members have access to necessary medications during treatment. The company also supports surrogacy and adoption expenses, catering to diverse family-building needs. Founded in 2008 and headquartered in New York, Progyny aims to improve fertility treatment outcomes, reduce overall costs, and enhance the fertility experience for employees.
ZappRx, Inc. is a digital health company based in Boston, Massachusetts, that specializes in a cloud-based platform designed to streamline the management of specialty medications. Founded in 2012, the company provides a secure online environment where healthcare providers, specialty pharmacies, and biopharma companies can collaborate efficiently. Its platform automates the prescription and prior authorization processes, facilitating communication among patients, physicians, and pharmacists while reducing reliance on traditional methods such as faxes and phone calls. By consolidating essential information related to specialty prescribing—including pharmacy details, payer requirements, and clinical history—the platform enhances the efficiency and transparency of healthcare delivery, particularly in the fields of pulmonary, cardiology, and gastroenterology. ZappRx operates as a subsidiary of Allscripts Healthcare Solutions, Inc. as of June 2019.
Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.
Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.
Spero Therapeutics, a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializes in developing novel therapies for multi-drug resistant (MDR) bacterial infections and rare diseases. Its key product candidates include tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic intended for adults with MDR Gram-negative infections, and SPR206, an intravenous agent targeting MDR Gram-negative infections in hospital settings. Additionally, the company is advancing SPR720, an oral antibiotic designed for treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has established various collaborations and license agreements to support the development and commercialization of its products, including partnerships with Meiji Seika Pharma, Everest Medicines, and the Bill & Melinda Gates Medical Research Institute. Founded in 2013, Spero aims to address the critical need for effective treatments against serious bacterial infections using innovative approaches and mechanisms.
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.
Propeller Health is a digital health platform focused on improving the management of chronic respiratory diseases such as asthma and COPD. The company provides a mobile application and sensors that attach to inhalers, enabling users to track their medication usage, triggers, and symptoms. This data is stored securely in a cloud-based infrastructure and facilitates personalized feedback and education for users. The platform also allows users to set medication reminders and alerts, as well as share their health data with physicians and family members. Propeller Health collaborates with various healthcare organizations and has received multiple awards for innovation in the field. Founded by David Van Sickle, Greg Tracy, and Mark Gehring, the company operates from Wisconsin, United States.
True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.
Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines which are referred to as Bicycles. The Bicycles are synthetic short peptides constrained to form two loops which stabilize their structural geometry. Its initial internal programs are focused on oncology indications with high unmet medical need. The company's product candidate, BT1718, is a Bicycle Toxin Conjugate, or BTC.
Arch Oncology is an immuno-oncology company focused on developing anti-CD47 antibodies for the treatment of solid and hematologic cancers. Founded in 2006 and based in St. Louis, Missouri, with a corporate office in Brisbane, California, the company aims to create innovative therapies that leverage both adaptive and innate immune responses. Arch Oncology's approach involves a new class of checkpoint inhibitors, which represents a significant advancement in cancer treatment. Through its research and development efforts, the company seeks to improve outcomes for patients battling various forms of cancer.
Progyny, Inc. is a benefits management company that focuses on fertility and family building solutions for employers in the United States. The company offers a comprehensive fertility benefits solution that includes personalized concierge-style support, a selective network of high-quality fertility specialists, and innovative treatment plans known as Smart Cycles. In addition to standard fertility services, Progyny provides integrated pharmacy benefits through Progyny Rx, which ensures members have access to necessary medications during treatment. The company also supports surrogacy and adoption expenses, catering to diverse family-building needs. Founded in 2008 and headquartered in New York, Progyny aims to improve fertility treatment outcomes, reduce overall costs, and enhance the fertility experience for employees.
Morphic Therapeutic is a biotechnology company focused on developing oral small-molecule integrin therapeutics for various medical conditions, including autoimmune diseases, cardiovascular issues, metabolic disorders, fibrosis, and cancer. Its lead candidates in clinical development include MORF-720, an avß6 specific integrin inhibitor targeting idiopathic pulmonary fibrosis and primary sclerosing cholangitis, and MORF-057, an a4ß7 specific integrin inhibitor for inflammatory bowel disease. The company is also exploring additional integrin inhibitors for similar applications. Founded in 2015, Morphic Therapeutic has established collaborations with various organizations, including Engitix Ltd, Schrödinger, LLC, AbbVie Biotechnology Ltd, and Janssen Pharmaceuticals, to advance its research and development efforts. The company is headquartered in Waltham, Massachusetts, and leverages scientific discoveries made by its founder Tim Springer to innovate in the field of integrin therapies.
Second Genome’s mission is to transform lives with medicines developed through innovative microbiome science. Second Genome has built a novel platform for microbiome drug discovery. Second Genome has completed more than 400 microbiome studies, analyzing more than 75,000 samples, for internal R&D, as well as for external partners across government, academia, pharmaceutical, nutrition and industrial companies. The team leverages its microbiome analysis platform with its partners’ specific expertise to generate insightful findings that can accelerate research programs by elucidating the role of the microbiome in human health conditions, agriculture, animal health and other industries. The company has established a pipeline of therapeutic products for the treatment of inflammation and metabolic diseases.
Spero Therapeutics, a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializes in developing novel therapies for multi-drug resistant (MDR) bacterial infections and rare diseases. Its key product candidates include tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic intended for adults with MDR Gram-negative infections, and SPR206, an intravenous agent targeting MDR Gram-negative infections in hospital settings. Additionally, the company is advancing SPR720, an oral antibiotic designed for treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has established various collaborations and license agreements to support the development and commercialization of its products, including partnerships with Meiji Seika Pharma, Everest Medicines, and the Bill & Melinda Gates Medical Research Institute. Founded in 2013, Spero aims to address the critical need for effective treatments against serious bacterial infections using innovative approaches and mechanisms.
MISSION Therapeutics Ltd. is a biotechnology company based in Cambridge, United Kingdom, focused on discovering and developing small-molecule drugs that selectively target deubiquitylating enzymes (DUBs). The company's pipeline includes promising candidates such as USP30, which is aimed at treating Parkinson's disease and cancer, and USP7, which is recognized as a significant target in oncology. In addition to these, MISSION Therapeutics has various DUB programs that address conditions in oncology, neurodegeneration, rare mitochondrial diseases, kidney disease, and inflammation. Founded in 2011, the company is actively engaged in advancing its innovative drug development platform, which capitalizes on the ubiquitin pathway to enhance protein homeostasis and offers potential therapeutic options in several challenging medical areas.
Effector Therapeutics is a biotechnology company based in San Diego, California, that specializes in the development of small molecule drugs aimed at treating cancer and other serious diseases. Founded in 2012, the company focuses on creating selective translation regulators that target the regulation of protein synthesis, a critical process in tumor growth and survival. By selectively blocking dysregulated translation, Effector Therapeutics aims to inhibit cancer cell proliferation while providing a potent and targeted therapeutic approach. This innovative strategy positions the company to potentially deliver new and effective treatments to patients facing challenging health conditions.
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.
True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.
Puridify Limited is a bioprocessing company based in Stevenage, United Kingdom, established in 2013. It specializes in the development of innovative purification technologies aimed at enhancing the manufacturing of industrial biomolecules. The company has created FibroSelect, a nanofiber-based platform purification technology that addresses limitations associated with traditional packed bed chromatography systems and chromatography membranes. This technology is designed to provide consistent purification performance across various scales, making it particularly suitable for the purification of macromolecular biotherapeutics such as gene therapies, viral vectors, and fusion proteins. By improving purification efficiencies, Puridify enables the production of cost-effective biomolecule products, supporting advancements in the biopharmaceutical sector. As of late 2017, Puridify operates as a subsidiary of GE Healthcare Limited.
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.
Asceneuron SA is a biotechnology company based in Lausanne, Switzerland, founded in 2012, that focuses on discovering and developing therapeutics for neurodegenerative disorders with significant unmet medical needs. The company targets conditions such as progressive supranuclear palsy, orphan tauopathies, Alzheimer’s disease, and Parkinson’s disease. Asceneuron aims to develop orally bioavailable small molecule drugs that address both symptomatic relief and disease progression for these debilitating conditions. Tauopathies, characterized by the accumulation of tau protein deposits in the brain, are a key area of focus, as they currently lack effective treatments. With the increasing prevalence of Alzheimer's disease posing a major healthcare challenge, Asceneuron aspires to become a leading entity in the biotech sector by providing innovative and more effective therapeutic options for patients suffering from neurodegenerative diseases. The company operates as a subsidiary of Merck KGaA.
Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.
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.
Gladius Pharmaceuticals Inc., a Montréal-based biotechnology firm developing novel drugs to treat life-threatening, multi-drug-resistant bacterial infections.Gladius is a disruptive platform technology play, capable of developing multiple novel drug candidates
Spero Therapeutics, a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializes in developing novel therapies for multi-drug resistant (MDR) bacterial infections and rare diseases. Its key product candidates include tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic intended for adults with MDR Gram-negative infections, and SPR206, an intravenous agent targeting MDR Gram-negative infections in hospital settings. Additionally, the company is advancing SPR720, an oral antibiotic designed for treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has established various collaborations and license agreements to support the development and commercialization of its products, including partnerships with Meiji Seika Pharma, Everest Medicines, and the Bill & Melinda Gates Medical Research Institute. Founded in 2013, Spero aims to address the critical need for effective treatments against serious bacterial infections using innovative approaches and mechanisms.
Akamis Bio is a biotechnology company dedicated to developing innovative gene-based immuno-oncology therapies for cancer treatment. The company focuses on discovering and advancing a range of candidate therapies, including those utilizing its proprietary T-SIGn virus platform, which allows for systemic delivery while exerting localized effects within tumors. Akamis Bio aims to become a leader in cancer gene therapy, emphasizing the importance of creating medicines that provide tangible benefits to patients. The company is actively engaged in both early and clinical stage development of its therapies and seeks to establish strategic partnerships with other leaders in the immuno-oncology field to enhance its product offerings and accelerate the availability of its treatments.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.
ZappRx, Inc. is a digital health company based in Boston, Massachusetts, that specializes in a cloud-based platform designed to streamline the management of specialty medications. Founded in 2012, the company provides a secure online environment where healthcare providers, specialty pharmacies, and biopharma companies can collaborate efficiently. Its platform automates the prescription and prior authorization processes, facilitating communication among patients, physicians, and pharmacists while reducing reliance on traditional methods such as faxes and phone calls. By consolidating essential information related to specialty prescribing—including pharmacy details, payer requirements, and clinical history—the platform enhances the efficiency and transparency of healthcare delivery, particularly in the fields of pulmonary, cardiology, and gastroenterology. ZappRx operates as a subsidiary of Allscripts Healthcare Solutions, Inc. as of June 2019.
Thrasos is a bio-therapeutics company dedicated to discovering and developing targeted therapies for severe organ failure, with a primary emphasis on kidney diseases. The company's lead program, THR-184, aims to prevent and treat acute kidney injury (AKI). Additionally, Thrasos is advancing a preclinical development program that addresses chronic kidney disease (CKD). The company also focuses on developing innovative treatments for rare kidney-related conditions, such as myeloma cast nephropathy, which occurs when protein aggregates obstruct renal tubules, leading to the risk of permanent kidney damage. Through its efforts, Thrasos seeks to provide timely and effective treatment options for patients suffering from these serious kidney issues.
Nimbus Therapeutics is a biotechnology company based in Cambridge, Massachusetts, that specializes in discovering and developing innovative small molecule therapeutics for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company employs a computational technology-driven approach to advance its diverse pipeline of drug candidates into clinical development. Its Nimbus Apollo program includes the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase, which is being investigated for treating non-alcoholic steatohepatitis and hepatocellular carcinoma, among other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, and the company aims to leverage its strategic partnerships to enhance its research and development efforts. Founded in 2009, Nimbus Therapeutics continues to focus on creating highly selective and potent medicines that address unmet medical needs.
Zikani Therapeutics, Inc. develops and commercializes therapeutics for patients with limited treatment options. It offers TURBO-ZM, a platform to develop novel ribosome modulating agents (RMAs) for the treatment of cystic fibrosis, familial adenomatous polyposis, APC mutant colon cancer, recessive dystrophic epidermolysis bullosa, rare cancers, and other rare genetic diseases. Zikani Therapeutics, Inc. was formerly known as Macrolide Pharmaceuticals, Inc. and changed its name to Zikani Therapeutics, Inc. in February 2015. Zikani Therapeutics, Inc. was incorporated in 2014 and is based in Watertown, Massachusetts.
Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for cancer treatment. Founded in 2015 and headquartered in South San Francisco, California, Nkarta aims to enhance the efficacy and tolerability of cell therapies, addressing both hematologic and solid tumor malignancies. The company's approach utilizes chimeric antigen receptors on NK cells, enabling them to recognize and attack tumor cells more effectively. Nkarta's lead product candidates include NKX101, designed to harness innate NK cell biology for cancer detection and destruction, and NKX019, targeting the CD19 antigen to treat various B cell malignancies. By leveraging its NK expansion platform and proprietary cell engineering technologies, Nkarta seeks to provide a more potent and accessible treatment option, capitalizing on the natural ability of NK cells to identify and eliminate abnormal cells while minimizing side effects typically associated with other therapies.
Avhana Health Inc. provides an application to simplify the healthcare provider workflow by offering the latest guidelines, actionable recommendations, and performance metrics—directly in their EHR. Its application features a dashboard for pre-visit planning, patient-specific recommendations for care, corresponding order suggestions that submit to the EHR, and a dashboard for tracking provider activity and performance. The company was founded in 2014 and is based in Baltimore, Maryland.
Aileron Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing and commercializing a novel class of drugs known as stabilized cell-permeating alpha-helical peptides, particularly in the field of oncology. The company’s lead drug candidate, ALRN-6924, is currently undergoing multiple clinical trials, including Phase 2a studies for advanced solid tumors and peripheral T-cell lymphoma, as well as Phase 1 trials for acute myeloid leukemia and advanced myelodysplastic syndrome. Aileron is also working on a next-generation wild type p53 reactivator. Collaborations with notable institutions, such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer, further enhance its research efforts, particularly in evaluating ALRN-6924 in combination therapies. Aileron Therapeutics was founded in 2001, originally named Renegade Therapeutics, and is headquartered in Watertown, Massachusetts.
Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines which are referred to as Bicycles. The Bicycles are synthetic short peptides constrained to form two loops which stabilize their structural geometry. Its initial internal programs are focused on oncology indications with high unmet medical need. The company's product candidate, BT1718, is a Bicycle Toxin Conjugate, or BTC.
Atopix Therapeutics Limited is a clinical-stage biopharmaceutical company based in Oxford, United Kingdom, focused on drug discovery for anti-inflammatory medicines. Founded in 2012, the company develops innovative oral treatments, specifically CRTH2 antagonists, aimed at addressing asthma, chronic allergic conditions, and various atopic diseases, including atopic dermatitis and allergic rhinitis. The lead candidate from Atopix is undergoing a Phase 2 clinical trial for moderate to severe atopic dermatitis at multiple prominent dermatology centers across Europe. As of December 2016, Atopix operates as a subsidiary of Chiesi Farmaceutici S.p.A.
Atox Bio Inc. is a biotechnology company established in 2003, based in Ness Ziona, Israel, with an additional office in North Carolina. The company specializes in developing immunomodulators aimed at treating critically ill patients suffering from acute and life-threatening conditions due to severe infections and inflammation. Atox Bio focuses on novel modulators of the immune response that target interconnected pathways of both the adaptive and innate immune systems, which are crucial in addressing infectious diseases, inflammatory diseases, and ischemia/reperfusion injuries.
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.
True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.
Puridify Limited is a bioprocessing company based in Stevenage, United Kingdom, established in 2013. It specializes in the development of innovative purification technologies aimed at enhancing the manufacturing of industrial biomolecules. The company has created FibroSelect, a nanofiber-based platform purification technology that addresses limitations associated with traditional packed bed chromatography systems and chromatography membranes. This technology is designed to provide consistent purification performance across various scales, making it particularly suitable for the purification of macromolecular biotherapeutics such as gene therapies, viral vectors, and fusion proteins. By improving purification efficiencies, Puridify enables the production of cost-effective biomolecule products, supporting advancements in the biopharmaceutical sector. As of late 2017, Puridify operates as a subsidiary of GE Healthcare Limited.
Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.
Spero Therapeutics, a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializes in developing novel therapies for multi-drug resistant (MDR) bacterial infections and rare diseases. Its key product candidates include tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic intended for adults with MDR Gram-negative infections, and SPR206, an intravenous agent targeting MDR Gram-negative infections in hospital settings. Additionally, the company is advancing SPR720, an oral antibiotic designed for treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has established various collaborations and license agreements to support the development and commercialization of its products, including partnerships with Meiji Seika Pharma, Everest Medicines, and the Bill & Melinda Gates Medical Research Institute. Founded in 2013, Spero aims to address the critical need for effective treatments against serious bacterial infections using innovative approaches and mechanisms.
Alios is discovering and developing novel therapeutic agents based on three platform technologies including: small molecule activators of innate immunity antiviral pathways (RNase L activation), phosphate protected nucleotide prodrug chemistry, and glycoprotein-engineering of interferons (Glycoferonâ). This complementary group of platform technologies has the potential to generate a number of distinct therapeutic products to treat a variety of serious viral infections such as chronic hepatitis B and C, HIV infection, and respiratory viruses (e.g. pandemic influenza) and emerging viral diseases (e.g. SARS).
Bird Rock Bio is focused on the discovery and development of novel biologic therapeutics that meet the medical and commercial needs for China’s patients and healthcare system and that have the potential to be disruptive globally. RYI-008 is a novel, extremely potent monoclonal antibody that is highly selective to IL-6, a cytokine widely implicated in inflammation and cancer. In addition to RYI-008, RuiYi has a growing pipeline of monoclonal antibodies from its internal discovery efforts utilizing a series of the Company’s technologies, including the iCAP (intramembranous Conformation Antigen Presenting) system. Targets include a select subset of G protein coupled receptors (GPCR), where specificity in binding is critical but has proven difficult to achieve with small molecule modulators.
ZappRx, Inc. is a digital health company based in Boston, Massachusetts, that specializes in a cloud-based platform designed to streamline the management of specialty medications. Founded in 2012, the company provides a secure online environment where healthcare providers, specialty pharmacies, and biopharma companies can collaborate efficiently. Its platform automates the prescription and prior authorization processes, facilitating communication among patients, physicians, and pharmacists while reducing reliance on traditional methods such as faxes and phone calls. By consolidating essential information related to specialty prescribing—including pharmacy details, payer requirements, and clinical history—the platform enhances the efficiency and transparency of healthcare delivery, particularly in the fields of pulmonary, cardiology, and gastroenterology. ZappRx operates as a subsidiary of Allscripts Healthcare Solutions, Inc. as of June 2019.
VHsquared Ltd. develops oral biologics for immuno-inflammatory targets in the gastro intestinal tract. The company identifies, screens, selects, and engineers Vorabodies, a transformational therapy for the treatment of inflammatory bowel disease. It uses its Vorabody platform to generate potent protease resistant domain antibodies for oral administration. The company was incorporated in 2010 and is based in Cambridge, United Kingdom.
MISSION Therapeutics Ltd. is a biotechnology company based in Cambridge, United Kingdom, focused on discovering and developing small-molecule drugs that selectively target deubiquitylating enzymes (DUBs). The company's pipeline includes promising candidates such as USP30, which is aimed at treating Parkinson's disease and cancer, and USP7, which is recognized as a significant target in oncology. In addition to these, MISSION Therapeutics has various DUB programs that address conditions in oncology, neurodegeneration, rare mitochondrial diseases, kidney disease, and inflammation. Founded in 2011, the company is actively engaged in advancing its innovative drug development platform, which capitalizes on the ubiquitin pathway to enhance protein homeostasis and offers potential therapeutic options in several challenging medical areas.
Aileron Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing and commercializing a novel class of drugs known as stabilized cell-permeating alpha-helical peptides, particularly in the field of oncology. The company’s lead drug candidate, ALRN-6924, is currently undergoing multiple clinical trials, including Phase 2a studies for advanced solid tumors and peripheral T-cell lymphoma, as well as Phase 1 trials for acute myeloid leukemia and advanced myelodysplastic syndrome. Aileron is also working on a next-generation wild type p53 reactivator. Collaborations with notable institutions, such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer, further enhance its research efforts, particularly in evaluating ALRN-6924 in combination therapies. Aileron Therapeutics was founded in 2001, originally named Renegade Therapeutics, and is headquartered in Watertown, Massachusetts.
F-star Therapeutics, Inc., a clinical-stage biopharmaceutical company develops tetravalent bispecific antibodies for cancer therapy. Its medicines used in immuno-oncology treatments. The company plans for the initiation of a Phase I, open label, first-in-human clinical study of FS222, a bispecific antibody targeting CD137 (4-1BB) and PD-L1. The company is based in Cambridge, the United Kingdom.
iPierian is engaged in the research and development of therapies aimed at treating neurodegenerative diseases, including spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), Parkinson's disease, and Alzheimer's disease. The company utilizes induced pluripotent stem cells to facilitate drug discovery and development, focusing on addressing significant unmet medical needs. In particular, iPierian is developing therapies and monoclonal antibodies that target Tauopathies, which are diseases characterized by the accumulation of tau protein in the brain. These therapies aim to slow the spread of tau throughout the brain, thereby inhibiting the progression of associated diseases.
Dicerna Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of innovative treatments primarily for liver-related diseases, including rare inherited conditions, chronic liver diseases, and viral infections. Utilizing its proprietary GalXC RNA interference technology, Dicerna focuses on silencing disease-causing genes to address various health issues. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and belcesiran for genetic liver disease, among others. The company's extensive research efforts also encompass treatments for cardiovascular diseases and neurodegeneration. Dicerna has established strategic collaborations with prominent pharmaceutical companies to enhance its development capabilities. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna is at the forefront of advancing RNA-based therapeutics.
Effector Therapeutics is a biotechnology company based in San Diego, California, that specializes in the development of small molecule drugs aimed at treating cancer and other serious diseases. Founded in 2012, the company focuses on creating selective translation regulators that target the regulation of protein synthesis, a critical process in tumor growth and survival. By selectively blocking dysregulated translation, Effector Therapeutics aims to inhibit cancer cell proliferation while providing a potent and targeted therapeutic approach. This innovative strategy positions the company to potentially deliver new and effective treatments to patients facing challenging health conditions.
Aileron Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing and commercializing a novel class of drugs known as stabilized cell-permeating alpha-helical peptides, particularly in the field of oncology. The company’s lead drug candidate, ALRN-6924, is currently undergoing multiple clinical trials, including Phase 2a studies for advanced solid tumors and peripheral T-cell lymphoma, as well as Phase 1 trials for acute myeloid leukemia and advanced myelodysplastic syndrome. Aileron is also working on a next-generation wild type p53 reactivator. Collaborations with notable institutions, such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer, further enhance its research efforts, particularly in evaluating ALRN-6924 in combination therapies. Aileron Therapeutics was founded in 2001, originally named Renegade Therapeutics, and is headquartered in Watertown, Massachusetts.
River Vision Development
Series A in 2012
River Vision Development develops RV001, a protein therapy for the treatment of Graves’ Orbitopathy (GO). River Vision Development was founded in 2011 and is based in New York, New York.
Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines which are referred to as Bicycles. The Bicycles are synthetic short peptides constrained to form two loops which stabilize their structural geometry. Its initial internal programs are focused on oncology indications with high unmet medical need. The company's product candidate, BT1718, is a Bicycle Toxin Conjugate, or BTC.
Applied Genetic Technologies Corporation is a clinical-stage biotechnology company focused on developing innovative genetic therapies for patients with rare and debilitating diseases. The company is advancing several product candidates in ophthalmology, including treatments for X-linked retinitis pigmentosa, which has completed Phase I/II clinical trials, and achromatopsia, currently in Phase I/II trials. Additionally, AGTC is working on an optogenetic therapy for patients with advanced retinal disease. The company has also initiated a preclinical program in otology and three preclinical programs targeting central nervous system disorders such as frontotemporal dementia, amyotrophic lateral sclerosis, and adrenoleukodystrophy. Collaborations with various organizations, including Synpromics Limited and the University of Florida, support its research efforts. Founded in 1999 and based in Alachua, Florida, AGTC employs advanced gene therapy techniques, aiming to replace defective genes with functional ones, thus improving patient outcomes with long-lasting treatments.
Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.