Nimbus Apollo is a program under Nimbus Therapeutics that focuses on the development of innovative treatments for serious diseases, particularly targeting non-alcoholic steatohepatitis (NASH) and hepatocellular carcinoma (HCC). The program's lead candidate, NDI-010976, is an allosteric inhibitor of Acetyl-CoA Carboxylase (ACC), which has received Fast Track designation from the U.S. Food and Drug Administration. Nimbus Apollo is engaged in advancing this candidate through various stages of clinical development, with Phase 1 data expected to be presented at notable medical conferences. Nimbus Therapeutics, founded in 2009 and based in Cambridge, Massachusetts, leverages computational technology to design selective and potent small molecule therapeutics aimed at addressing metabolic diseases, cancer, and immune-inflammatory disorders. The company has established strategic partnerships to enhance its research and development efforts.
MiroBio Ltd develops and manufactures antibody modulators of immune cell receptors for auto-immune diseases. The company is developing antibodies and variants to
stimulate specific immune cell signals to harness the natural control mechanisms of the immune system. The company was incorporated in 2018 and is headquartered in Oxford, United Kingdom.
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., a private, clinical-stage biopharmaceutical company founded by Catalys Pacific committed to developing best-in-class, novel therapy for the treatment of hypertension. The Company is driven to bring a targeted approach to the management of hypertension via the development of MLS-101. MLS-101, licensed from Mitsubishi Tanabe Pharma Corporation, is a highly selective and potent aldosterone synthase inhibitor that is being investigated for the treatment of hypertension.
Simcha Therapeutics is a biopharmaceutical company developing engineered cytokine immunotherapy for cancer. The company uses directed evolution to engineer novel cytokines designed to unlock the precision and power of the immune system. Simcha’s lead program, ST-067, is a designer IL-18 cytokine that has shown potent antitumor effects in animal models, both as a monotherapy and when combined with anti-PD-1 checkpoint inhibitors, as described in Nature in June 2020. It was founded in 2018 and is headquartered in New Haven, Connecticut, USA.
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 is a genetic medicine company focusing on the base editing of mRNA transcripts. It discovers and develops innovative therapeutics that can utilize a family of endogenous enzymes called adenosine deaminase acting on RNA (ADAR) to precise target and correct single point mutations on an mRNA, thus the production of desired and functional protein is restored.
It was founded in 2019 and is headquartered in San Diego, California.
ARS Pharmaceuticals is a stealth pharmaceutical company that developed a novel intranasal epinephrine spray with a unique absorption technology that could be easy-to-use, convenient, and more reliable for patients and loved ones at risk of severe allergic reactions to food, medications and insect bites that could lead to life-threatening anaphylaxis.
Effector Therapeutics, Inc. is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of small molecule drugs designed to treat cancer and other serious diseases by selectively regulating protein synthesis, specifically targeting mRNA translation. Effector Therapeutics focuses on developing translation regulators that inhibit disregulated translation, which is crucial for tumor growth and survival. This innovative approach aims to provide potent and selective therapeutic options, offering a promising avenue for delivering new cancer treatments to patients in need.
Pulmocide Ltd, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.
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. develops therapies for the treatment of degenerative disorders caused by nucleotide repeat expansions. The company engages in the development of a program for the treatment of Friedreich’s ataxia and degenerative diseases such as Fragile X syndrome and myotonic dystrophy. The company was incorporated in 2017 and is based in Solana Beach, California.
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 Operations, Inc. is a clinical-stage biotechnology company focused on developing therapeutics for autoimmune diseases. Its lead product candidate, PT101, is an engineered variant of interleukin-2 (IL-2) currently undergoing Phase 1a clinical trials for various autoimmune and inflammatory conditions. Other product candidates include PT627, a systemic PD-1 agonist in preclinical studies; PT001, a bifunctional molecule designed to achieve tissue-selective immunomodulation in the gastrointestinal tract; and PT002, which combines an IL-2 mutein with a tether module for similar localized effects. The company collaborates with Astellas Pharma Inc. to develop immunomodulators aimed at autoimmune diseases specifically affecting the pancreas. Headquartered in Watertown, Massachusetts, Pandion Operations seeks to create innovative therapies that can provide durable and tissue-specific treatment options for patients suffering from these conditions.
Design Therapeutics, Inc. develops therapies for the treatment of degenerative disorders caused by nucleotide repeat expansions. The company engages in the development of a program for the treatment of Friedreich’s ataxia and degenerative diseases such as Fragile X syndrome and myotonic dystrophy. The company was incorporated in 2017 and is based in Solana Beach, California.
MiroBio Ltd develops and manufactures antibody modulators of immune cell receptors for auto-immune diseases. The company is developing antibodies and variants to
stimulate specific immune cell signals to harness the natural control mechanisms of the immune system. The company was incorporated in 2018 and is headquartered in Oxford, United Kingdom.
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, Inc., a biopharmaceutical company, develops and commercializes cell therapies for cancer treatment. The company’s approach for cellular immunotherapy involves chimeric antigen receptors on the surface of a natural killer (NK) cell that enable the cell to recognize specific proteins or antigens that are present on the surface of tumor cells. Its co-lead product candidates are NKX101, a pre-clinical product that enhances the power of innate NK biology to detect and kill cancerous cells; and NKX019, a pre-clinical product, which is based on the ability to treat various B cell malignancies by targeting the CD19 antigen found on these types of cancerous cells. The company was founded in 2015 and is headquartered in South San Francisco, California.
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 Apollo is a program under Nimbus Therapeutics that focuses on the development of innovative treatments for serious diseases, particularly targeting non-alcoholic steatohepatitis (NASH) and hepatocellular carcinoma (HCC). The program's lead candidate, NDI-010976, is an allosteric inhibitor of Acetyl-CoA Carboxylase (ACC), which has received Fast Track designation from the U.S. Food and Drug Administration. Nimbus Apollo is engaged in advancing this candidate through various stages of clinical development, with Phase 1 data expected to be presented at notable medical conferences. Nimbus Therapeutics, founded in 2009 and based in Cambridge, Massachusetts, leverages computational technology to design selective and potent small molecule therapeutics aimed at addressing metabolic diseases, cancer, and immune-inflammatory disorders. The company has established strategic partnerships to enhance its research and development efforts.
Propeller Health is a mobile platform that offers sensors, mobile apps, analytics, and services to support respiratory health management. The HIPAA compliant platform stores users’ data in a cloud computing infrastructure. The Propeller sensor and mobile application help users learn more about and better manage their asthma, COPD, and respiratory disease. The Propeller sensor attaches to users’ inhalers and wirelessly syncs with their smartphones. It tracks their triggers and symptoms and sends personalized feedback and education to their phones based on the symptoms. Users can see how often they use the medication, and set up medication reminders and alerts. Propeller community enables users to share their Propeller data with their physician and family members. Propeller Health is backed by Safeguard Scientifics, The Social+Capital Partnership, California HealthCare Foundation, and other investors. The platform works with organizations such as Dignity Health, Wyckoff Heights Medical Center, Amerigroup Florida, and the City of Louisville. The system has been featured in The Economist, Washington Post, Fast Company, Wired, and Scientific American. Propeller Health has received TEDMED Innovation Showcase, White House Champion of Change, and Bluetooth Breakthrough Product awards. Propeller Health was launched by David Van Sickle, Greg Tracy, and Mark Gehring and its operations are based in 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 are among the most widely used, most effective, and safest antibiotics to reach clinical practice; in fact, azithromycin has been among the world’s most widely used antibiotics for the past several years. But, while current macrolides are highly effective against some major bacteria, they are ineffective against others, especially gram-negative bacteria, the cause of most serious infections in the US. The first three generations of macrolides have very limited activity against gram-negative, and the sole drug in the current fourth-generation, solithromycin, has only slightly greater activity. Within other antibiotic classes, a limited number of compounds are in development to treat resistant gram-negative infections, and even fewer are available both intravenously and orally, as would be expected with macrolides. The company was founded in 2014 and is headquartered in Newton, Massachusetts.
Pandion Operations, Inc. is a clinical-stage biotechnology company focused on developing therapeutics for autoimmune diseases. Its lead product candidate, PT101, is an engineered variant of interleukin-2 (IL-2) currently undergoing Phase 1a clinical trials for various autoimmune and inflammatory conditions. Other product candidates include PT627, a systemic PD-1 agonist in preclinical studies; PT001, a bifunctional molecule designed to achieve tissue-selective immunomodulation in the gastrointestinal tract; and PT002, which combines an IL-2 mutein with a tether module for similar localized effects. The company collaborates with Astellas Pharma Inc. to develop immunomodulators aimed at autoimmune diseases specifically affecting the pancreas. Headquartered in Watertown, Massachusetts, Pandion Operations seeks to create innovative therapies that can provide durable and tissue-specific treatment options for patients suffering from these conditions.
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, Inc. is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of small molecule drugs designed to treat cancer and other serious diseases by selectively regulating protein synthesis, specifically targeting mRNA translation. Effector Therapeutics focuses on developing translation regulators that inhibit disregulated translation, which is crucial for tumor growth and survival. This innovative approach aims to provide potent and selective therapeutic options, offering a promising avenue for delivering new cancer treatments to patients in need.
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 is a fertility benefits management company that combines clinical and emotional guidance and support, science, technology, and data to provide comprehensive, inclusive, and effective value-based fertility solutions for self-insured employers.
Progyny’s benefit plans are designed to improve outcomes, shorten time to pregnancy and reduce total fertility-related costs. Progyny is privately held and is headquartered in New York, NY. Please visit www.progyny.com.
ZappRx, Inc. is a digital health company based in Boston, Massachusetts, focused on modernizing the management of specialty medications. Its cloud-based prescription and prior authorization platform enhances efficiency for healthcare providers, specialty pharmacies, and biopharma companies by streamlining the ordering process for specialty drugs. The platform facilitates secure, collaborative interactions among providers, pharmacists, and payers, significantly reducing reliance on traditional communication methods like faxes and phone calls. ZappRx's services are particularly utilized in pulmonary, cardiology, and gastroenterology fields. The company was incorporated in 2012 and, as of June 2019, operates as a subsidiary of Allscripts Healthcare Solutions, Inc.
Pulmocide Ltd, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.
Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of novel treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. The company's key product candidates include tebipenem pivoxil hydrobromide, an oral antibiotic aimed at treating MDR gram-negative infections in adults; SPR206, an intravenous agent targeting MDR gram-negative infections in hospital settings; and SPR720, an oral antibiotic for pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has entered various agreements, including a licensing partnership with Meiji Seika Pharma to develop tebipenem HBr and collaborations with Everest Medicines and the Bill & Melinda Gates Medical Research Institute for the development and commercialization of SPR206 and SPR720, respectively. Founded in 2013, Spero Therapeutics aims to address urgent medical needs through innovative therapeutic solutions for serious bacterial infections.
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 mobile platform that offers sensors, mobile apps, analytics, and services to support respiratory health management. The HIPAA compliant platform stores users’ data in a cloud computing infrastructure. The Propeller sensor and mobile application help users learn more about and better manage their asthma, COPD, and respiratory disease. The Propeller sensor attaches to users’ inhalers and wirelessly syncs with their smartphones. It tracks their triggers and symptoms and sends personalized feedback and education to their phones based on the symptoms. Users can see how often they use the medication, and set up medication reminders and alerts. Propeller community enables users to share their Propeller data with their physician and family members. Propeller Health is backed by Safeguard Scientifics, The Social+Capital Partnership, California HealthCare Foundation, and other investors. The platform works with organizations such as Dignity Health, Wyckoff Heights Medical Center, Amerigroup Florida, and the City of Louisville. The system has been featured in The Economist, Washington Post, Fast Company, Wired, and Scientific American. Propeller Health has received TEDMED Innovation Showcase, White House Champion of Change, and Bluetooth Breakthrough Product awards. Propeller Health was launched by David Van Sickle, Greg Tracy, and Mark Gehring and its operations are based in Wisconsin, United States.
True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
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 is a fertility benefits management company that combines clinical and emotional guidance and support, science, technology, and data to provide comprehensive, inclusive, and effective value-based fertility solutions for self-insured employers.
Progyny’s benefit plans are designed to improve outcomes, shorten time to pregnancy and reduce total fertility-related costs. Progyny is privately held and is headquartered in New York, NY. Please visit www.progyny.com.
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.
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.
Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of novel treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. The company's key product candidates include tebipenem pivoxil hydrobromide, an oral antibiotic aimed at treating MDR gram-negative infections in adults; SPR206, an intravenous agent targeting MDR gram-negative infections in hospital settings; and SPR720, an oral antibiotic for pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has entered various agreements, including a licensing partnership with Meiji Seika Pharma to develop tebipenem HBr and collaborations with Everest Medicines and the Bill & Melinda Gates Medical Research Institute for the development and commercialization of SPR206 and SPR720, respectively. Founded in 2013, Spero Therapeutics aims to address urgent medical needs through innovative therapeutic solutions for serious bacterial infections.
Effector Therapeutics, Inc. is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of small molecule drugs designed to treat cancer and other serious diseases by selectively regulating protein synthesis, specifically targeting mRNA translation. Effector Therapeutics focuses on developing translation regulators that inhibit disregulated translation, which is crucial for tumor growth and survival. This innovative approach aims to provide potent and selective therapeutic options, offering a promising avenue for delivering new cancer treatments to patients in need.
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.
True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
Puridify Limited is a bioprocessing company that specializes in developing purification technologies for the manufacturing of industrial biomolecules. Founded in March 2013 and based in Stevenage, United Kingdom, the company has created FibroSelect, a platform purification technology designed to overcome the limitations of traditional packed bed chromatography and the capacity issues of chromatography membranes. This innovative technology provides uniform purification performance across various scales, making it suitable for the purification of macromolecular biotherapeutics, including gene therapies, viral vectors, and fusion proteins. Puridify Limited conducts ongoing research and development to advance its bioprocessing solutions, enhancing the efficiency and effectiveness of biomolecule manufacturing.
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. It specializes in the discovery and development of therapeutics for neurodegenerative disorders, particularly focusing on conditions with significant unmet medical needs, such as progressive supranuclear palsy, orphan tauopathies, Alzheimer's disease, and Parkinson's disease. Asceneuron aims to address the challenges posed by tauopathies, which are currently untreatable and characterized by the accumulation of tau protein deposits in the brain, leading to severe neurodegeneration. The company seeks to develop small molecule drugs that can provide effective treatment options, as existing therapies for Alzheimer's disease and related conditions offer limited benefits. With an increasing aging population, the burden of neurodegenerative diseases is expected to grow, highlighting the urgent demand for innovative and better-tolerated therapeutic solutions. Asceneuron operates as a subsidiary of Merck KGaA, reinforcing its commitment to advancing treatments in this critical area of healthcare.
Principia Biopharma is a private, pre-clinical stage company initially focused on the discovery and development of differentiated small molecule drugs targeting inflammatory/autoimmune diseases as well as cancer. The company expects to submit an IND for its lead program in 2013 and continues to invest in additional programs and its reversible covalent platform.
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, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of novel treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. The company's key product candidates include tebipenem pivoxil hydrobromide, an oral antibiotic aimed at treating MDR gram-negative infections in adults; SPR206, an intravenous agent targeting MDR gram-negative infections in hospital settings; and SPR720, an oral antibiotic for pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has entered various agreements, including a licensing partnership with Meiji Seika Pharma to develop tebipenem HBr and collaborations with Everest Medicines and the Bill & Melinda Gates Medical Research Institute for the development and commercialization of SPR206 and SPR720, respectively. Founded in 2013, Spero Therapeutics aims to address urgent medical needs through innovative therapeutic solutions for serious bacterial infections.
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 is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
ZappRx, Inc. is a digital health company based in Boston, Massachusetts, focused on modernizing the management of specialty medications. Its cloud-based prescription and prior authorization platform enhances efficiency for healthcare providers, specialty pharmacies, and biopharma companies by streamlining the ordering process for specialty drugs. The platform facilitates secure, collaborative interactions among providers, pharmacists, and payers, significantly reducing reliance on traditional communication methods like faxes and phone calls. ZappRx's services are particularly utilized in pulmonary, cardiology, and gastroenterology fields. The company was incorporated in 2012 and, as of June 2019, operates as a subsidiary of Allscripts Healthcare Solutions, Inc.
Thrasos is a bio-therapeutics company focused on the discovery and development of targeted therapies for the prevention and treatment of severe organ failure, with a principle focus on kidney disease. The Company's lead therapeutic program, THR-184, is focused on the prevention and treatment of acute kidney injury (AKI). The Company also has a preclinical development program for treating chronic kidney disease (CKD).
Nimbus Apollo is a program under Nimbus Therapeutics that focuses on the development of innovative treatments for serious diseases, particularly targeting non-alcoholic steatohepatitis (NASH) and hepatocellular carcinoma (HCC). The program's lead candidate, NDI-010976, is an allosteric inhibitor of Acetyl-CoA Carboxylase (ACC), which has received Fast Track designation from the U.S. Food and Drug Administration. Nimbus Apollo is engaged in advancing this candidate through various stages of clinical development, with Phase 1 data expected to be presented at notable medical conferences. Nimbus Therapeutics, founded in 2009 and based in Cambridge, Massachusetts, leverages computational technology to design selective and potent small molecule therapeutics aimed at addressing metabolic diseases, cancer, and immune-inflammatory disorders. The company has established strategic partnerships to enhance its research and development efforts.
Zikani Therapeutics are among the most widely used, most effective, and safest antibiotics to reach clinical practice; in fact, azithromycin has been among the world’s most widely used antibiotics for the past several years. But, while current macrolides are highly effective against some major bacteria, they are ineffective against others, especially gram-negative bacteria, the cause of most serious infections in the US. The first three generations of macrolides have very limited activity against gram-negative, and the sole drug in the current fourth-generation, solithromycin, has only slightly greater activity. Within other antibiotic classes, a limited number of compounds are in development to treat resistant gram-negative infections, and even fewer are available both intravenously and orally, as would be expected with macrolides. The company was founded in 2014 and is headquartered in Newton, Massachusetts.
Nkarta, Inc., a biopharmaceutical company, develops and commercializes cell therapies for cancer treatment. The company’s approach for cellular immunotherapy involves chimeric antigen receptors on the surface of a natural killer (NK) cell that enable the cell to recognize specific proteins or antigens that are present on the surface of tumor cells. Its co-lead product candidates are NKX101, a pre-clinical product that enhances the power of innate NK biology to detect and kill cancerous cells; and NKX019, a pre-clinical product, which is based on the ability to treat various B cell malignancies by targeting the CD19 antigen found on these types of cancerous cells. The company was founded in 2015 and is headquartered in South San Francisco, California.
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 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.
True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
Puridify Limited is a bioprocessing company that specializes in developing purification technologies for the manufacturing of industrial biomolecules. Founded in March 2013 and based in Stevenage, United Kingdom, the company has created FibroSelect, a platform purification technology designed to overcome the limitations of traditional packed bed chromatography and the capacity issues of chromatography membranes. This innovative technology provides uniform purification performance across various scales, making it suitable for the purification of macromolecular biotherapeutics, including gene therapies, viral vectors, and fusion proteins. Puridify Limited conducts ongoing research and development to advance its bioprocessing solutions, enhancing the efficiency and effectiveness of biomolecule manufacturing.
Principia Biopharma is a private, pre-clinical stage company initially focused on the discovery and development of differentiated small molecule drugs targeting inflammatory/autoimmune diseases as well as cancer. The company expects to submit an IND for its lead program in 2013 and continues to invest in additional programs and its reversible covalent platform.
Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of novel treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. The company's key product candidates include tebipenem pivoxil hydrobromide, an oral antibiotic aimed at treating MDR gram-negative infections in adults; SPR206, an intravenous agent targeting MDR gram-negative infections in hospital settings; and SPR720, an oral antibiotic for pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has entered various agreements, including a licensing partnership with Meiji Seika Pharma to develop tebipenem HBr and collaborations with Everest Medicines and the Bill & Melinda Gates Medical Research Institute for the development and commercialization of SPR206 and SPR720, respectively. Founded in 2013, Spero Therapeutics aims to address urgent medical needs through innovative therapeutic solutions for serious bacterial infections.
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, focused on modernizing the management of specialty medications. Its cloud-based prescription and prior authorization platform enhances efficiency for healthcare providers, specialty pharmacies, and biopharma companies by streamlining the ordering process for specialty drugs. The platform facilitates secure, collaborative interactions among providers, pharmacists, and payers, significantly reducing reliance on traditional communication methods like faxes and phone calls. ZappRx's services are particularly utilized in pulmonary, cardiology, and gastroenterology fields. The company was incorporated in 2012 and, as of June 2019, operates as a subsidiary of Allscripts Healthcare Solutions, Inc.
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.
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.
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.
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.
We are using induced pluripotent stem cells for drug discovery and development. Focused on neurodegenerative diseases such as SMA, ALS and Parkinson's, and other major unmet medical needs.
Dicerna Pharmaceuticals is a biotechnology company involved in the discovery and development of innovative treatments for rare inherited diseases affecting the liver, along with cancers, and other therapeutic areas to address liver problems. It is using ribonucleic acid interference (RNA) technology platform to develop its products. The product candidates are DCR-PHXC (for primary hyperoxaluria), DCR-undisclosed (for orphan genetic disease), DCR-PCSK9 (for cardiovascular disease), and DCR-HBV (for hepatitis B virus) among others.
The company was founded in 2007 and is headquartered in Lexington, Massachusetts.
Effector Therapeutics, Inc. is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of small molecule drugs designed to treat cancer and other serious diseases by selectively regulating protein synthesis, specifically targeting mRNA translation. Effector Therapeutics focuses on developing translation regulators that inhibit disregulated translation, which is crucial for tumor growth and survival. This innovative approach aims to provide potent and selective therapeutic options, offering a promising avenue for delivering new cancer treatments to patients in need.
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 is a private, pre-clinical stage company initially focused on the discovery and development of differentiated small molecule drugs targeting inflammatory/autoimmune diseases as well as cancer. The company expects to submit an IND for its lead program in 2013 and continues to invest in additional programs and its reversible covalent platform.
Thrasos is a bio-therapeutics company focused on the discovery and development of targeted therapies for the prevention and treatment of severe organ failure, with a principle focus on kidney disease. The Company's lead therapeutic program, THR-184, is focused on the prevention and treatment of acute kidney injury (AKI). The Company also has a preclinical development program for treating chronic kidney disease (CKD).