Scholar Rock is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of innovative therapies targeting growth factor signaling in serious diseases. The company’s lead product candidate, SRK-015, is a selective inhibitor of myostatin activation and is currently undergoing Phase II clinical trials for spinal muscular atrophy. Additionally, Scholar Rock is advancing SRK-181, which is in Phase I trials for treating cancers resistant to checkpoint inhibitors. The company employs a proprietary platform to design monoclonal antibodies that selectively modulate the activation of growth factors in disease microenvironments, addressing challenges associated with traditional growth factor inhibition. Scholar Rock is also developing a pipeline of candidates for a variety of conditions, including neuromuscular disorders, cancer, fibrosis, and anemia. Furthermore, the company has a collaboration with Gilead Sciences to explore specific inhibitors of transforming growth factor beta for fibrotic diseases. Founded in 2012, Scholar Rock aims to improve patient outcomes through its targeted therapeutic approach.
Upstream Bio is a clinical-stage biotechnology company established in 2004 and headquartered in the United States. The company specializes in developing antibody therapies aimed at treating inflammatory diseases, with a primary focus on severe respiratory disorders, particularly severe asthma. Upstream Bio is advancing its lead candidate, verekitug, an antagonist that targets the receptor for Thymic Stromal Lymphopoietin (TSLP), a cytokine known to play a significant role in driving inflammatory responses. The approach of targeting TSLP positions Upstream Bio to address various immune-mediated conditions by intervening at a crucial point in the signaling pathways involved in inflammation.
Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.
Seismic Therapeutic is a biotechnology company focused on accelerating immunology drug development through the integration of machine learning in the biologics discovery process. Utilizing its IMPACT™ platform, Seismic Therapeutic aims to tackle key challenges in biologics discovery by combining structural biology, protein engineering, and translational immunology. This comprehensive approach facilitates the creation of optimized therapies that can reach patients more quickly. The company is developing a pipeline of innovative biologics targeting adaptive immune system dysregulation, with the goal of treating autoimmune diseases.
Dewpoint Therapeutics, Inc. is a Boston-based biotechnology company focused on the research and development of biomolecular condensates to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunology, cardiovascular issues, women's health, and virology. Founded in 2018, the company utilizes a proprietary, AI-powered drug discovery platform that employs machine learning and advanced image analysis to visualize and manipulate biomolecular condensates. This technology aids in identifying novel drug candidates and has applications across various therapeutic areas. Dewpoint Therapeutics collaborates with leading academic and pharmaceutical partners to enhance its research and development efforts, aiming to deliver impactful healthcare solutions.
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.
Exo Therapeutics, Inc. is a molecule drug discovery and development company to address intractable pharmaceutical targets. The company develops a pipeline of drug candidates that bind exosites, distal, and unique binding pockets that reprogram enzyme activity for therapeutic effect in oncology, inflammation. Exo Therapeutics, Inc. was founded in 2018 and is based in Cambridge, Massachusetts.
Obsidian Therapeutics develops innovative cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Founded in 2015 and based in Cambridge, Massachusetts, the company focuses on creating next-generation therapies that utilize pharmacologic operating systems to provide precise control over protein activity within cells. This technology allows for the development of adoptive cell therapies with advanced functionalities that can be managed by physicians using simple, safe, and orally active medications. By offering improved control over treatment, Obsidian Therapeutics aims to provide better outcomes for patients compared to existing cell therapy options.
HilleVax is a clinical-stage biopharmaceutical company established in 2021 and based in Boston, Massachusetts. The company specializes in the development and commercialization of innovative vaccines. Its primary focus is on HIL-214, a vaccine candidate that utilizes virus-like particle (VLP) technology aimed at preventing moderate-to-severe acute gastroenteritis caused by norovirus infection.
Tango Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cancer therapies utilizing the principle of synthetic lethality. Established in 2014, the company aims to discover novel drug targets that address driver genes in cancer. Its research is concentrated on three primary areas: counteracting the loss of tumor suppressor genes, reversing cancer cells' ability to evade the immune system, and identifying effective drug combinations that surpass the efficacy of single-agent therapies. By leveraging these strategies, Tango Therapeutics seeks to deliver next-generation targeted treatments for patients battling cancer.
Abata Therapeutics, a company focused on translating the biology of regulatory T cells (Tregs) into transformational medicines for patients living with progressive multiple sclerosis (MS) and other severe autoimmune and inflammatory diseases.
Vedere Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on developing gene therapy products aimed at restoring functional vision for patients who have experienced vision loss due to Inherited Retinal Degenerations (IRDs) and other causes of vision impairment, both genetic and non-genetic. Unlike many ocular gene therapies that primarily slow the progression of vision loss, Vedere Bio seeks to restore lost vision, regardless of the patient's genetic background or stage of their condition. As of September 2020, Vedere Bio operates as a subsidiary of Novartis AG.
ElevateBio, LLC, based in Cambridge, Massachusetts, is a holding company focused on the development of cell and gene-based therapies. Established in 2017, ElevateBio operates a portfolio of subsidiary companies that aim to create innovative treatments for cancer and viral infections. The company partners with leading scientists and inventors to foster its portfolio and has established a centralized facility designed to streamline the translation of research and development into commercially viable therapies. This integrated approach enables the efficient development, manufacturing, and commercialization of life-transforming medicines, ultimately helping to provide patients with effective treatments for severe diseases.
EGenesis Inc. is a biotechnology company based in Cambridge, Massachusetts, with an additional office in New York. Founded in 2015, the company specializes in gene editing and genome engineering, focusing on the development of human-compatible organs, tissues, and cells for transplantation. EGenesis employs advanced gene editing techniques to create transplantable cells and solid organs, specifically targeting therapeutic applications in kidney and islet cell transplantation. By leveraging its innovative platform, the company aims to transform the field of organ transplantation, providing safe and effective solutions for patients with life-threatening diseases globally.
Centessa Pharmaceuticals is a clinical-stage biopharmaceutical company focused on transforming the drug development process through an asset-centric research and development model. The company advances a diverse portfolio of validated programs, each managed by a dedicated subsidiary and backed by a centralized infrastructure and experienced management team. Among its pipeline assets are SerpinPC, targeting Hemophilia A and B, LB101 for solid tumors, ORX750 for narcolepsy type 1 and other sleep disorders, and MGX292 for pulmonary arterial hypertension. Additionally, Centessa is developing an undisclosed asset for solid tumors.
Abcuro, Inc. is focused on developing immunotherapies aimed at treating autoimmunity and cancer. Incorporated in 2015 and based in Newton, Massachusetts, the company specializes in creating therapeutic antibodies that target novel aspects of the immune system. By employing advanced bioinformatics and analyzing transcription data from human diseases, Abcuro identifies new therapeutic targets and validates them through ex-vivo studies in diseased tissues. This innovative approach aims to enhance treatment strategies and improve patient outcomes in the fields of autoimmunity and oncology.
Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.
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.
Dewpoint Therapeutics, Inc. is a Boston-based biotechnology company focused on the research and development of biomolecular condensates to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunology, cardiovascular issues, women's health, and virology. Founded in 2018, the company utilizes a proprietary, AI-powered drug discovery platform that employs machine learning and advanced image analysis to visualize and manipulate biomolecular condensates. This technology aids in identifying novel drug candidates and has applications across various therapeutic areas. Dewpoint Therapeutics collaborates with leading academic and pharmaceutical partners to enhance its research and development efforts, aiming to deliver impactful healthcare solutions.
Unum Therapeutics is a biopharmaceutical company focused on developing immunotherapy products for cancer treatment, utilizing proprietary T-cell engineering technology. The company’s lead programs, based on its Antibody-Coupled T-cell Receptor (ACTR) technology, are in Phase I clinical trials. These include ACTR707 and ACTR087, both being tested in combination with rituximab for relapsed or refractory CD20+ non-Hodgkin lymphoma, as well as ACTR087 combined with SEA-BCMA for relapsed multiple myeloma. Additionally, ACTR707 is being evaluated with trastuzumab for HER2+ solid tumors. Unum Therapeutics is also exploring BOXR1030, a pre-clinical candidate targeting glypican-3 to enhance T cell functionality in the solid tumor microenvironment. Founded in 2014 and headquartered in Cambridge, Massachusetts, Unum aims to activate the body’s immune system to effectively combat cancer through its innovative therapies.
ElevateBio, LLC, based in Cambridge, Massachusetts, is a holding company focused on the development of cell and gene-based therapies. Established in 2017, ElevateBio operates a portfolio of subsidiary companies that aim to create innovative treatments for cancer and viral infections. The company partners with leading scientists and inventors to foster its portfolio and has established a centralized facility designed to streamline the translation of research and development into commercially viable therapies. This integrated approach enables the efficient development, manufacturing, and commercialization of life-transforming medicines, ultimately helping to provide patients with effective treatments for severe diseases.
Fluent BioSciences is a privately funded life science company focused on developing high-sensitivity molecular analytical products. Utilizing proprietary instrument-free micro-self-assembly technology, the company enables the analysis of extremely low molecular and cellular sample inputs. Its platform facilitates the instantaneous self-assembly of individual cells or molecules into millions of uniform partitions, allowing for sensitive and unbiased preparation of proteins and nucleic acids for sequencing applications. This innovative approach provides researchers in the biotechnology sector with enhanced capabilities for single-cell analysis, broadening their access to advanced analytical techniques.
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and cancers. The company's key product candidates include FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, aimed at treating metastatic castration-resistant prostate cancer. Additionally, Forma is advancing FT-2102, an oral investigational agent targeting mutated IDH1 enzymes, which is being assessed in a Phase 2 trial for relapsed/refractory acute myeloid leukemia and an exploratory Phase 1 trial for glioma. The pipeline also features FT-4101 and FT-8225, which are selective inhibitors of fatty acid synthase. Founded in 2007 and headquartered in Watertown, Massachusetts, Forma integrates advanced drug discovery technologies with oncology expertise to target significant challenges in cancer treatment and address unmet medical needs.
Pieris Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on discovering and developing anticalin-based therapeutics. Anticalins are low molecular-weight proteins derived from lipocalins, which are naturally occurring proteins in the human body. The company’s lead respiratory candidate, PRS-060, is in Phase I trials for asthma and other inflammatory diseases, while its lead immuno-oncology candidate, PRS-343, is also in Phase I trials targeting various cancers. Additionally, Pieris is advancing PRS-080, a treatment for functional iron deficiency in patients with chronic kidney disease, currently in Phase IIa trials, and PRS-344, a bispecific anticalin-antibody fusion protein in preclinical development for oncology. The company collaborates with several partners, including Les Laboratoires Servier, AstraZeneca, and Seattle Genetics, and has research collaborations with academic institutions such as the University of Pittsburgh. Founded in 2000, Pieris Pharmaceuticals aims to address critical medical needs through its innovative drug development approach.
AlloVir, Inc. is a clinical-stage cell therapy company based in Cambridge, Massachusetts, specializing in the development of allogeneic, off-the-shelf multi-virus specific T cell (VST) therapies for viral-associated diseases. Its lead product, Viralym-M, targets several viruses including BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. The company's pipeline also includes candidates for treating respiratory syncytial virus, influenza, SARS-CoV-2, hepatitis B, and human herpesvirus-8. AlloVir's proprietary VST therapy platform aims to provide accessible treatment options for patients facing severe viral infections, addressing the pressing need for effective therapies in this area. Founded in 2013, AlloVir was previously known as ViraCyte, Inc. and rebranded in May 2019.
ElevateBio, LLC, based in Cambridge, Massachusetts, is a holding company focused on the development of cell and gene-based therapies. Established in 2017, ElevateBio operates a portfolio of subsidiary companies that aim to create innovative treatments for cancer and viral infections. The company partners with leading scientists and inventors to foster its portfolio and has established a centralized facility designed to streamline the translation of research and development into commercially viable therapies. This integrated approach enables the efficient development, manufacturing, and commercialization of life-transforming medicines, ultimately helping to provide patients with effective treatments for severe diseases.
SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for underserved patient populations affected by rare diseases and cancer. Founded in 2017 and headquartered in Stamford, Connecticut, the company’s lead product candidate, nirogacestat, is an oral small molecule gamma secretase inhibitor currently in Phase III trials for treating desmoid tumors. Other significant candidates include mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for relapsed or refractory multiple myeloma. The company is also exploring additional therapies, such as BGB-3245, an investigational oral inhibitor targeting BRAF mutations, currently in Phase I trials. SpringWorks has established collaborations with several biotechnology and pharmaceutical firms to enhance its research and development efforts.
Dewpoint Therapeutics, Inc. is a Boston-based biotechnology company focused on the research and development of biomolecular condensates to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunology, cardiovascular issues, women's health, and virology. Founded in 2018, the company utilizes a proprietary, AI-powered drug discovery platform that employs machine learning and advanced image analysis to visualize and manipulate biomolecular condensates. This technology aids in identifying novel drug candidates and has applications across various therapeutic areas. Dewpoint Therapeutics collaborates with leading academic and pharmaceutical partners to enhance its research and development efforts, aiming to deliver impactful healthcare solutions.