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.
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.
Founded in April 2006, Concert Pharmaceuticals is a clinical stage biotechnology company dedicated to creating new medicines through a novel scientific approach utilizing the naturally-occurring element deuterium.
Concert applies its innovative precision deuterium chemistry platform to modify specific properties of validated drug molecules, yielding a rich pipeline of new chemical entities (NCEs). Concert leverages decades of pharmaceutical experience to create novel drug candidates with potential for best-in-class efficacy and safety, while greatly reducing R&D risk, time and expense.
Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells within the immune system. The company aims to address significant limitations associated with existing cell and gene therapies, such as the challenges of administering multiple doses, unpredictable patient reactions, and the necessity of potentially harmful chemotherapy regimens prior to treatment. Be Biopharma's approach allows for the creation of durable therapies that can be re-administered without toxic conditioning, making these treatments suitable for a range of diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020, Be Biopharma is headquartered in Cambridge, Massachusetts.
Ventus Therapeutics U.S., Inc. is a biopharmaceutical company based in Natick, Massachusetts, with an additional location in Montreal, Canada. Founded in 2019, it focuses on discovering and developing novel small-molecule medicines that target the innate immune system to address autoimmune diseases, inflammatory diseases, and cancer. The company employs a structural immunology platform that integrates protein engineering to elucidate molecular structures and mechanisms of action. This platform facilitates the development of precise binding and functional screening assays, as well as structure-based modeling. Ventus Therapeutics is advancing an emerging pipeline of drug programs aimed at key targets within the innate immune system, leveraging its proprietary capabilities in rational and structure-based drug design.
Third Harmonic Bio is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for severe allergies and inflammation. The company is focused on creating first-in-class oral small-molecule inhibitors targeting the KIT receptor, which plays a crucial role in the survival and function of mast cells—key immune cells involved in allergic responses. Its lead product candidate, THB001, is designed to provide selective inhibition of KIT, while another candidate, THB335, aims to address multiple mast cell-driven inflammatory diseases affecting the skin, respiratory system, and gastrointestinal tract. Through its research and development efforts, Third Harmonic Bio seeks to advance the management of inflammatory diseases, enhancing patient outcomes and quality of life.
NanoMosaic specializes in innovative solutions for early illness detection, prognostic monitoring, and biomarker identification. The company has developed a unique platform that utilizes nanoneedles for protein detection and quantification. This technology facilitates the analysis of multiple biomarkers on a single chip, making it possible for the medical and healthcare sectors to enhance the speed and accuracy of biomarker discovery and validation. By enabling more efficient proteomic analysis, NanoMosaic aims to improve early disease detection and monitoring, ultimately contributing to better health outcomes.
Curie Therapeutics is a fully integrated, next-generation radiopharmaceuticals company focused on pioneering a new class of targeted radiopharmaceuticals that are both safe and effective. Curie's radiopharmaceuticals are composed of rationally designed conjugates against carefully selected biological targets, to deliver alpha and beta-emitting radionuclide payloads.
Avilar Therapeutics is a biopharmaceutical company focusing on the discovery and development of extracellular protein degraders, a new frontier in targeted protein degradation.
Cedilla is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation.
The company was founded in 2018 and based in Cambridge, Massachusetts.
Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.
Jnana Therapeutics Inc. is a Boston-based company founded in 2016 that operates a drug discovery platform centered on solute carrier (SLC) transporters. The company focuses on key disease pathways related to immunometabolism, lysosomal function, and mucosal defense, targeting areas such as immuno-oncology, inflammatory disorders, and neurological diseases. By leveraging its innovative approach, Jnana Therapeutics aims to discover and develop transformative medicines that address significant medical needs and improve patient outcomes.
GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) to treat a range of conditions, including autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and based in Boston, Massachusetts, with additional locations in Seattle and Israel, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs. This innovative approach aims to restore immune tolerance and address the limitations found in current regulatory T-cell therapies. The company was co-founded by experts in Treg biology and synthetic immunology, emphasizing its commitment to addressing the underlying causes of diseases linked to immune system dysfunction.
Tango Therapeutics, Inc., a biotechnology company, develops cancer drugs based on synthetic lethality. The company uses the genetic principle of synthetic lethality to discover and develop therapies that take aim at driver genes in cancer. It focuses counteracting tumor suppressor gene loss; reversing the ability of cancer cells to evade the immune system; and identifying novel combinations that are more effective than single agent therapy. Tango Therapeutics, Inc. was formerly known as Cancer Combos NewCo, Inc. The company was incorporated in 2014 and is based in Cambridge, Massachusetts.
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.
Eliem Therapeutics, Inc. develops therapeutic drugs for the treatment of hyperexcitability disorders. The company focuses on the clinical development of analgesics for the treatment of hyperexcitability disorders, such as chronic pain using neuroscience and translational medicine. The company was incorporated in 2018 and is based in Boston, Massachusetts.
Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.
Interius BioTherapeutics
Series A in 2021
Interius BioTherapeutics is a biopharmaceutical company that targets cancer. The company spun out the University of Pennsylvania and is targeting cancer.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
Invivyd is a biotechnology company focused on developing advanced antibodies to neutralize SARS-CoV-2, SARS-CoV-1, and other potentially emerging coronaviruses. Founded in 2020 and headquartered in Waltham, Massachusetts, the company aims to create best-in-class therapeutic and prophylactic treatments that surpass the efficacy of existing antibody programs. Its innovative portfolio features multiple non-competing antibodies, each targeting distinct viral epitopes, which is designed to minimize the risk of viral escape. By leveraging cutting-edge antibody discovery technologies, Invivyd seeks to enhance the potency and durability of its antibody candidates, addressing a critical need in the ongoing fight against coronaviruses.
Ventus Therapeutics U.S., Inc. is a biopharmaceutical company based in Natick, Massachusetts, with an additional location in Montreal, Canada. Founded in 2019, it focuses on discovering and developing novel small-molecule medicines that target the innate immune system to address autoimmune diseases, inflammatory diseases, and cancer. The company employs a structural immunology platform that integrates protein engineering to elucidate molecular structures and mechanisms of action. This platform facilitates the development of precise binding and functional screening assays, as well as structure-based modeling. Ventus Therapeutics is advancing an emerging pipeline of drug programs aimed at key targets within the innate immune system, leveraging its proprietary capabilities in rational and structure-based drug design.
New Equilibrium Biosciences provides a platform that reveals opportunities for structure-based design by learning biophysics with AI.
Pyxis Oncology, Inc. is a biotechnology company focused on developing antibody therapeutics that enhance the immune response against cancer. Founded in 2018 and based in Boston, Massachusetts, the company specializes in analyzing tumor antigen-specific tumor-infiltrating lymphocytes within hot tumors and tumor cell signaling pathways in cold tumors. This research informs the creation of novel antibody-based immunotherapies aimed at targeting difficult-to-treat cancers. Pyxis Oncology's goal is to develop next-generation therapeutics that not only kill tumor cells but also address the underlying issues that facilitate cancer's proliferation and immune evasion, ultimately improving the quality of life for patients.
Eliem Therapeutics, Inc. develops therapeutic drugs for the treatment of hyperexcitability disorders. The company focuses on the clinical development of analgesics for the treatment of hyperexcitability disorders, such as chronic pain using neuroscience and translational medicine. The company was incorporated in 2018 and is based in Boston, Massachusetts.
Century Therapeutics, Inc. is a biotechnology company focused on developing induced pluripotent stem cell (iPSC)-based therapies for cancer treatment. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company aims to create allogeneic living drugs that specifically target both hematologic and solid tumors. By utilizing iPSCs, Century Therapeutics can engineer immune effector cells through multiple rounds of cellular modification, producing master cell banks that can be expanded and differentiated for therapeutic use. Their innovative approach incorporates advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, along with Allo-Evasion technology, which helps prevent rejection by the host immune system. These advancements position Century Therapeutics to enhance the effectiveness and accessibility of cancer treatments.
Xilio Therapeutics is a biotechnology company advancing next-generation cancer immunotherapies designed to improve patient outcomes by unleashing the power of the immune system selectively at the site of the tumor. The company’s tumor-selective immunotherapies are based on its proprietary technology, which maximizes the potency of proven immuno-oncology therapies and restricts their activity to the tumor to minimize peripheral side effects. The broad applicability of these therapies across cancer types means that all patients could benefit from these potentially curative medicines. These tumor-selective therapies are designed to overcome the significant toxicities associated with validated IO therapies, such as IL-2 and aCTLA4, which have historically limited the number of patients that can be treated and prevented patients from completing full courses of treatment Xilio Therapeutics proprietary technology enables the explosion of therapeutic activity specifically within the tumor while reducing toxicity throughout the body a nod to the company name, Xilio, derived from the Latin term, Ex Nihilo, meaning creation or big-bang. The company applying its technology to build a broad pipeline of engineered antibodies, cytokines and chemokines as potential new options for people living with cancer. Xilio Therapeutics was founded in 2016 and is headquartered in Waltham, Massachusetts, United States.
Cyteir is developing the next generation of synthetic lethal therapies to treat cancer.Cyteir Therapeutics is creating breakthrough solutions to the major challenges facing cancer researchers today. Currently in development, is the answer to the oncology triumvirate: reducing side effects, reducing therapy resistance, inducing diseased cell self destruction.
TScan Therapeutics is a biopharmaceutical company dedicated to developing innovative T cell therapies to treat various cancers, including liquid tumors and solid tumors. The company focuses on engineering T cell receptor (TCR) therapies, with specific candidates aimed at addressing hematologic malignancies and preventing relapse after hematopoietic stem cell transplantation. TScan utilizes a specialized platform that identifies shared T cell antigens and minimizes off-target effects, enhancing the safety and effectiveness of its treatments. The company’s pipeline includes both liquid tumor therapies and multiplexed TCR-T candidates for solid tumors. Founded in 2018, TScan Therapeutics is headquartered in Waltham, Massachusetts, and comprises a team of experts committed to expanding treatment options for patients with limited therapeutic alternatives.
Verve Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to reduce the risk of coronary artery disease, a leading cause of death globally. Founded in 2018 and headquartered in Cambridge, Massachusetts, with a research facility in Philadelphia, the company leverages human genetic analysis and gene-editing technologies to create treatments that offer lifelong protection from this condition. Verve has established strategic alliances, including collaborations with Beam Therapeutics to advance delivery technologies targeting cardiovascular issues and with Verily for developing gene editing delivery vehicles. Formerly known as Endcadia, Verve Therapeutics rebranded in September 2019 to reflect its commitment to addressing heart disease through advanced genetic solutions.
Abcuro’s mission is to develop a new class of immune-modulatory bio-therapeutics for treating both autoimmunity and cancer. They leverage the systematic target validation and clinical insights gained by its founders to identify new targets. Their intensive use of bioinformatics to interrogate the transcriptome of human disease together with ex-vivo validation in diseased tissue has uncovered new approaches to target key compartments of the immune system.
The company was incorporated in 2015 and is based in Newton, Massachusetts.
Werewolf Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative receptor agonists for cancer immunotherapy. Established in 2017, the company is advancing a pipeline of transformative biotherapeutics aimed at treating cancer. Werewolf Therapeutics creates medicines intended for systemic delivery that remain inactive within the body until they arrive at the tumor microenvironment. At this point, the treatments activate to stimulate a robust immune response, thereby targeting and attacking cancer cells effectively.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
AavantiBio, Inc. engages in developing gene transfer therapy and gene editing technologies to improve the lives of people living with fatal diseases. It focuses on Friedreich’s Ataxia (FA), a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. The company was incorporated in 2019 and is based in Gainesville, Florida.
Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells within the immune system. The company aims to address significant limitations associated with existing cell and gene therapies, such as the challenges of administering multiple doses, unpredictable patient reactions, and the necessity of potentially harmful chemotherapy regimens prior to treatment. Be Biopharma's approach allows for the creation of durable therapies that can be re-administered without toxic conditioning, making these treatments suitable for a range of diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020, Be Biopharma is headquartered in Cambridge, Massachusetts.
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.
GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) to treat a range of conditions, including autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and based in Boston, Massachusetts, with additional locations in Seattle and Israel, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs. This innovative approach aims to restore immune tolerance and address the limitations found in current regulatory T-cell therapies. The company was co-founded by experts in Treg biology and synthetic immunology, emphasizing its commitment to addressing the underlying causes of diseases linked to immune system dysfunction.
Cerevel (www.cerevel.com) is a biopharmaceutical company focused on developing drug candidates to treat disorders of the central nervous system (CNS). The company has a portfolio of pre-commercial neuroscience assets, which include three clinical-stage compounds and several pre-clinical compounds designed to target a broad range of CNS disorders including Parkinson’s, Alzheimer’s, epilepsy, schizophrenia and addiction. Headquartered in the Greater Boston area, Cerevel was formed in 2018 through a partnership between Bain Capital and Pfizer.
Vor Biopharma, Inc. develops engineered hematopoietic stem cell (eHSC) therapies for cancer patients. It offers VOR33, an eHSC product candidate that is in preclinical development to treat acute myeloid leukemia (AML). The company’s VOR33 eHSCs lacks CD33, a protein that is expressed by AML cancer cells. Vor Biopharma, Inc. was incorporated in 2015 and is based in Cambridge, Massachusetts.
C4 is building on the knowledge with synthetic chemical compounds which it calls Degronimids. These compounds can be engineered and linked together to bind with many molecular targets that today’s drugs can’t bind with, Cohen said. The key insight, he said, is that the scientific founders have discovered a way to bind with select disease-causing proteins, and flag them as cellular trash, so that the ordinary proteasome garbage disposal system can get rid of them. Selecting the precise disease-related proteins to send to the trash is obviously critical, because any drug like this could cause serious side effects if it sent healthy proteins involved in normal cell processes to the trash. C4 Therapeutics is developing a new class of targeted protein degradation (TPD) therapeutics for the treatment of a broad range of diseases. Our Degronimid™ platform incorporates highly selective small molecule binders to target disease-causing proteins and facilitate their rapid destruction and clearance from the cell through the natural ubiquitin/proteasome system (UPS). Because of this distinctive mechanism, Degronimids are capable of hitting many more targets, including those previously thought to be undruggable, while reducing the potential for drug resistance. The broad applicability of Degronimids, and our chemical biology platform designed for accelerated validation, have the potential to make an unprecedented impact across many diseases through multiple industry collaborations as well as proprietary programs. The Company has received an exclusive worldwide license to the Degronimid platform and related IP from Dana-Farber.
Everest Medicines Limited, a clinical-stage biopharmaceutical company, focuses on licensing, developing, and commercializing therapies for addressing critical unmet medical needs in Greater China and other Asia Pacific markets. It has a portfolio of eight clinical-stage drug candidates for the treatment of oncology, immunology, cardio-renal disease, and infectious diseases. The company was founded in 2017 and is headquartered in Shanghai, China.
Atea Pharmaceuticals is a biopharmaceutical company engaged in the discovery and development of next-generation therapeutics for severe human viral infections. It caters to the healthcare industry and engages in the discovery and development of antiviral therapeutics that enable patients to have treatment for infections caused by RNA and DNA viruses.
Atea Pharmaceuticals was established in 2014 and is headquartered in Boston, Massachusetts.
Legend Biotech is a biopharmaceutical company bringing impactful innovations in cell therapy, and accelerating the science. They are committed to improving the lives of patients worldwide. They are also steadfast in their goal to develop innovative cellular therapies that bring us closer to a cure. It develops cell therapies from discovery through commercialization.
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.
Iteos Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of immuno-oncology therapeutics for cancer patients. Its product pipeline includes EOS-850, a small molecule antagonist of the adenosine A2a receptor that is an open-label Phase 1/2a clinical trial in adult patients; and EOS-448, an antagonist of TIGIT or T-cell immunoreceptor with Ig and ITIM domains, which is in Phase 1/2a clinical trial. The company was founded in 2011 and is headquartered in Cambridge, Massachusetts.
Akouos is a biotechnology company focusing on restoring and preserving hearing. The company leverages adeno-associated viral (AAV) vector-based gene therapy in order to restore and preserve hearing in genetically-defined patient populations in a range of forms, from a single genetic mutation to ototoxic drug exposure and to the aging process, enabling the healthcare industry to treat sensorineural hearing loss in an efficient manner.
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.
Quench Bio is a developer of gasdermin biology drugs designed to treat severe inflammatory diseases.The company leverages insights into gasdermin biology and innate immunity to develop medicines with lead compounds that target and inhibit the pore-forming protein that mediates the release of inflammatory cytokines, alarmins, DNA and NETs, enabling patients to get relief from inflammatory diseases.
Aerovate Therapeutics is a biotechnology company focused on developing drugs that meaningfully improve the lives of patients with rare cardiopulmonary disease. Aerovate's program is a potentially disease-modifying drug, AV-101, for people who suffer from pulmonary arterial hypertension (PAH). AV-101 is a proprietary inhaled dry powder formulation of the drug imatinib that delivers this medicine directly to diseased lung tissues while reducing systemic adverse side effects.
The company was founded and incubated by RA Capital Management, a multi-stage investment manager focused on healthcare and life sciences.
Forma Therapeutics Holdings, Inc., a clinical-stage biopharmaceutical company, engages in the development and commercialization of novel therapeutics for treatment of rare hematologic diseases and cancers. Its core product candidates for development include FT-4202, which is Phase 1 trial for the treatment of sickle cell disease and other hemoglobinopathies; and FT-7051 for the treatment of metastatic castration-resistant prostate cancer. The company is also developing FT-2102, an oral selective small molecule investigational agent that is designed to bind to and inhibit mutated IDH1 enzymes, which is being evaluated in a Phase 2 trial for relapsed/refractory AML, as well as an exploratory Phase 1 trial for glioma; and FT-4101 and FT-8225, which are selective fatty acid synthase inhibitors. The company was founded in 2007 and is headquartered in Watertown, Massachusetts.
Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule, tumor-agnostic therapies. The company's lead product candidate, BDTX-189, is designed to inhibit specific mutations in ErbB kinases, including the epidermal growth factor receptor. Additionally, Black Diamond is advancing a pipeline of allosteric-EGFR mutation inhibitors and other early-stage programs targeting allosteric mutations in kinases associated with cancer and rare genetic diseases. The company employs a technology platform known as Mutation-Allostery-Pharmacology, which aims to address undrugged mutations in patients with genetically defined cancers. Founded in 2014 and based in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its rebranding in 2018.
Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.
Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company’s lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson’s disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.
Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.
Eliem Therapeutics, Inc. develops therapeutic drugs for the treatment of hyperexcitability disorders. The company focuses on the clinical development of analgesics for the treatment of hyperexcitability disorders, such as chronic pain using neuroscience and translational medicine. The company was incorporated in 2018 and is based in Boston, Massachusetts.
Vor Biopharma, Inc. develops engineered hematopoietic stem cell (eHSC) therapies for cancer patients. It offers VOR33, an eHSC product candidate that is in preclinical development to treat acute myeloid leukemia (AML). The company’s VOR33 eHSCs lacks CD33, a protein that is expressed by AML cancer cells. Vor Biopharma, Inc. was incorporated in 2015 and is based in Cambridge, Massachusetts.
Attune Pharmaceuticals, Inc. is a biotechnology research and development company. The company develops novel oral small molecule therapeutics for treatment of rare diseases. Attune Pharmaceuticals, Inc. was incorporated in 2015 and is based in New York, New York.
Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule, tumor-agnostic therapies. The company's lead product candidate, BDTX-189, is designed to inhibit specific mutations in ErbB kinases, including the epidermal growth factor receptor. Additionally, Black Diamond is advancing a pipeline of allosteric-EGFR mutation inhibitors and other early-stage programs targeting allosteric mutations in kinases associated with cancer and rare genetic diseases. The company employs a technology platform known as Mutation-Allostery-Pharmacology, which aims to address undrugged mutations in patients with genetically defined cancers. Founded in 2014 and based in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its rebranding in 2018.
Sojournix, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, that specializes in developing novel therapies aimed at addressing women's health and neuroendocrine disorders. Founded in 2016, the company is currently focused on the development of SJX-653, a selective neurokinin-3 (NK3) antagonist designed as a non-hormonal treatment for moderate to severe vasomotor symptoms. Through its innovative approach, Sojournix seeks to enhance the quality of life for patients affected by these conditions.
SeLux Diagnostics, Inc. is focused on advancing antibiotic susceptibility testing and infectious disease diagnostics through its innovative phenotyping platform. Founded in 2014 and located in Charlestown, Massachusetts, the company employs a unique synthetic amplifier technology that enhances the speed and complexity of antibiotic susceptibility testing. This platform significantly improves the accuracy and timeliness of diagnosing infections, enabling healthcare providers to prescribe targeted and personalized antibiotic therapies. By facilitating informed treatment decisions, SeLux Diagnostics aims to enhance patient care, save lives, and improve overall health outcomes in hospital settings.
Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide medicines to treat the underlying causes of severe genetic diseases. Its lead product candidate, STK-001 used to treat Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics, Inc. has a partnership with Invitae Corporation to offer genetic testing. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was founded in 2014 and is headquartered in Bedford, Massachusetts.
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.
Akouos is a biotechnology company focusing on restoring and preserving hearing. The company leverages adeno-associated viral (AAV) vector-based gene therapy in order to restore and preserve hearing in genetically-defined patient populations in a range of forms, from a single genetic mutation to ototoxic drug exposure and to the aging process, enabling the healthcare industry to treat sensorineural hearing loss in an efficient manner.
Farcast is a clinical diagnostic company dedicated to providing personalized cancer treatment solutions. Its core technology utilizes a human tumor microenvironment to enable oncologists and drug developers to assess individual tumors and determine their treatment response profiles. This innovative approach aims to surpass traditional biomarker-guided strategies, thereby enhancing access to personalized medicine and improving the clinical management of cancer. Headquartered in Boston, Massachusetts, Farcast also operates a laboratory in Bangalore, India, reflecting its commitment to advancing cancer care through cutting-edge technology.
Imbria Pharmaceuticals’ mission is to translate our understanding of cellular metabolism into innovative medicines that improve the lives of patients with limited or inadequate treatment options. The company was founded by doctors and launched by an all doctor executive team motivated by the promise of bringing new therapies to people in need. We are propelled by a deep scientific interest in cellular metabolism and the mitochondria, and are guided by both values and a code of business conduct built upon our commitment to always put the patients, families and communities we serve at the forefront of all that we do.
Arvinas is a pharmaceutical company that focuses on developing novel therapeutics to treat a variety of diseases including cancer, pro-inflammatory, autoimmune and rare diseases. Its proprietary approach is the targeting of disease-causing proteins for degradation thus eliminating them from the body.
The company was founded in 2013 and is headquartered in Connecticut, United States.
Lantos Technologies is the only FDA-cleared 3D ear scanning system that enables custom, in-ear products with superior comfort, sound quality, and performance. Our technology enables the efficient and effective delivery of customized hearing aids and other hearing-related products.
Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company’s lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson’s disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.
Braeburn is dedicated to delivering solutions for people living with the serious, often fatal consequences of opioid use disorder. We are dedicated to advancing next-generation therapies, with individualized dosing regimens and delivery options, to address the escalating disease burden of opioid use disorder faced by patients, healthcare professionals, payers, and society.
Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It develops various programs for myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapies. The company was founded in 2017 and is based in Waltham, Massachusetts.
TARIS BioMedical, Inc., a therapeutically urology company, develops treatments for patients suffering from difficult-to-treat bladder diseases. It develops therapies for debilitating conditions, including bladder cancer and overactive bladder. The company was founded in 2008 and is based in Lexington, Massachusetts. As of December 20, 2019, TARIS BioMedical, Inc. operates as a subsidiary of Janssen Research & Development, LLC.
Kadmon is a biopharmaceutical company focused on discovering, developing, and commercializing small molecules and biologics for the treatment of inflammatory and fibrotic diseases, as well as various cancers. Its key product candidates include KD025, an oral inhibitor targeting rho-associated coiled-coil kinase 2, currently undergoing Phase II clinical trials for chronic graft-versus-host disease and fibrotic conditions. Other candidates include KD045, aimed at fibrotic diseases, and KD033, an anti-PD-L1/IL-15 fusion protein for cancer treatment. Additionally, Kadmon is developing Tesevatinib for autosomal dominant polycystic kidney disease and CLOVIQUE for Wilson's disease. The company also offers ribavirin products for hepatitis treatment and provides patient support services. Founded in 2010 and headquartered in New York, Kadmon has established collaborations and license agreements with other firms to enhance its research and development efforts.
Realm Therapeutics is a biopharmaceutical company passionately committed to leveraging its proprietary technologies to protect and improve the health of adults and children. The Company's drug development focus, utilizing its immunomodulatory formulations, is targeted initially on developing small molecule therapies in inflammatory diseases with potential application in dermatology and ophthalmology, and the potential for broad applicability across a number of other diseases.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
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.
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.
Bristol Myers Squibb is a global biopharmaceutical company that specializes in the discovery, development, manufacturing, and marketing of innovative medicines. Founded in 1887 and headquartered in New York City, the company focuses on therapeutic areas such as hematology, oncology, cardiovascular, and immunology. Its notable products include Opdivo for cancer treatment, Eliquis for stroke prevention and venous thromboembolic disorders, and Orencia for rheumatoid arthritis. Additionally, its portfolio features treatments for chronic hepatitis B, HIV, and multiple myeloma, among others. The company operates through two segments: Pharmaceuticals and Nutritionals, with the latter managed by its subsidiary, Mead Johnson Nutrition. Bristol Myers Squibb collaborates with various partners, including Pfizer and AstraZeneca, to enhance its research and development capabilities. The company has a long history and has evolved significantly, changing its name to Bristol-Myers Squibb in 1989.
Janssen, a subsidiary of Johnson & Johnson, is a pharmaceutical company focused on transforming healthcare through innovative medicines and solutions. Founded in 1953 by Dr. Paul Janssen, the company conducts extensive research and development across various therapeutic areas, including oncology, mental health, neurological disorders, gastrointestinal issues, and immunology. Janssen aims to address serious health concerns by delivering effective and affordable treatments, ensuring that patients have access to the best available care. The company is committed to improving healthcare delivery and patient outcomes through the integration of new technologies and consumer empowerment. By addressing unmet medical needs, Janssen continues to advance the science of medicine while striving to create a healthier world for all.
Biohaven is a clinical-stage biopharmaceutical company. It has a portfolio of late-stage product candidates targeting neurological diseases, including rare disorders. The company product candidates are based on multiple mechanisms-calcitonin gene-related peptide receptor antagonists, glutamate modulators and myeloperoxidase inhibitor. Its pipeline products include BHV3000-301, BHV3000-302, BHV3000-303, and others.
Intarcia Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapies for chronic conditions, particularly type 2 diabetes. The company is advancing its lead product, ITCA 650, through a phase 3 clinical program involving four separate clinical trials, aimed at providing patients with long-term stable dosing of an incretin mimetic therapy. Intarcia also offers the Medici Drug Delivery System, a subcutaneous platform designed to enhance the administration of drug therapies for various chronic diseases, including obesity and autoimmune disorders. Founded in 1997 as BioMedicines, the company rebranded to Intarcia Therapeutics in 2004. It is headquartered in Boston, Massachusetts, with additional facilities in Hayward, California, and a research and development center in Durham, North Carolina.
Farcast is a clinical diagnostic company dedicated to providing personalized cancer treatment solutions. Its core technology utilizes a human tumor microenvironment to enable oncologists and drug developers to assess individual tumors and determine their treatment response profiles. This innovative approach aims to surpass traditional biomarker-guided strategies, thereby enhancing access to personalized medicine and improving the clinical management of cancer. Headquartered in Boston, Massachusetts, Farcast also operates a laboratory in Bangalore, India, reflecting its commitment to advancing cancer care through cutting-edge technology.
Antera Therapeutics is a biotechnology company that specializes in creating therapies that will prevent allergy development in children. It helps families introduce peanuts into infants' diets in a structured and convenient way. It was founded in 2014 and headquartered in Boston, Massachusetts.
Adeo Health Science, Inc. develops and produces Aralyte, an organic food for special dietary use that offers parents a structured means of implementing the LEAP (learning early about peanut allergy) regimen. Its Aralyte contains natural distilled peanut extract and Vitamin D for babies just starting solid food. The company offers its products online. Adeo Health Science, Inc. was formerly known as Antera Therapeutics, Inc. and changed its name to Adeo Health Science, Inc. in September 2018. The company founded in 2014 and is based in Boston, Massachusetts.
TARIS BioMedical, Inc., a therapeutically urology company, develops treatments for patients suffering from difficult-to-treat bladder diseases. It develops therapies for debilitating conditions, including bladder cancer and overactive bladder. The company was founded in 2008 and is based in Lexington, Massachusetts. As of December 20, 2019, TARIS BioMedical, Inc. operates as a subsidiary of Janssen Research & Development, LLC.
Arvinas is a pharmaceutical company that focuses on developing novel therapeutics to treat a variety of diseases including cancer, pro-inflammatory, autoimmune and rare diseases. Its proprietary approach is the targeting of disease-causing proteins for degradation thus eliminating them from the body.
The company was founded in 2013 and is headquartered in Connecticut, United States.
Aclaris Therapeutics started by the founders of Vicept Therapeutics, is a privately held specialty pharmaceutical dermatology company focused on the development of novel dermatologic therapies. It is committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in dermatology, both medical and aesthetic, and immunology. Aclaris is focused on market segments with no FDA-approved medications or where treatment gaps exist.
Wave Life Sciences Ltd. is a clinical stage genetic medicine company that specializes in the design and production of novel stereopure oligonucleotides using its proprietary PRISM platform. Focused on addressing genetic defects, the company develops therapies aimed at reducing the expression of harmful proteins or converting dysfunctional proteins into functional ones. Its primary research areas include neurologic conditions affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to advance its oligonucleotide therapeutics. Founded in 2012, the company is headquartered in Singapore and maintains research facilities in Boston and Japan. The founding scientists, Gregory Verdine and Takeshi Wada, are recognized leaders in the field, contributing their expertise to the development of innovative nucleic acid therapeutics.
Ra Pharma is developing Cyclomimetics, a new drug class with the diversity and specificity of antibodies, coupled with the many benefits of small molecules. Ra Pharma is leveraging its ability to rapidly generate drug candidates to develop its own portfolio of products and partnerships focused on intracellular protein-protein interactions and other innovative approaches to addressing unmet medical needs.
It was founded in 2008 and headquartered in Cambridge, Massachusetts.
KalVista Pharmaceuticals, Inc., a clinical stage pharmaceutical company, discovers, develops, and commercializes small molecule protease inhibitors for diseases with unmet needs. The company’s product portfolio comprises small molecule plasma kallikrein inhibitors targeting hereditary angioedema (HAE) and diabetic macular edema (DME); and oral plasma kallikrein inhibitors. Its products include KVD001, a plasma kallikrein inhibitor that completed a Phase II clinical trial for the treatment of DME; KVD900, which is in Phase II clinical trial for treating HAE attacks; and KVD824, an oral plasma kallikrein inhibitor that completed a Phase I clinical trial for treating DME or HAE. The company is headquartered in Cambridge, Massachusetts.
Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. Their team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases.
The company is focused on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics.
In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX Biosciences. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV vector technology and related applications. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights within REGENX product programs in multiple rare disease indications. Dimension has completed a Series A financing led by Fidelity Biosciences.
Collegium Pharmaceutical is a specialty pharmaceutical company that develops and commercializes products aimed at treating patients suffering from pain, particularly in relation to opioid medications. The company utilizes its proprietary DETERx platform technology, which enhances the safety and extended-release properties of medications while minimizing the risk of misuse through methods such as chewing, crushing, and injecting. Among its key offerings is Xtampza ER, an abuse-deterrent, extended-release formulation of oxycodone, alongside Nucynta ER and Nucynta IR, which are formulations of tapentadol designed for managing severe and moderate to severe pain, respectively. Collegium focuses on addressing the challenges related to the non-medical use of prescription drugs by emphasizing tamper-resistant features and innovative delivery systems in its product portfolio. Founded in 2002 and based in Stoughton, Massachusetts, the company aims to provide effective pain management solutions while prioritizing patient safety.
Agile Therapeutics, Inc., a specialty pharmaceutical company, engages in the development of women's healthcare products. It offers a low estrogen dose seven-day transdermal contraceptive patch system that delivers a combination of levonorgestrel and Ethinylestradiol.
The company was founded in 1997 as Levotech, Inc. and changed its name to Agile Therapeutics, Inc. in April 2001. It is based in Princeton, New Jersey.
Wave Life Sciences Ltd. is a clinical stage genetic medicine company that specializes in the design and production of novel stereopure oligonucleotides using its proprietary PRISM platform. Focused on addressing genetic defects, the company develops therapies aimed at reducing the expression of harmful proteins or converting dysfunctional proteins into functional ones. Its primary research areas include neurologic conditions affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to advance its oligonucleotide therapeutics. Founded in 2012, the company is headquartered in Singapore and maintains research facilities in Boston and Japan. The founding scientists, Gregory Verdine and Takeshi Wada, are recognized leaders in the field, contributing their expertise to the development of innovative nucleic acid therapeutics.
Moderna, Inc. is a biotechnology company based in Cambridge, Massachusetts, founded in 2010. It specializes in the development of therapeutics and vaccines utilizing messenger RNA technology, which instructs cells to produce proteins essential for various biological functions. The company's extensive pipeline includes 40 mRNA development candidates targeting a range of therapeutic areas such as infectious diseases, oncology, cardiovascular diseases, and rare genetic disorders. Moderna gained significant recognition with its COVID-19 vaccine, which was authorized for use in the United States in December 2020. The company maintains strategic partnerships with organizations such as AstraZeneca, Merck & Co., and the Bill & Melinda Gates Foundation, enhancing its research capabilities and manufacturing processes. Additionally, it has research collaborations with esteemed institutions like Harvard University.
Blueprint Medicines Corporation focuses on developing small molecule kinase inhibitors aimed at treating genomic drivers in cancers, rare diseases, and enhancing cancer immunotherapy. The company is advancing several key drug candidates, including avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis and other mast cell disorders, and fisogatinib for advanced hepatocellular carcinoma, currently in Phase I clinical trials. Additionally, pralsetinib targets RET-altered non-small cell lung cancer and other solid tumors, while BLU-782 is being investigated for fibrodysplasia ossificans progressive. Founded in 2008 and headquartered in Cambridge, Massachusetts, Blueprint Medicines emphasizes the development of personalized cancer therapies by leveraging insights into cancer genomics and utilizing a proprietary chemical library to identify new therapeutic compounds. The company collaborates with various partners, including Clementia Pharmaceuticals and Genentech, to enhance its research and development efforts.
Civitas Therapeutics is a biopharmaceutical company dedicated to developing therapies for pulmonary delivery. The company focuses on creating innovative treatments for central nervous system and respiratory disorders, with a particular emphasis on conditions such as multiple sclerosis and spinal cord injuries. Civitas Therapeutics aims to improve the health and quality of life for patients across the United States through its specialized drug delivery systems.
Intarcia Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapies for chronic conditions, particularly type 2 diabetes. The company is advancing its lead product, ITCA 650, through a phase 3 clinical program involving four separate clinical trials, aimed at providing patients with long-term stable dosing of an incretin mimetic therapy. Intarcia also offers the Medici Drug Delivery System, a subcutaneous platform designed to enhance the administration of drug therapies for various chronic diseases, including obesity and autoimmune disorders. Founded in 1997 as BioMedicines, the company rebranded to Intarcia Therapeutics in 2004. It is headquartered in Boston, Massachusetts, with additional facilities in Hayward, California, and a research and development center in Durham, North Carolina.