Third Rock Ventures

Casma Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing innovative therapeutic strategies centered on the natural cellular process of autophagy. Founded in 2017, the company aims to enhance autophagy to improve the clearance of unwanted proteins, organelles, and pathogens, thereby addressing significant medical challenges. Casma Therapeutics is advancing preclinical programs targeting various conditions, including muscle disorders, liver disease, inflammatory disorders, and neurodegenerative diseases. By harnessing and manipulating the autophagy system, the company seeks to arrest or reverse disease progression, ultimately enabling physicians to meet unmet medical needs and provide effective treatments for patients with serious health issues.

Founded in 2019 and based in Cambridge, Massachusetts, MoMa Therapeutics discovers precision medicines by targeting molecular machines underlying human diseases. The company's platform enables drug development from previously intractable enzyme classes.

Celsius Therapeutics, Inc. is a biotechnology company focused on developing precision medicines for patients suffering from cancer and autoimmune diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company employs a multidisciplinary approach, integrating the expertise of scientists, computational biologists, and clinicians. Celsius Therapeutics utilizes advanced techniques such as single-cell genomic analysis and machine learning to identify and develop innovative therapeutic solutions. By leveraging these cutting-edge technologies, the company aims to create life-changing medicines that address unmet medical needs.

Cedilla Therapeutics, Inc. is a biotechnology company focused on developing targeted small-molecule therapeutics for cancer and diseases linked to protein dysregulation. The company employs innovative strategies to destabilize oncoprotein targets, utilizing methods such as direct ligand-induced degradation, disruption of stabilizing protein-protein interactions, and the identification of upstream regulators that affect protein stability. Cedilla's comprehensive approach also includes large-scale proteomic analyses to map protein susceptibility, enhancing the potential for effective therapies. By concentrating on the transitions between stable and susceptible protein states, Cedilla aims to create novel treatments that broaden the applicability of small molecule therapeutics beyond oncology. Founded in 2017 and headquartered in Cambridge, Massachusetts, the company was formerly known as Protein Stability Newco, Inc. before rebranding in 2018.

Faze Medicines is a biotechnology company focused on developing small molecule therapies that target biomolecular condensates to address the underlying biology of disease. It uses screening and proteomics to define condensate interaction networks and identify therapeutic candidates aimed at cellular mechanisms implicated in conditions such as amyotrophic lateral sclerosis and myotonic dystrophy type 1, with research extending to other diseases including frontotemporal dementia. Founded in 2020 and based in Cambridge, Massachusetts.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Cedilla Therapeutics, Inc. is a biotechnology company focused on developing targeted small-molecule therapeutics for cancer and diseases linked to protein dysregulation. The company employs innovative strategies to destabilize oncoprotein targets, utilizing methods such as direct ligand-induced degradation, disruption of stabilizing protein-protein interactions, and the identification of upstream regulators that affect protein stability. Cedilla's comprehensive approach also includes large-scale proteomic analyses to map protein susceptibility, enhancing the potential for effective therapies. By concentrating on the transitions between stable and susceptible protein states, Cedilla aims to create novel treatments that broaden the applicability of small molecule therapeutics beyond oncology. Founded in 2017 and headquartered in Cambridge, Massachusetts, the company was formerly known as Protein Stability Newco, Inc. before rebranding in 2018.

Casma Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing innovative therapeutic strategies centered on the natural cellular process of autophagy. Founded in 2017, the company aims to enhance autophagy to improve the clearance of unwanted proteins, organelles, and pathogens, thereby addressing significant medical challenges. Casma Therapeutics is advancing preclinical programs targeting various conditions, including muscle disorders, liver disease, inflammatory disorders, and neurodegenerative diseases. By harnessing and manipulating the autophagy system, the company seeks to arrest or reverse disease progression, ultimately enabling physicians to meet unmet medical needs and provide effective treatments for patients with serious health issues.

Thrive Earlier Detection is a healthcare company dedicated to saving lives through early detection of cancer. It develops CancerSEEK, a liquid biopsy test designed to identify various cancers in their earliest stages via blood analysis.

Founded in 2019 and based in Cambridge, Massachusetts, MoMa Therapeutics discovers precision medicines by targeting molecular machines underlying human diseases. The company's platform enables drug development from previously intractable enzyme classes.

Thrive Earlier Detection is a healthcare company dedicated to saving lives through early detection of cancer. It develops CancerSEEK, a liquid biopsy test designed to identify various cancers in their earliest stages via blood analysis.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Celsius Therapeutics, Inc. is a biotechnology company focused on developing precision medicines for patients suffering from cancer and autoimmune diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company employs a multidisciplinary approach, integrating the expertise of scientists, computational biologists, and clinicians. Celsius Therapeutics utilizes advanced techniques such as single-cell genomic analysis and machine learning to identify and develop innovative therapeutic solutions. By leveraging these cutting-edge technologies, the company aims to create life-changing medicines that address unmet medical needs.

Casma Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing innovative therapeutic strategies centered on the natural cellular process of autophagy. Founded in 2017, the company aims to enhance autophagy to improve the clearance of unwanted proteins, organelles, and pathogens, thereby addressing significant medical challenges. Casma Therapeutics is advancing preclinical programs targeting various conditions, including muscle disorders, liver disease, inflammatory disorders, and neurodegenerative diseases. By harnessing and manipulating the autophagy system, the company seeks to arrest or reverse disease progression, ultimately enabling physicians to meet unmet medical needs and provide effective treatments for patients with serious health issues.

Cedilla Therapeutics, Inc. is a biotechnology company focused on developing targeted small-molecule therapeutics for cancer and diseases linked to protein dysregulation. The company employs innovative strategies to destabilize oncoprotein targets, utilizing methods such as direct ligand-induced degradation, disruption of stabilizing protein-protein interactions, and the identification of upstream regulators that affect protein stability. Cedilla's comprehensive approach also includes large-scale proteomic analyses to map protein susceptibility, enhancing the potential for effective therapies. By concentrating on the transitions between stable and susceptible protein states, Cedilla aims to create novel treatments that broaden the applicability of small molecule therapeutics beyond oncology. Founded in 2017 and headquartered in Cambridge, Massachusetts, the company was formerly known as Protein Stability Newco, Inc. before rebranding in 2018.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Rheos Medicines, Inc. is a biopharmaceutical company focused on developing innovative therapeutics that modulate metabolic pathways in immune cells to address immune-mediated diseases such as inflammatory bowel disease, rheumatoid arthritis, and lupus. Founded in 2017 and based in Cambridge, Massachusetts, Rheos Medicines employs a unique approach that targets the cellular metabolism of immune cells, allowing for the redirection of specific cell types to enhance treatment efficacy. The company aims to bring precision to the management of these diseases by identifying new drug targets and characterizing relevant biomarkers. Rheos Medicines has assembled a team of leading scientists and clinicians with expertise in immunometabolism and immune-mediated disorders, supported by an experienced biotech leadership team.

Relay Therapeutics, Inc. is a clinical-stage precision medicine company based in Cambridge, Massachusetts, focused on transforming the drug discovery process. The company employs a novel allosteric drug-discovery platform, known as Dynamo, which integrates advanced computational techniques with experimental methods to study protein motion and interactions. This innovative approach enables the identification and development of therapies targeting complex diseases, particularly in oncology. Relay Therapeutics is advancing a pipeline of product candidates, including RLY-1971, a small molecule inhibitor targeting SHP2, RLY-4008, a selective inhibitor of FGFR2 for advanced solid tumors, and RLY-PI3K1047, aimed at mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, the company aims to deliver life-changing therapies to patients by addressing previously intractable targets in precision oncology and genetic diseases.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Magenta Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on advancing stem cell transplantation and related therapies. Founded in 2015, the company aims to enhance the patient experience in transplant medicine by developing innovative treatments that make stem cell transplants safer and more accessible. Its pipeline includes several key candidates, such as C100, C200, and C300 targeted antibody-drug conjugates designed for transplant conditioning; MGTA-145, a stem cell mobilization product; and MGTA-456, an allogeneic stem cell therapy. Additionally, Magenta is exploring E478, a small molecule aimed at expanding gene-modified stem cells, and G100, an antibody-drug conjugate to mitigate acute graft-versus-host disease. The company has established research collaborations with other biotechnology firms to further investigate the potential of its therapies in treating blood cancers, genetic disorders, and autoimmune diseases, ultimately striving to transform stem cell transplantation from a last-resort option into a more viable and effective treatment for patients.

Tango Therapeutics is a biotechnology company focused on developing cancer therapies based on the genetic principle of synthetic lethality. It aims to counteract tumor suppressor gene loss, reverse immune evasion in cancer cells, and identify effective drug combinations.

Goldfinch Bio, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, dedicated to discovering and developing precision therapies for patients with kidney diseases. The company focuses on conditions such as focal segmental glomerulosclerosis, diabetic nephropathy, and polycystic kidney disease. Key product candidates include GFB-887, a selective inhibitor of the TRPC5 channel, and GFB-024, a cannabinoid receptor 1 inverse agonist. Goldfinch Bio integrates advancements in kidney genetics and biology through its Kidney Genome Atlas, a genomic registry that aids in identifying new disease targets and biomarkers. Additionally, the company employs stem cell science to create human organoid models, which facilitate the validation of novel therapeutic targets. Goldfinch Bio has established a strategic collaboration with Gilead Sciences to further enhance its research and development efforts. Founded in 2016, Goldfinch Bio aims to transform the treatment landscape for progressive kidney disease.

Magenta Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on advancing stem cell transplantation and related therapies. Founded in 2015, the company aims to enhance the patient experience in transplant medicine by developing innovative treatments that make stem cell transplants safer and more accessible. Its pipeline includes several key candidates, such as C100, C200, and C300 targeted antibody-drug conjugates designed for transplant conditioning; MGTA-145, a stem cell mobilization product; and MGTA-456, an allogeneic stem cell therapy. Additionally, Magenta is exploring E478, a small molecule aimed at expanding gene-modified stem cells, and G100, an antibody-drug conjugate to mitigate acute graft-versus-host disease. The company has established research collaborations with other biotechnology firms to further investigate the potential of its therapies in treating blood cancers, genetic disorders, and autoimmune diseases, ultimately striving to transform stem cell transplantation from a last-resort option into a more viable and effective treatment for patients.

Relay Therapeutics, Inc. is a clinical-stage precision medicine company based in Cambridge, Massachusetts, focused on transforming the drug discovery process. The company employs a novel allosteric drug-discovery platform, known as Dynamo, which integrates advanced computational techniques with experimental methods to study protein motion and interactions. This innovative approach enables the identification and development of therapies targeting complex diseases, particularly in oncology. Relay Therapeutics is advancing a pipeline of product candidates, including RLY-1971, a small molecule inhibitor targeting SHP2, RLY-4008, a selective inhibitor of FGFR2 for advanced solid tumors, and RLY-PI3K1047, aimed at mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, the company aims to deliver life-changing therapies to patients by addressing previously intractable targets in precision oncology and genetic diseases.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Rhythm Metabolic is the subsidiary of Rhythm Pharmaceuticals that develops setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway. It was established in 2013 and is headquartered in Boston, Massachusetts.

Editas Medicine is a clinical-stage biotechnology company based in Cambridge, Massachusetts, with an additional site in Boulder, Colorado. Founded in 2013, the company focuses on the discovery and development of genome editing therapeutics, primarily utilizing its proprietary CRISPR/Cas9 technology. This innovative platform enables precise molecular modifications at the genetic level, with the aim of treating a wide array of serious diseases by addressing their underlying causes. Editas Medicine has established a strong intellectual property portfolio, which includes numerous patents related to foundational genome editing technologies and advancements that facilitate the translation of research into viable human therapeutics. The company's mission is to pioneer transformative genomic medicines that can significantly improve patient outcomes across various genetic disorders.

Allena Pharmaceuticals, Inc. is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, focused on the development and commercialization of innovative oral enzyme therapeutics for rare and severe metabolic and kidney disorders. Founded in 2011, the company’s lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a condition characterized by elevated urinary oxalate levels that can lead to kidney stones and chronic kidney disease. Additionally, Allena is developing ALLN-346, a novel enzyme intended to address hyperuricemia in patients with moderate to severe chronic kidney diseases. By targeting metabolic disorders associated with the accumulation of harmful metabolites, Allena Pharmaceuticals aims to provide effective treatments for patients facing serious health challenges.

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

Corvia Medical, Inc., established in 2009 and based in Andover, Massachusetts, is a medical device company specializing in structural heart devices for heart failure treatment. Its primary product is the InterAtrial Shunt Device (IASD), a transcatheter device designed to alleviate symptoms and slow the progression of diastolic heart failure, also known as heart failure with preserved ejection fraction (HFpEF). The IASD system reduces elevated left atrial pressure, the primary cause of DHF symptoms, by creating a small opening between the left and right atria, enabling patients to lead healthier lives with reduced heart failure risk.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

NinePoint Medical, Inc. is a medical device company based in Bedford, Massachusetts, that specializes in the development of innovative optical coherence tomography (OCT) imaging systems for assessing human tissue microstructure. The company's flagship product, the NvisionVLE Imaging System, enables real-time volumetric imaging of the esophagus and bile duct, allowing physicians across various specialties—including gastroenterology, pulmonology, urology, gynecology, and ENT—to perform advanced diagnostic procedures. By providing high-resolution cross-sectional images, NinePoint's system empowers physicians to make accurate diagnoses and implement effective therapies during the same procedure, enhancing patient care and outcomes. The company markets its imaging systems through a network of distributors in the United States and internationally. Originally founded as Convergence Devices, Inc. in 2008, NinePoint Medical underwent a name change in 2009 and has since focused on advancing telepathology capabilities to improve healthcare efficiency and patient experiences.

SAGE Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, dedicated to developing innovative treatments for central nervous system (CNS) disorders. The company's lead product, ZULRESSO, is an intravenous formulation of brexanolone specifically designed for the treatment of postpartum depression. SAGE Therapeutics is advancing a diverse product pipeline, including SAGE-217, a novel neuroactive steroid in Phase III trials for various depressive disorders and anxiety, and SAGE-324, currently in Phase II trials for essential tremors and other neurological conditions. Additionally, SAGE-718, an oxysterol-based positive allosteric modulator, has completed Phase I trials targeting multiple CNS-related disorders. The company also explores other compounds, such as SAGE-904 and SAGE-689. SAGE Therapeutics has established strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. for the development and commercialization of its key product candidates, emphasizing its commitment to addressing the challenges of brain health.

Voyager Therapeutics, Inc. is a clinical-stage gene therapy company dedicated to developing innovative treatments for severe neurological diseases. The company’s lead clinical candidate, VY-AADC, is currently undergoing an open-label Phase 1b trial for Parkinson’s disease. Its diverse preclinical pipeline includes candidates for various conditions, such as VY-SOD102 for amyotrophic lateral sclerosis, VY-HTT01 for Huntington’s disease, VY-FXN01 for Friedreich’s ataxia, and programs targeting tauopathies and synucleinopathies, which encompass Alzheimer’s disease and related disorders. Voyager Therapeutics employs a gene therapy platform known as TRACER, which enables the engineering and optimization of adeno-associated virus (AAV) therapies aimed at producing lasting therapeutic effects from a single treatment. The company collaborates with several institutions, including the University of Massachusetts and ClearPoint Neuro, as well as industry partners like Brammer Bio and Fujifilm Diosynth Biotechnologies, to enhance its gene therapy initiatives. Founded in 2013 and headquartered in Cambridge, Massachusetts, Voyager Therapeutics is committed to addressing unmet medical needs in the central nervous system.

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

Soffio Medical, Inc. is a Boston-based company focused on developing innovative medical devices for respiratory diseases. The firm specializes in implantable technologies that aim to address chronic obstructive pulmonary disease (COPD) and similar conditions. Its devices are designed to implement an airway bypass concept, allowing for the venting of trapped air from areas distal to high-resistance airways. This approach helps reduce residual lung volume and alleviates symptoms associated with respiratory diseases, ultimately enhancing the quality of life for patients.

Editas Medicine is a clinical-stage biotechnology company based in Cambridge, Massachusetts, with an additional site in Boulder, Colorado. Founded in 2013, the company focuses on the discovery and development of genome editing therapeutics, primarily utilizing its proprietary CRISPR/Cas9 technology. This innovative platform enables precise molecular modifications at the genetic level, with the aim of treating a wide array of serious diseases by addressing their underlying causes. Editas Medicine has established a strong intellectual property portfolio, which includes numerous patents related to foundational genome editing technologies and advancements that facilitate the translation of research into viable human therapeutics. The company's mission is to pioneer transformative genomic medicines that can significantly improve patient outcomes across various genetic disorders.

SAGE Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, dedicated to developing innovative treatments for central nervous system (CNS) disorders. The company's lead product, ZULRESSO, is an intravenous formulation of brexanolone specifically designed for the treatment of postpartum depression. SAGE Therapeutics is advancing a diverse product pipeline, including SAGE-217, a novel neuroactive steroid in Phase III trials for various depressive disorders and anxiety, and SAGE-324, currently in Phase II trials for essential tremors and other neurological conditions. Additionally, SAGE-718, an oxysterol-based positive allosteric modulator, has completed Phase I trials targeting multiple CNS-related disorders. The company also explores other compounds, such as SAGE-904 and SAGE-689. SAGE Therapeutics has established strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. for the development and commercialization of its key product candidates, emphasizing its commitment to addressing the challenges of brain health.

TARIS Biomedical, Inc. is a therapeutically focused urology company based in Lexington, Massachusetts, established in 2008. The company specializes in developing innovative treatments for challenging bladder diseases, including bladder cancer and overactive bladder. TARIS Biomedical's proprietary system is designed to continuously release medications directly into the bladder over extended periods, ranging from weeks to months. This technology is administered through minimally invasive, in-office procedures, allowing for customized drug delivery that aligns with the specific needs of various bladder conditions. As of December 2019, TARIS Biomedical operates as a subsidiary of Janssen Research & Development, LLC.

Corvia Medical, Inc., established in 2009 and based in Andover, Massachusetts, is a medical device company specializing in structural heart devices for heart failure treatment. Its primary product is the InterAtrial Shunt Device (IASD), a transcatheter device designed to alleviate symptoms and slow the progression of diastolic heart failure, also known as heart failure with preserved ejection fraction (HFpEF). The IASD system reduces elevated left atrial pressure, the primary cause of DHF symptoms, by creating a small opening between the left and right atria, enabling patients to lead healthier lives with reduced heart failure risk.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Jounce Therapeutics, Inc. is a clinical-stage immunotherapy company based in Cambridge, Massachusetts, focused on developing innovative therapies for cancer treatment. The company aims to harness the immune system to target and eliminate cancerous cells, thereby enhancing patient outcomes and quality of life. Among its key product candidates is vopratelimab, a monoclonal antibody currently undergoing Phase II clinical trials for patients with non-small cell lung cancer and urothelial cancer. Jounce is also advancing JTX-4014, an anti-PD-1 antibody for combination therapy, and JTX-1811, which is designed to deplete T regulatory cells in the tumor microenvironment. Additionally, JTX-8064 targets a specific receptor on macrophages to modulate the immune response. Founded in 2013 by experts in immunobiology and cancer biology, Jounce utilizes a proprietary Translational Science Platform to analyze the cellular and molecular characteristics of tumors, driving its commitment to developing first-in-class cancer immunotherapies.

Motus Therapeutics is a biotechnology company located in Boston, Massachusetts, focused on developing peptide therapeutics to address unmet needs in metabolic diseases. The company specializes in treatments for gastrointestinal disorders, with its lead product candidate, relamorelin, acting as a ghrelin agonist. Relamorelin is specifically designed for the treatment of diabetic gastroparesis, a gastrointestinal complication associated with diabetes, as well as other gastrointestinal functional disorders. Through its innovative approach, Motus Therapeutics aims to improve the quality of life for patients suffering from these conditions.

Foundation Medicine, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in providing molecular information products for cancer diagnosis and treatment. The company utilizes advanced genomic profiling technologies to analyze tumor specimens, enabling healthcare providers to gain insights into individual patients' cancer profiles. This information assists in optimizing treatment strategies, particularly for targeted therapies and immunotherapies. Foundation Medicine’s product offerings include FoundationOne for solid tumors, FoundationOne Heme for blood-based cancers, and various diagnostic assays to support clinical decision-making in oncology. The company also maintains a knowledgebase, FoundationCORE, to disseminate scientific advancements and strengthen collaborations within the oncology community. Foundation Medicine has established strategic partnerships with major pharmaceutical companies and research organizations to enhance precision medicine initiatives and develop companion diagnostics for specific therapies. Founded in 2009, Foundation Medicine operates as a subsidiary of Roche Holdings, Inc.

Bluebird Bio is a clinical-stage biotechnology company focused on developing gene therapies to treat severe genetic and rare diseases. Its proprietary lentiviral vector platform enables the creation of potentially curative treatments by genetically modifying patients' cells to address the root cause of these conditions.

Motus Therapeutics is a biotechnology company located in Boston, Massachusetts, focused on developing peptide therapeutics to address unmet needs in metabolic diseases. The company specializes in treatments for gastrointestinal disorders, with its lead product candidate, relamorelin, acting as a ghrelin agonist. Relamorelin is specifically designed for the treatment of diabetic gastroparesis, a gastrointestinal complication associated with diabetes, as well as other gastrointestinal functional disorders. Through its innovative approach, Motus Therapeutics aims to improve the quality of life for patients suffering from these conditions.

Sesen Bio, Inc. is a late-stage clinical company based in Cambridge, Massachusetts, that focuses on the design, development, and commercialization of targeted fusion protein therapeutics for cancer treatment. The company's lead product candidate, Vicinium, is a locally-administered therapy currently in phase 3 clinical trials aimed at treating high-risk non-muscle invasive bladder cancer. Additionally, Sesen Bio is developing VB6-845d, a systemically-administered product for various EpCAM-positive solid tumors. The company is also exploring combination therapies, including Vicinium with Durvalumab for high-risk non-muscle invasive bladder cancer and another combination for squamous cell carcinoma of the head and neck. Sesen Bio has partnered with Leiden University Medical Center to co-develop an imaging agent. Established in 2008 and previously known as Eleven Biotherapeutics, Sesen Bio has developed a proprietary platform that combines antibody fragments with cytotoxic proteins to create innovative therapies.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Warp Drive Bio, LLC is a biotechnology company based in Cambridge, Massachusetts, that specializes in the development of genomic technology to discover drugs derived from natural sources. Founded in 2010, the company has created a proprietary genomic search engine that utilizes customized search queries to identify hidden natural products based on their unique genomic signatures. This innovative approach enables researchers to uncover novel drug candidates and analyze their mechanisms of action, facilitating the conversion of genomes into therapeutic drugs. As of October 2018, Warp Drive Bio operates as a subsidiary of REVOLUTION Medicines, Inc.

Agios Pharmaceuticals is a biopharmaceutical company focused on developing targeted therapies in the field of cellular metabolism. It discovers and develops medicines for various indications, including hematologic malignancies, solid tumors, and rare genetic diseases.

Allena Pharmaceuticals, Inc. is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, focused on the development and commercialization of innovative oral enzyme therapeutics for rare and severe metabolic and kidney disorders. Founded in 2011, the company’s lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a condition characterized by elevated urinary oxalate levels that can lead to kidney stones and chronic kidney disease. Additionally, Allena is developing ALLN-346, a novel enzyme intended to address hyperuricemia in patients with moderate to severe chronic kidney diseases. By targeting metabolic disorders associated with the accumulation of harmful metabolites, Allena Pharmaceuticals aims to provide effective treatments for patients facing serious health challenges.

Foundation Medicine, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in providing molecular information products for cancer diagnosis and treatment. The company utilizes advanced genomic profiling technologies to analyze tumor specimens, enabling healthcare providers to gain insights into individual patients' cancer profiles. This information assists in optimizing treatment strategies, particularly for targeted therapies and immunotherapies. Foundation Medicine’s product offerings include FoundationOne for solid tumors, FoundationOne Heme for blood-based cancers, and various diagnostic assays to support clinical decision-making in oncology. The company also maintains a knowledgebase, FoundationCORE, to disseminate scientific advancements and strengthen collaborations within the oncology community. Foundation Medicine has established strategic partnerships with major pharmaceutical companies and research organizations to enhance precision medicine initiatives and develop companion diagnostics for specific therapies. Founded in 2009, Foundation Medicine operates as a subsidiary of Roche Holdings, Inc.

SAGE Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, dedicated to developing innovative treatments for central nervous system (CNS) disorders. The company's lead product, ZULRESSO, is an intravenous formulation of brexanolone specifically designed for the treatment of postpartum depression. SAGE Therapeutics is advancing a diverse product pipeline, including SAGE-217, a novel neuroactive steroid in Phase III trials for various depressive disorders and anxiety, and SAGE-324, currently in Phase II trials for essential tremors and other neurological conditions. Additionally, SAGE-718, an oxysterol-based positive allosteric modulator, has completed Phase I trials targeting multiple CNS-related disorders. The company also explores other compounds, such as SAGE-904 and SAGE-689. SAGE Therapeutics has established strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. for the development and commercialization of its key product candidates, emphasizing its commitment to addressing the challenges of brain health.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Bluebird Bio is a clinical-stage biotechnology company focused on developing gene therapies to treat severe genetic and rare diseases. Its proprietary lentiviral vector platform enables the creation of potentially curative treatments by genetically modifying patients' cells to address the root cause of these conditions.

TARIS Biomedical, Inc. is a therapeutically focused urology company based in Lexington, Massachusetts, established in 2008. The company specializes in developing innovative treatments for challenging bladder diseases, including bladder cancer and overactive bladder. TARIS Biomedical's proprietary system is designed to continuously release medications directly into the bladder over extended periods, ranging from weeks to months. This technology is administered through minimally invasive, in-office procedures, allowing for customized drug delivery that aligns with the specific needs of various bladder conditions. As of December 2019, TARIS Biomedical operates as a subsidiary of Janssen Research & Development, LLC.

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

NinePoint Medical, Inc. is a medical device company based in Bedford, Massachusetts, that specializes in the development of innovative optical coherence tomography (OCT) imaging systems for assessing human tissue microstructure. The company's flagship product, the NvisionVLE Imaging System, enables real-time volumetric imaging of the esophagus and bile duct, allowing physicians across various specialties—including gastroenterology, pulmonology, urology, gynecology, and ENT—to perform advanced diagnostic procedures. By providing high-resolution cross-sectional images, NinePoint's system empowers physicians to make accurate diagnoses and implement effective therapies during the same procedure, enhancing patient care and outcomes. The company markets its imaging systems through a network of distributors in the United States and internationally. Originally founded as Convergence Devices, Inc. in 2008, NinePoint Medical underwent a name change in 2009 and has since focused on advancing telepathology capabilities to improve healthcare efficiency and patient experiences.

Motus Therapeutics is a biotechnology company located in Boston, Massachusetts, focused on developing peptide therapeutics to address unmet needs in metabolic diseases. The company specializes in treatments for gastrointestinal disorders, with its lead product candidate, relamorelin, acting as a ghrelin agonist. Relamorelin is specifically designed for the treatment of diabetic gastroparesis, a gastrointestinal complication associated with diabetes, as well as other gastrointestinal functional disorders. Through its innovative approach, Motus Therapeutics aims to improve the quality of life for patients suffering from these conditions.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Bluebird Bio is a clinical-stage biotechnology company focused on developing gene therapies to treat severe genetic and rare diseases. Its proprietary lentiviral vector platform enables the creation of potentially curative treatments by genetically modifying patients' cells to address the root cause of these conditions.

Sesen Bio, Inc. is a late-stage clinical company based in Cambridge, Massachusetts, that focuses on the design, development, and commercialization of targeted fusion protein therapeutics for cancer treatment. The company's lead product candidate, Vicinium, is a locally-administered therapy currently in phase 3 clinical trials aimed at treating high-risk non-muscle invasive bladder cancer. Additionally, Sesen Bio is developing VB6-845d, a systemically-administered product for various EpCAM-positive solid tumors. The company is also exploring combination therapies, including Vicinium with Durvalumab for high-risk non-muscle invasive bladder cancer and another combination for squamous cell carcinoma of the head and neck. Sesen Bio has partnered with Leiden University Medical Center to co-develop an imaging agent. Established in 2008 and previously known as Eleven Biotherapeutics, Sesen Bio has developed a proprietary platform that combines antibody fragments with cytotoxic proteins to create innovative therapies.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Agios Pharmaceuticals is a biopharmaceutical company focused on developing targeted therapies in the field of cellular metabolism. It discovers and develops medicines for various indications, including hematologic malignancies, solid tumors, and rare genetic diseases.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.