Third Rock Ventures

Merida Biosciences is a new biotech company focused on developing precision treatments, particularly for autoimmune diseases. The company utilize a protein engineering platform to create unique Fc therapeutics, aiming to selectively bind to specific antibodies.

Trace Neuroscience is a biopharmaceutical company that aims to extend the promise of genetic medicine to patients with neurodegenerative illnesses.

Seaport Therapeutics is a clinical stage biopharmaceutical company focused on developing innovative neuropsychiatric medicines for patients with significant unmet needs. The company's approach emphasizes the creation of treatments that offer oral bioavailability, which allows the medications to bypass first-pass metabolism. This method aims to reduce hepatotoxicity and other adverse side effects commonly associated with neuropsychiatric treatments. By addressing these critical issues, Seaport Therapeutics seeks to improve the quality of life for individuals living with neuropsychiatric disorders.

Candid Therapeutics is a biotechnology company that focuses on the treatment of a range of immunological conditions.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Seaport Therapeutics is a clinical stage biopharmaceutical company focused on developing innovative neuropsychiatric medicines for patients with significant unmet needs. The company's approach emphasizes the creation of treatments that offer oral bioavailability, which allows the medications to bypass first-pass metabolism. This method aims to reduce hepatotoxicity and other adverse side effects commonly associated with neuropsychiatric treatments. By addressing these critical issues, Seaport Therapeutics seeks to improve the quality of life for individuals living with neuropsychiatric disorders.

Clasp Therapeutics is focused on advancing cancer treatment through its innovative immunotherapeutics platform. The company develops precision medicines designed to enhance the immune system's ability to identify and eliminate challenging tumors that possess common driver mutations, irrespective of the tumor type. By enabling the targeting of cancer-specific markers, Clasp Therapeutics aims to allow for precise and selective destruction of tumor cells, thereby improving therapeutic outcomes in the field of immuno-oncology.

Synnovation Therapeutics is a precision oncology company dedicated to developing small molecule therapies that target key mechanisms driving cancer. By leveraging expertise in medicinal chemistry, cancer biology, and patient-focused precision medicine, the company aims to create a diverse pipeline of innovative targeted therapies. Its focus on highly validated disease targets allows healthcare providers to address limitations in current treatment standards, ultimately striving to enhance patient outcomes and transform cancer care. Synnovation Therapeutics is committed to advancing these therapies efficiently into clinical trials, with the goal of improving the lives of individuals affected by cancer.

ARTBIO is a clinical-stage biotechnology company focused on developing innovative alpha radioligand therapies for cancer treatment. By utilizing radioligand therapy, ARTBIO aims to deliver alpha or beta emitters that target and disrupt cancer cell DNA, offering a new approach to oncology. The company is committed to creating a supportive ecosystem that enhances the efficacy of its therapeutics, thereby enabling medical professionals to utilize novel mechanisms in cancer care. Through its advancements, ARTBIO seeks to transform the landscape of cancer treatment and improve patient outcomes.

Terremoto Biosciences is a biotechnology company focused on drug delivery and discovery, specializing in small-molecule medicines. The company utilizes lysine-based covalency to develop optimized therapies with enhanced clinical profiles and superior therapeutic benefits. Terremoto's innovative platform aims to improve existing medicines and create new treatments for serious diseases, contributing to advancements in the healthcare sector.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing innovative small-molecule therapies for neurological disorders. The company focuses on understanding the intricate biology of neuronal receptor complexes, which are essential for proper neuronal function and signaling. By mapping and targeting these receptor systems, Rapport Therapeutics aims to create precision medicines that can effectively address the unmet needs of patients suffering from central nervous system disorders. Through its foundational research, the company seeks to transform treatment options and improve outcomes for individuals with these challenging conditions.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Flare Therapeutics is a biotechnology company focused on developing small molecule medicines by exploring the biology of transcription factors. The company aims to identify 'switch sites,' which are druggable regions crucial for regulating transcription factors, to target mutations associated with various diseases. Flare's innovative approach has advanced its drug discovery efforts, resulting in a promising pipeline of programs that primarily address well-established transcription factors. Initially concentrating on precision oncology, the company's research also holds potential applications in neurology, rare genetic disorders, immunology, and inflammation. By leveraging insights from its scientific founders, Flare Therapeutics seeks to transform the treatment landscape for cancer patients and other disease areas.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing innovative small-molecule therapies for neurological disorders. The company focuses on understanding the intricate biology of neuronal receptor complexes, which are essential for proper neuronal function and signaling. By mapping and targeting these receptor systems, Rapport Therapeutics aims to create precision medicines that can effectively address the unmet needs of patients suffering from central nervous system disorders. Through its foundational research, the company seeks to transform treatment options and improve outcomes for individuals with these challenging conditions.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

Casma Therapeutics, Inc. specializes in developing innovative therapeutic strategies centered on the natural cellular process of autophagy and lysosomal flux. By focusing on enhancing autophagy, the company aims to improve the clearance of unwanted proteins, organelles, and pathogens, thereby addressing the progression of various diseases. Its research targets several serious medical conditions, including lysosomal storage disorders, muscle disorders, inflammatory disorders, and neurodegeneration. Founded in 2017 and based in Cambridge, Massachusetts, Casma Therapeutics is dedicated to creating effective treatment options that meet significant unmet medical needs in the field of drug discovery and development.

Terremoto Biosciences is a biotechnology company focused on drug delivery and discovery, specializing in small-molecule medicines. The company utilizes lysine-based covalency to develop optimized therapies with enhanced clinical profiles and superior therapeutic benefits. Terremoto's innovative platform aims to improve existing medicines and create new treatments for serious diseases, contributing to advancements in the healthcare sector.

MOMA Therapeutics is a biotechnology company based in Cambridge, Massachusetts, founded in 2019. The company focuses on discovering precision medicines by targeting molecular machines that are fundamental to human disease. MOMA Therapeutics utilizes a unique platform that leverages the common features of the ATPase target class, which includes significant conformational changes and energy-driven mechanics. By combining these insights with advancements in structural biology and small-molecule drug discovery, MOMA Therapeutics aims to develop drugs targeting previously challenging enzymes, contributing to the next generation of targeted therapies. The company was formerly known as ATPases NewCo, Inc. before rebranding in March 2020.

Celsius Therapeutics is a biotechnology company focused on developing precision medicines for patients with cancer and autoimmune diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company employs a team of scientists, technologists, computational biologists, clinicians, and drug hunters. Utilizing single-cell genomic analysis and machine learning techniques, Celsius Therapeutics aims to discover and create innovative therapies that can lead to significant improvements in patient outcomes. The company's research efforts are centered on identifying new drug candidates that address unmet medical needs in the therapeutic landscape.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Merida Biosciences is a new biotech company focused on developing precision treatments, particularly for autoimmune diseases. The company utilize a protein engineering platform to create unique Fc therapeutics, aiming to selectively bind to specific antibodies.

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, founded in 2016. The company is dedicated to discovering and developing transformative therapies for severe liver diseases. Ambys focuses on creating innovative gene, drug, and cell therapies aimed at restoring liver function, halting the progression of liver disease, and addressing the complications associated with liver failure. By leveraging regenerative and restorative medical approaches, Ambys Medicines seeks to improve the lives of individuals affected by chronic liver conditions, providing potential relief through advanced therapeutic options.

Cedilla Therapeutics, Inc. is focused on developing targeted small-molecule medicines aimed at treating cancer and other diseases resulting from protein dysregulation. The company leverages unique insights into protein stability to create innovative therapeutic approaches, including direct ligand-induced degradation, disruption of protein-protein interactions, and the identification of upstream regulators that influence protein stability. Cedilla’s technology allows for the mapping of protein susceptibility to enhance endogenous degradation pathways, facilitating the transition from stable to susceptible protein states. Founded in 2018 and headquartered in Cambridge, Massachusetts, Cedilla Therapeutics aims to broaden the applicability of its therapies beyond oncology, addressing a range of conditions associated with protein dysregulation.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Abata Therapeutics is dedicated to developing innovative cell therapies aimed at treating autoimmune and inflammatory diseases, particularly progressive multiple sclerosis and type 1 diabetes. The company focuses on harnessing regulatory T cells (Tregs) to restore balance in the immune system, engineering these cells to specifically target disease-related antigens for precise treatment. This approach allows for effective disease management without the need for systemic immune suppression, enabling patients to maintain stable and active tissue residency. Founded by experts in immunology and cell therapy, Abata has garnered substantial investment from venture capital and biopharmaceutical firms, with the mission of translating advanced scientific research into meaningful therapies that enhance the quality of life for individuals affected by chronic autoimmune conditions.

Flare Therapeutics is a biotechnology company focused on developing small molecule medicines by exploring the biology of transcription factors. The company aims to identify 'switch sites,' which are druggable regions crucial for regulating transcription factors, to target mutations associated with various diseases. Flare's innovative approach has advanced its drug discovery efforts, resulting in a promising pipeline of programs that primarily address well-established transcription factors. Initially concentrating on precision oncology, the company's research also holds potential applications in neurology, rare genetic disorders, immunology, and inflammation. By leveraging insights from its scientific founders, Flare Therapeutics seeks to transform the treatment landscape for cancer patients and other disease areas.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Insitro, Inc. is a data-driven company focused on drug discovery and development, particularly targeting nonalcoholic steatohepatitis (NASH). Founded in 2018 by Daphne Koller and based in South San Francisco, California, Insitro harnesses machine learning and high-throughput biology to revolutionize the process of drug discovery. The company creates extensive functional genomic data sets and integrates them with patient data through innovative machine learning techniques. This approach allows Insitro to build predictive models that enhance target selection, streamline the design of therapeutics, and optimize drug development strategies. By addressing critical challenges in pharmaceutical research and development, Insitro aims to accelerate the delivery of new and effective treatments to patients.

Faze Medicines is a biotechnology company founded in 2020 and located in Cambridge, Massachusetts. The company focuses on developing small molecule drugs that target the underlying mechanisms of disease pathology, with initial therapeutic efforts directed towards conditions such as amyotrophic lateral sclerosis (ALS) and myotonic dystrophy type 1 (DM1). Faze Medicines employs advanced screening and proteomics techniques to explore biomolecular condensate interaction networks, which aids in the identification and development of novel therapeutics. Through its innovative approach, Faze aims to provide effective treatments for patients suffering from these challenging diseases.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

Cedilla Therapeutics, Inc. is focused on developing targeted small-molecule medicines aimed at treating cancer and other diseases resulting from protein dysregulation. The company leverages unique insights into protein stability to create innovative therapeutic approaches, including direct ligand-induced degradation, disruption of protein-protein interactions, and the identification of upstream regulators that influence protein stability. Cedilla’s technology allows for the mapping of protein susceptibility to enhance endogenous degradation pathways, facilitating the transition from stable to susceptible protein states. Founded in 2018 and headquartered in Cambridge, Massachusetts, Cedilla Therapeutics aims to broaden the applicability of its therapies beyond oncology, addressing a range of conditions associated with protein dysregulation.

Casma Therapeutics, Inc. specializes in developing innovative therapeutic strategies centered on the natural cellular process of autophagy and lysosomal flux. By focusing on enhancing autophagy, the company aims to improve the clearance of unwanted proteins, organelles, and pathogens, thereby addressing the progression of various diseases. Its research targets several serious medical conditions, including lysosomal storage disorders, muscle disorders, inflammatory disorders, and neurodegeneration. Founded in 2017 and based in Cambridge, Massachusetts, Casma Therapeutics is dedicated to creating effective treatment options that meet significant unmet medical needs in the field of drug discovery and development.

Thrive Earlier Detection Corp. is a healthcare company that specializes in developing advanced blood testing technologies for early cancer detection. Its flagship product, CancerSEEK, is a liquid biopsy test that analyzes genomic mutations in tumor DNA and protein markers in plasma, allowing for the identification of various cancers at their nascent stages, often before symptoms manifest. Founded in 2018 and headquartered in Cambridge, Massachusetts, with an additional office in Baltimore, Maryland, Thrive aims to integrate cancer screening into standard medical practice. The company leverages real-world data and machine learning to enhance the accuracy of cancer detection, facilitate follow-up testing, and streamline the transition to oncological care. Thrive operates as a subsidiary of Exact Sciences Corporation and has received investments from notable firms including Third Rock Ventures and Section 32.

Insitro, Inc. is a data-driven company focused on drug discovery and development, particularly targeting nonalcoholic steatohepatitis (NASH). Founded in 2018 by Daphne Koller and based in South San Francisco, California, Insitro harnesses machine learning and high-throughput biology to revolutionize the process of drug discovery. The company creates extensive functional genomic data sets and integrates them with patient data through innovative machine learning techniques. This approach allows Insitro to build predictive models that enhance target selection, streamline the design of therapeutics, and optimize drug development strategies. By addressing critical challenges in pharmaceutical research and development, Insitro aims to accelerate the delivery of new and effective treatments to patients.

MOMA Therapeutics is a biotechnology company based in Cambridge, Massachusetts, founded in 2019. The company focuses on discovering precision medicines by targeting molecular machines that are fundamental to human disease. MOMA Therapeutics utilizes a unique platform that leverages the common features of the ATPase target class, which includes significant conformational changes and energy-driven mechanics. By combining these insights with advancements in structural biology and small-molecule drug discovery, MOMA Therapeutics aims to develop drugs targeting previously challenging enzymes, contributing to the next generation of targeted therapies. The company was formerly known as ATPases NewCo, Inc. before rebranding in March 2020.

Nurix Therapeutics is a biopharmaceutical company specializing in the discovery, development, and commercialization of small molecule therapies aimed at modulating cellular protein levels to treat cancer and immune disorders. The company's pipeline includes promising preclinical candidates such as NX-2127, an orally available Bruton’s tyrosine kinase degrader targeted at relapsed or refractory B-cell malignancies, and NX-1607, a Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology applications. Nurix's innovative drug discovery platform, DELigase, leverages its expertise in E3 ligases—enzymes that regulate protein modulation within cells—to identify and advance novel drug candidates. Founded in 2009 and based in San Francisco, Nurix Therapeutics aims to provide effective therapies for challenging diseases through its cutting-edge research and development efforts.

Element Science, Inc. is a medical device and digital health company focused on developing solutions for high-risk cardiovascular patients. The company's flagship product is a patch-based wearable cardioverter defibrillator that continuously monitors heart activity to protect against sudden cardiac death. This device integrates advanced data science and machine learning algorithms with strict electromechanical medical device standards, creating a proprietary digital platform that enhances patient care as individuals transition from hospital to home. Founded in 2011 and headquartered in San Francisco, California, Element Science aims to address critical health needs and reduce hospitalization rates associated with heart disease. The company was previously known as Revive Defibrillation Systems, Inc. before adopting its current name in 2014.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing novel targeted therapies for cancer treatment. Founded by Martin D. Burke, the company leverages an innovative approach to synthesize complex natural products into optimized drug candidates. This method involves rapidly assembling simple chemical building blocks into refined structures with significant therapeutic potential. Revolution Medicines focuses on inhibiting elusive targets within key growth and survival pathways, particularly RAS and mTOR signaling pathways. The company's portfolio includes therapies like RMC-4630, a SHP2 inhibitor, as well as programs targeting SOS1 and 4EBP1/mTORC1.

Thrive Earlier Detection Corp. is a healthcare company that specializes in developing advanced blood testing technologies for early cancer detection. Its flagship product, CancerSEEK, is a liquid biopsy test that analyzes genomic mutations in tumor DNA and protein markers in plasma, allowing for the identification of various cancers at their nascent stages, often before symptoms manifest. Founded in 2018 and headquartered in Cambridge, Massachusetts, with an additional office in Baltimore, Maryland, Thrive aims to integrate cancer screening into standard medical practice. The company leverages real-world data and machine learning to enhance the accuracy of cancer detection, facilitate follow-up testing, and streamline the transition to oncological care. Thrive operates as a subsidiary of Exact Sciences Corporation and has received investments from notable firms including Third Rock Ventures and Section 32.

Maze Therapeutics is a biotechnology company based in South San Francisco, California, focused on developing innovative medicines that leverage the effects of rare genetic variants to address unmet medical needs. Founded in 2017 and originally known as Modulus Therapeutics, the company utilizes its proprietary Compass platform to identify genetic variants linked to diseases and map them to the biological pathways that drive these conditions in specific patient populations. By integrating large-scale human genetics data and functional genomics with various drug discovery methods, Maze Therapeutics aims to reveal modifier genes that offer protective benefits. This approach enhances the understanding of target biology and informs the development of novel therapies, ultimately enabling pharmaceutical companies to create effective treatments.

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, founded in 2016. The company is dedicated to discovering and developing transformative therapies for severe liver diseases. Ambys focuses on creating innovative gene, drug, and cell therapies aimed at restoring liver function, halting the progression of liver disease, and addressing the complications associated with liver failure. By leveraging regenerative and restorative medical approaches, Ambys Medicines seeks to improve the lives of individuals affected by chronic liver conditions, providing potential relief through advanced therapeutic options.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

Celsius Therapeutics is a biotechnology company focused on developing precision medicines for patients with cancer and autoimmune diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company employs a team of scientists, technologists, computational biologists, clinicians, and drug hunters. Utilizing single-cell genomic analysis and machine learning techniques, Celsius Therapeutics aims to discover and create innovative therapies that can lead to significant improvements in patient outcomes. The company's research efforts are centered on identifying new drug candidates that address unmet medical needs in the therapeutic landscape.

Casma Therapeutics, Inc. specializes in developing innovative therapeutic strategies centered on the natural cellular process of autophagy and lysosomal flux. By focusing on enhancing autophagy, the company aims to improve the clearance of unwanted proteins, organelles, and pathogens, thereby addressing the progression of various diseases. Its research targets several serious medical conditions, including lysosomal storage disorders, muscle disorders, inflammatory disorders, and neurodegeneration. Founded in 2017 and based in Cambridge, Massachusetts, Casma Therapeutics is dedicated to creating effective treatment options that meet significant unmet medical needs in the field of drug discovery and development.

Insitro, Inc. is a data-driven company focused on drug discovery and development, particularly targeting nonalcoholic steatohepatitis (NASH). Founded in 2018 by Daphne Koller and based in South San Francisco, California, Insitro harnesses machine learning and high-throughput biology to revolutionize the process of drug discovery. The company creates extensive functional genomic data sets and integrates them with patient data through innovative machine learning techniques. This approach allows Insitro to build predictive models that enhance target selection, streamline the design of therapeutics, and optimize drug development strategies. By addressing critical challenges in pharmaceutical research and development, Insitro aims to accelerate the delivery of new and effective treatments to patients.

Cedilla Therapeutics, Inc. is focused on developing targeted small-molecule medicines aimed at treating cancer and other diseases resulting from protein dysregulation. The company leverages unique insights into protein stability to create innovative therapeutic approaches, including direct ligand-induced degradation, disruption of protein-protein interactions, and the identification of upstream regulators that influence protein stability. Cedilla’s technology allows for the mapping of protein susceptibility to enhance endogenous degradation pathways, facilitating the transition from stable to susceptible protein states. Founded in 2018 and headquartered in Cambridge, Massachusetts, Cedilla Therapeutics aims to broaden the applicability of its therapies beyond oncology, addressing a range of conditions associated with protein dysregulation.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing novel targeted therapies for cancer treatment. Founded by Martin D. Burke, the company leverages an innovative approach to synthesize complex natural products into optimized drug candidates. This method involves rapidly assembling simple chemical building blocks into refined structures with significant therapeutic potential. Revolution Medicines focuses on inhibiting elusive targets within key growth and survival pathways, particularly RAS and mTOR signaling pathways. The company's portfolio includes therapies like RMC-4630, a SHP2 inhibitor, as well as programs targeting SOS1 and 4EBP1/mTORC1.

Constellation Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for cancers linked to abnormal gene expression and drug resistance. The company specializes in the field of epigenetics, targeting selective regulators of epigenetic function that are vital for gene expression control. Its lead product candidates include CPI-0610, currently in Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is undergoing Phase Ib/II clinical trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is advancing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial aimed at treating solid tumors. Founded in 2008 and based in Cambridge, Massachusetts, the company was previously known as Epigenetix, Inc. before rebranding in March 2008.

Rheos Medicines, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing novel therapeutics that modulate metabolic pathways in immune cells to address immune-mediated diseases, such as inflammatory bowel disease, rheumatoid arthritis, and lupus. By targeting the cellular metabolism of specific immune cell types, Rheos aims to redirect their function and fate, thereby enhancing treatment precision. The company combines the expertise of leading scientists in immunometabolism with experienced clinicians and a seasoned leadership team to identify new drug targets and characterize disease biomarkers, ultimately striving to improve outcomes for patients suffering from autoimmune conditions.

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.

PanOptica, Inc. is a biopharmaceutical company based in Mount Arlington, New Jersey, focused on developing innovative treatments for sight-threatening eye diseases caused by abnormal or leaky blood vessels. Founded in 2009, the company is advancing its lead compound, PAN-90806, a small-molecule selective VEGF receptor antagonist, which shows potential as a topical treatment for conditions such as wet age-related macular degeneration and diabetic retinopathy. By seeking to license and develop novel therapeutics, PanOptica aims to address the limitations of existing treatments and improve patient outcomes in ophthalmology. The company serves pharmaceutical firms and is committed to enhancing the quality of life for patients experiencing vision loss due to these debilitating conditions.

Constellation Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for cancers linked to abnormal gene expression and drug resistance. The company specializes in the field of epigenetics, targeting selective regulators of epigenetic function that are vital for gene expression control. Its lead product candidates include CPI-0610, currently in Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is undergoing Phase Ib/II clinical trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is advancing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial aimed at treating solid tumors. Founded in 2008 and based in Cambridge, Massachusetts, the company was previously known as Epigenetix, Inc. before rebranding in March 2008.

Magenta Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapies to enhance stem cell transplantation for patients with blood cancers, genetic diseases, and autoimmune disorders. The company aims to improve the safety and efficacy of stem cell transplants, which are currently high-risk procedures often considered only as a last resort. Its pipeline includes several key product candidates, such as targeted antibody-drug conjugates for transplant conditioning, a novel stem cell mobilization agent, and allogeneic stem cell therapies. Magenta is also exploring collaborations to assess the effectiveness of its therapies in combination with existing treatments, such as gene therapies for sickle cell disease. Founded in 2015 and headquartered in Cambridge, Massachusetts, Magenta Therapeutics seeks to expand the curative potential of stem cell therapies to a broader patient population by addressing the toxicity and long-term side effects associated with current transplant methods.

Tango Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cancer therapies through the principle of synthetic lethality. Established in 2014, the company aims to discover and target specific driver genes involved in cancer progression. Its research concentrates on three main areas: counteracting the loss of tumor suppressor genes, reversing cancer cells' ability to evade the immune system, and identifying effective drug combinations that outperform single-agent therapies. Tango's pipeline includes TNG908, a selective inhibitor of PRMT5 designed for cancer cells with an MTAP deletion, alongside other discovery programs like TNG462, TNG348, and TNG260, which target various cancer types with limited treatment options.

Rhythm Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development and commercialization of therapeutics for rare genetic disorders, particularly those that lead to life-threatening metabolic conditions. The company’s primary product candidate, setmelanotide, is a first-in-class melanocortin-4 receptor agonist currently undergoing Phase III clinical trials for the treatment of obesity related to pro-opiomelanocortin (POMC) and leptin receptor deficiencies, as well as Bardet-Biedl and Alström syndromes. Additionally, it is in Phase II trials for various other genetic obesity disorders. Rhythm Pharmaceuticals is also advancing RM-853, an orally available ghrelin o-acyltransferase inhibitor in preclinical development aimed at addressing Prader-Willi syndrome. Founded in 2008 and based in Boston, Massachusetts, the company previously operated under the name Rhythm Metabolic, Inc. and rebranded in October 2015.

Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at advancing cancer treatment. By focusing on the immune system's response to neoantigens, the company seeks to develop innovative therapies that enhance the body's ability to fight cancer. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to leverage the unique characteristics of neoantigens to improve patient outcomes.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing novel targeted therapies for cancer treatment. Founded by Martin D. Burke, the company leverages an innovative approach to synthesize complex natural products into optimized drug candidates. This method involves rapidly assembling simple chemical building blocks into refined structures with significant therapeutic potential. Revolution Medicines focuses on inhibiting elusive targets within key growth and survival pathways, particularly RAS and mTOR signaling pathways. The company's portfolio includes therapies like RMC-4630, a SHP2 inhibitor, as well as programs targeting SOS1 and 4EBP1/mTORC1.

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 is a clinical-stage biotechnology company focused on developing innovative therapies to enhance stem cell transplantation for patients with blood cancers, genetic diseases, and autoimmune disorders. The company aims to improve the safety and efficacy of stem cell transplants, which are currently high-risk procedures often considered only as a last resort. Its pipeline includes several key product candidates, such as targeted antibody-drug conjugates for transplant conditioning, a novel stem cell mobilization agent, and allogeneic stem cell therapies. Magenta is also exploring collaborations to assess the effectiveness of its therapies in combination with existing treatments, such as gene therapies for sickle cell disease. Founded in 2015 and headquartered in Cambridge, Massachusetts, Magenta Therapeutics seeks to expand the curative potential of stem cell therapies to a broader patient population by addressing the toxicity and long-term side effects associated with current transplant methods.

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.

Element Science, Inc. is a medical device and digital health company focused on developing solutions for high-risk cardiovascular patients. The company's flagship product is a patch-based wearable cardioverter defibrillator that continuously monitors heart activity to protect against sudden cardiac death. This device integrates advanced data science and machine learning algorithms with strict electromechanical medical device standards, creating a proprietary digital platform that enhances patient care as individuals transition from hospital to home. Founded in 2011 and headquartered in San Francisco, California, Element Science aims to address critical health needs and reduce hospitalization rates associated with heart disease. The company was previously known as Revive Defibrillation Systems, Inc. before adopting its current name in 2014.

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.

PanOptica, Inc. is a biopharmaceutical company based in Mount Arlington, New Jersey, focused on developing innovative treatments for sight-threatening eye diseases caused by abnormal or leaky blood vessels. Founded in 2009, the company is advancing its lead compound, PAN-90806, a small-molecule selective VEGF receptor antagonist, which shows potential as a topical treatment for conditions such as wet age-related macular degeneration and diabetic retinopathy. By seeking to license and develop novel therapeutics, PanOptica aims to address the limitations of existing treatments and improve patient outcomes in ophthalmology. The company serves pharmaceutical firms and is committed to enhancing the quality of life for patients experiencing vision loss due to these debilitating conditions.

Pliant Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, specializing in the discovery and development of therapies for fibrotic diseases. The company's lead candidate, PLN-74809, is an oral small-molecule dual selective inhibitor of avß6 and avß1 integrins, currently being developed for idiopathic pulmonary fibrosis and primary sclerosing cholangitis. PLN-74809 has received Orphan Drug Designation from the U.S. Food and Drug Administration for both conditions and is undergoing Phase 2a trials for IPF, with plans for a Phase 2a trial in PSC. Pliant is also advancing PLN-1474, a small-molecule selective inhibitor of avß1, aimed at treating liver fibrosis associated with nonalcoholic steatohepatitis, which is in Phase 1 clinical trials and has a partnership with Novartis. In addition to these clinical efforts, Pliant has two preclinical programs targeting oncology and muscular dystrophies. Founded in 2015, Pliant Therapeutics is committed to addressing unmet medical needs in the field of fibrosis.

Constellation Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for cancers linked to abnormal gene expression and drug resistance. The company specializes in the field of epigenetics, targeting selective regulators of epigenetic function that are vital for gene expression control. Its lead product candidates include CPI-0610, currently in Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is undergoing Phase Ib/II clinical trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is advancing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial aimed at treating solid tumors. Founded in 2008 and based in Cambridge, Massachusetts, the company was previously known as Epigenetix, Inc. before rebranding in March 2008.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at advancing cancer treatment. By focusing on the immune system's response to neoantigens, the company seeks to develop innovative therapies that enhance the body's ability to fight cancer. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to leverage the unique characteristics of neoantigens to improve patient outcomes.

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.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing novel targeted therapies for cancer treatment. Founded by Martin D. Burke, the company leverages an innovative approach to synthesize complex natural products into optimized drug candidates. This method involves rapidly assembling simple chemical building blocks into refined structures with significant therapeutic potential. Revolution Medicines focuses on inhibiting elusive targets within key growth and survival pathways, particularly RAS and mTOR signaling pathways. The company's portfolio includes therapies like RMC-4630, a SHP2 inhibitor, as well as programs targeting SOS1 and 4EBP1/mTORC1.

CytomX Therapeutics, Inc. is an oncology-focused biopharmaceutical company based in South San Francisco, California. It specializes in developing a novel class of antibody therapeutics utilizing its Probody technology platform, which aims to enhance the targeting of tumors while minimizing drug activity in healthy tissues. The company's clinical-stage product candidates include CX-072, a therapeutic targeting programmed cell death ligand 1; CX-2009, a drug conjugate targeting CD166; BMS-986249 and BMS-986288, both targeting CTLA-4 for metastatic melanoma and solid tumors; and CX-2029, a drug conjugate for CD71 in solid tumors. CytomX collaborates strategically with several industry leaders, including AbbVie, Amgen, and Bristol-Myers Squibb, to advance its Probody therapeutics. Founded in 2008, CytomX Therapeutics is committed to providing innovative and less toxic treatment options for patients with severe illnesses such as cancer.

Allena Pharmaceuticals is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, that specializes in developing and commercializing oral enzyme therapeutics for rare and severe metabolic and kidney disorders. The company's primary focus is on conditions that involve the excessive accumulation of metabolites such as oxalate and urate, which can lead to complications like kidney stones and chronic kidney disease. Allena's lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a disorder linked to significantly high urinary oxalate levels. Additionally, the company is developing ALLN-346, a novel enzyme designed to address hyperuricemia in patients with moderate to severe chronic kidney diseases. Allena Pharmaceuticals aims to provide innovative treatments that address unmet medical needs in these patient populations.

Blueprint Medicines Corporation is a biopharmaceutical company that specializes in developing small molecule kinase inhibitors aimed at treating genomically defined cancers and rare diseases. Founded in 2008 and headquartered in Cambridge, Massachusetts, the company focuses on personalized cancer therapies by leveraging its proprietary chemical library and advanced research capabilities. Its key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, and pralsetinib for RET-altered non-small cell lung cancer and medullary thyroid carcinoma. Additionally, Blueprint Medicines is advancing BLU-263, a KIT inhibitor for mast cell disorders, and fisogatinib for advanced hepatocellular carcinoma, both currently in clinical trials. The company’s innovative approach seeks to address the molecular drivers of cancer and resistance mechanisms, aiming to enhance treatment effectiveness and patient outcomes. Blueprint Medicines has established collaborations with several pharmaceutical companies to further its research and development efforts.

MyoKardia, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing targeted therapies for rare cardiovascular diseases. Founded in 2012 and headquartered in Brisbane, California, the company primarily addresses heritable cardiomyopathies and genetically-driven heart failure caused by biomechanical defects in cardiac muscle contraction. Its lead product candidate, Mavacamten, is an orally administered allosteric modulator of cardiac myosin currently in Phase III trials for hypertrophic cardiomyopathy (HCM). Additionally, MyoKardia is developing Danicamtiv, an orally administered small molecule that has completed Phase I and IIa trials for dilated cardiomyopathy. The company also has several preclinical programs, including MYK-224 for HCM, ACT-1 for diastolic dysfunction, and LUS-1 for diastolic dysfunction. As of late 2020, MyoKardia operates as a subsidiary of Bristol-Myers Squibb Company.

Corvia Medical is a medical device company based in Andover, Massachusetts, established in 2009. The company specializes in developing innovative solutions for heart failure, particularly through its InterAtrial Shunt Device (IASD). This transcatheter device is designed to address diastolic heart failure, also known as heart failure with preserved ejection fraction. The IASD works by creating a small opening between the left and right atria, which helps to lower blood pressure in the left atrium and lungs. This reduction in pressure aims to alleviate symptoms and slow the progression of heart failure, allowing patients to maintain a healthier lifestyle and reduce their risk of severe heart-related complications.

Nurix Therapeutics is a biopharmaceutical company specializing in the discovery, development, and commercialization of small molecule therapies aimed at modulating cellular protein levels to treat cancer and immune disorders. The company's pipeline includes promising preclinical candidates such as NX-2127, an orally available Bruton’s tyrosine kinase degrader targeted at relapsed or refractory B-cell malignancies, and NX-1607, a Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology applications. Nurix's innovative drug discovery platform, DELigase, leverages its expertise in E3 ligases—enzymes that regulate protein modulation within cells—to identify and advance novel drug candidates. Founded in 2009 and based in San Francisco, Nurix Therapeutics aims to provide effective therapies for challenging diseases through its cutting-edge research and development efforts.

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.

PanOptica, Inc. is a biopharmaceutical company based in Mount Arlington, New Jersey, focused on developing innovative treatments for sight-threatening eye diseases caused by abnormal or leaky blood vessels. Founded in 2009, the company is advancing its lead compound, PAN-90806, a small-molecule selective VEGF receptor antagonist, which shows potential as a topical treatment for conditions such as wet age-related macular degeneration and diabetic retinopathy. By seeking to license and develop novel therapeutics, PanOptica aims to address the limitations of existing treatments and improve patient outcomes in ophthalmology. The company serves pharmaceutical firms and is committed to enhancing the quality of life for patients experiencing vision loss due to these debilitating conditions.

Element Science, Inc. is a medical device and digital health company focused on developing solutions for high-risk cardiovascular patients. The company's flagship product is a patch-based wearable cardioverter defibrillator that continuously monitors heart activity to protect against sudden cardiac death. This device integrates advanced data science and machine learning algorithms with strict electromechanical medical device standards, creating a proprietary digital platform that enhances patient care as individuals transition from hospital to home. Founded in 2011 and headquartered in San Francisco, California, Element Science aims to address critical health needs and reduce hospitalization rates associated with heart disease. The company was previously known as Revive Defibrillation Systems, Inc. before adopting its current name in 2014.

NinePoint Medical, Inc. is a medical device company based in Bedford, Massachusetts, that specializes in optical coherence tomography (OCT) imaging systems for assessing human tissue microstructure. The company's flagship product, the NvisionVLE Imaging System, provides real-time volumetric images of the esophagus and bile duct, enabling healthcare professionals across various specialties, including gastroenterology and pulmonology, to conduct advanced imaging procedures. This technology allows for high-resolution, cross-sectional views that facilitate accurate diagnoses and effective therapies during medical procedures. NinePoint aims to enhance the efficiency of patient care by integrating access to imaging, diagnosis, and treatment in a single procedure, ultimately improving patient outcomes and reducing healthcare costs. The company markets its products through distributors both domestically and internationally. Established in 2008, NinePoint Medical was formerly known as Convergence Devices, Inc. and underwent a name change in 2009.

SAGE Therapeutics is a biopharmaceutical company focused on developing innovative treatments for central nervous system (CNS) disorders. The company's lead product is ZULRESSO, an intravenous formulation of brexanolone designed for treating postpartum depression. SAGE's pipeline includes several advanced candidates, such as SAGE-217, currently in Phase III trials for various depressive disorders and anxiety, and SAGE-324, which is in Phase II trials for essential tremors. Additionally, SAGE-718 is being explored for multiple conditions, including depression and Alzheimer's disease, having completed its Phase I trials. The company has established strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. to enhance the development and commercialization of its therapies. Founded in 2010 and headquartered in Cambridge, Massachusetts, SAGE Therapeutics aims to address significant unmet needs in brain health through its scientific innovations and partnerships.

Voyager Therapeutics, Inc. is a clinical-stage gene therapy company dedicated to developing innovative treatments for severe neurological diseases that significantly impact patients' lives. The company's lead candidate, VY-AADC, is currently undergoing an open-label Phase 1b clinical trial for Parkinson's disease. In addition to this, Voyager's preclinical pipeline includes therapies targeting amyotrophic lateral sclerosis (VY-SOD102), Huntington's disease (VY-HTT01), Friedreich's ataxia (VY-FXN01), and various tauopathies and synucleinopathies, such as Alzheimer's disease and Lewy Body Dementia. Voyager employs an adeno-associated virus (AAV) gene therapy approach, which is designed to modify protein production to alleviate disease symptoms, thereby aiming for clinically meaningful outcomes. The company has established strategic collaborations with institutions like the University of Massachusetts and ClearPoint Neuro, as well as partnerships with Brammer Bio and Fujifilm Diosynth Biotechnologies to advance its gene therapy programs. Founded in 2013 and based in Cambridge, Massachusetts, Voyager Therapeutics is committed to addressing unmet medical needs within the central nervous system.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, dedicated to the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, Igenica specializes in immunotherapies and functional antibodies designed to target tumors effectively. The company's notable products include IGN523, a humanized monoclonal antibody aimed at treating various hematologic and solid tumors; IGN786, an ADC; and IGN381, which targets immune checkpoints that complement existing therapies in the tumor microenvironment. Igenica has established a strategic oncology research agreement with MedImmune and is supported by a group of prominent life science investors, including The Column Group and OrbiMed. The company operates as a subsidiary of Pierre Fabre Medicament SA, with a leadership team that has significant experience in antibody drug discovery and commercialization.

Blueprint Medicines Corporation is a biopharmaceutical company that specializes in developing small molecule kinase inhibitors aimed at treating genomically defined cancers and rare diseases. Founded in 2008 and headquartered in Cambridge, Massachusetts, the company focuses on personalized cancer therapies by leveraging its proprietary chemical library and advanced research capabilities. Its key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, and pralsetinib for RET-altered non-small cell lung cancer and medullary thyroid carcinoma. Additionally, Blueprint Medicines is advancing BLU-263, a KIT inhibitor for mast cell disorders, and fisogatinib for advanced hepatocellular carcinoma, both currently in clinical trials. The company’s innovative approach seeks to address the molecular drivers of cancer and resistance mechanisms, aiming to enhance treatment effectiveness and patient outcomes. Blueprint Medicines has established collaborations with several pharmaceutical companies to further its research and development efforts.

Soffio Medical is a Boston-based company that develops medical devices aimed at treating respiratory diseases. The company's innovative approach focuses on implantable devices designed to implement an airway bypass concept. This technology allows for the venting of trapped air from areas distal to high-resistance airways, which helps to reduce residual lung volume. By alleviating symptoms associated with chronic obstructive pulmonary disease, Soffio Medical's solutions aim to enhance the quality of life for patients suffering from these conditions.

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 is a biopharmaceutical company focused on developing innovative treatments for central nervous system (CNS) disorders. The company's lead product is ZULRESSO, an intravenous formulation of brexanolone designed for treating postpartum depression. SAGE's pipeline includes several advanced candidates, such as SAGE-217, currently in Phase III trials for various depressive disorders and anxiety, and SAGE-324, which is in Phase II trials for essential tremors. Additionally, SAGE-718 is being explored for multiple conditions, including depression and Alzheimer's disease, having completed its Phase I trials. The company has established strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. to enhance the development and commercialization of its therapies. Founded in 2010 and headquartered in Cambridge, Massachusetts, SAGE Therapeutics aims to address significant unmet needs in brain health through its scientific innovations and partnerships.

Edimer Pharmaceuticals focuses on developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder characterized by symptoms such as insufficient sweat glands, impaired temperature regulation, respiratory difficulties, and abnormal hair and tooth development. The company aims to enhance the health and quality of life for individuals affected by this orphan disease. Edimer is supported by a team of experienced biotechnology professionals and a network of esteemed clinical and scientific advisors, all committed to advancing drug development for XLHED. Through its innovative approach, Edimer seeks to provide significant and lasting improvements in the management of this condition for future generations.

Alcresta Therapeutics, Inc. is focused on developing and commercializing innovative enzyme-based products aimed at treating gastrointestinal disorders and rare diseases. The company's flagship product, RELiZORB, is a digestive enzyme cartridge that mimics pancreatic lipase, facilitating fat absorption in adults receiving enteral tube feeding. Founded in 2011 and headquartered in Warren, New Jersey, Alcresta addresses the nutritional needs of infants, aging adults, and individuals facing acute or chronic health challenges. The company leverages its expertise in adult and pediatric nutrition, protein manufacturing, and regulatory matters to support the development and commercialization of its products, ensuring they meet the needs of those with compromised health. Alcresta's research-driven approach aims to improve nutrition and effectively manage disease in its target populations.

Afferent Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, founded in 2009. The company focuses on developing small molecule drugs aimed at treating chronic pain and various neurogenic disorders by targeting P2X3 receptors in nerve fibers. Its lead compound, AF-219, has completed two Phase 1 clinical studies and is designed to address conditions such as osteoarthritis, back pain, visceral pain, and neuropathy, as well as respiratory and urological issues.

Nurix Therapeutics is a biopharmaceutical company specializing in the discovery, development, and commercialization of small molecule therapies aimed at modulating cellular protein levels to treat cancer and immune disorders. The company's pipeline includes promising preclinical candidates such as NX-2127, an orally available Bruton’s tyrosine kinase degrader targeted at relapsed or refractory B-cell malignancies, and NX-1607, a Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology applications. Nurix's innovative drug discovery platform, DELigase, leverages its expertise in E3 ligases—enzymes that regulate protein modulation within cells—to identify and advance novel drug candidates. Founded in 2009 and based in San Francisco, Nurix Therapeutics aims to provide effective therapies for challenging diseases through its cutting-edge research and development efforts.

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's unique drug delivery system allows for the continuous release of medications within the bladder over extended periods, ranging from weeks to months. This system is designed to be deployed and retrieved through minimally invasive, in-office procedures, enabling a tailored approach to drug release that meets the specific needs of various bladder conditions. As of December 2019, TARIS Biomedical operates as a subsidiary of Janssen Research & Development, LLC.

Corvia Medical is a medical device company based in Andover, Massachusetts, established in 2009. The company specializes in developing innovative solutions for heart failure, particularly through its InterAtrial Shunt Device (IASD). This transcatheter device is designed to address diastolic heart failure, also known as heart failure with preserved ejection fraction. The IASD works by creating a small opening between the left and right atria, which helps to lower blood pressure in the left atrium and lungs. This reduction in pressure aims to alleviate symptoms and slow the progression of heart failure, allowing patients to maintain a healthier lifestyle and reduce their risk of severe heart-related complications.

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 is a clinical-stage immunotherapy company focused on developing innovative therapies for cancer treatment. Headquartered in Cambridge, Massachusetts, the company is advancing several product candidates, including vopratelimab, a monoclonal antibody currently in Phase II clinical trials for patients with non-small cell lung cancer and urothelial cancer. Jounce is also working on JTX-4014, an anti-PD-1 antibody intended for combination therapy, and JTX-1811, which aims to selectively deplete T regulatory cells in the tumor microenvironment. Additionally, the company is developing JTX-8064, an antibody targeting a receptor expressed on macrophages. Founded in 2013 by experts in immunobiology and cancer biology, Jounce Therapeutics employs a proprietary product engine and a Translational Science Platform to enhance its understanding of tumor composition and improve patient outcomes.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Motus Therapeutics is a biotechnology company based in Boston, Massachusetts, focused on developing peptide therapeutics to address unmet medical needs in metabolic diseases, particularly gastrointestinal disorders. The company's lead product candidate, relamorelin, is a ghrelin agonist designed to treat diabetic gastroparesis, a complication associated with diabetes, as well as other gastrointestinal functional disorders. Motus Therapeutics aims to provide innovative solutions for patients suffering from these conditions.

Rhythm Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development and commercialization of therapeutics for rare genetic disorders, particularly those that lead to life-threatening metabolic conditions. The company’s primary product candidate, setmelanotide, is a first-in-class melanocortin-4 receptor agonist currently undergoing Phase III clinical trials for the treatment of obesity related to pro-opiomelanocortin (POMC) and leptin receptor deficiencies, as well as Bardet-Biedl and Alström syndromes. Additionally, it is in Phase II trials for various other genetic obesity disorders. Rhythm Pharmaceuticals is also advancing RM-853, an orally available ghrelin o-acyltransferase inhibitor in preclinical development aimed at addressing Prader-Willi syndrome. Founded in 2008 and based in Boston, Massachusetts, the company previously operated under the name Rhythm Metabolic, Inc. and rebranded in October 2015.

Cibiem Inc. is a medical device company that specializes in the development of minimally invasive, catheter-based technologies for treating diseases mediated by the sympathetic nervous system, including hypertension, heart failure, diabetes, and renal failure. The company focuses on Carotid Body Modulation (CBM), which offers a novel approach to managing these serious health conditions. Founded in 2010 and based in Los Altos, California, with an additional office in Teaneck, New Jersey, Cibiem was co-founded by Mark Gelfand and Howard R. Levin, M.D. The company is supported by prominent venture investors, including Third Rock Ventures and SV Life Sciences, reflecting its innovative potential in the medical device sector.