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.

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 leveraging the power of lysine-based covalency to develop optimized medicines.

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 leveraging the power of lysine-based covalency to develop optimized medicines.

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.

Cedilla is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation. The company was founded in 2018 and based in Cambridge, Massachusetts.

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 developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.

Cedilla is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation. The company was founded in 2018 and based in Cambridge, Massachusetts.

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 is a medical device and digital health company that develops solutions at the intersection of clinical-grade wearable devices, machine learning algorithms, and lifesaving therapies to address the needs of high-risk cardiovascular patients, primarily as they transition from the hospital to home. The company developing a proprietary next-generation wearable digital platform that unites patient-centric human factor engineering, sophisticated machine learning algorithm development principles, and rigorous electromechanical medical device development standards. The company was founded in 2011 and is headquartered in San Francisco, California.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing innovative therapies targeting challenging cancer pathways, particularly the RAS and mTOR signaling pathways. The company utilizes a unique approach that reconfigures natural substances into optimized drug candidates, building on the pioneering work of its founder, Martin D. Burke. Revolution Medicines has established an exclusive licensing agreement with the University of Illinois to utilize and further develop this transformative synthetic technology. Among its key product candidates are RMC-4630, a SHP2 inhibitor, and various programs within its RAS(ON) portfolio, along with SOS1 and 4EBP1/mTORC1 initiatives. The company's focus on harnessing complex natural products to create refined, effective therapies positions it to address significant unmet medical needs in oncology.

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 developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.

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 is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation. The company was founded in 2018 and based in Cambridge, Massachusetts.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing innovative therapies targeting challenging cancer pathways, particularly the RAS and mTOR signaling pathways. The company utilizes a unique approach that reconfigures natural substances into optimized drug candidates, building on the pioneering work of its founder, Martin D. Burke. Revolution Medicines has established an exclusive licensing agreement with the University of Illinois to utilize and further develop this transformative synthetic technology. Among its key product candidates are RMC-4630, a SHP2 inhibitor, and various programs within its RAS(ON) portfolio, along with SOS1 and 4EBP1/mTORC1 initiatives. The company's focus on harnessing complex natural products to create refined, effective therapies positions it to address significant unmet medical needs in oncology.

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 is a clinical-stage precision medicine company based in Cambridge, Massachusetts, focused on transforming the drug discovery process, particularly in targeted oncology. Utilizing its innovative Dynamo platform, the company combines advanced computational techniques with experimental methods to enhance small molecule therapeutic discovery. Relay Therapeutics is developing several product candidates, including RLY-1971, an oral inhibitor targeting the SHP2 protein, currently in Phase 1 trials for advanced solid tumors; RLY-4008, a selective inhibitor for patients with FGFR2 alterations; and RLY-PI3K1047, aimed at cancer-associated mutations of the PI3K alpha protein. Founded in 2015, Relay Therapeutics is dedicated to addressing complex diseases through its unique approach to drug discovery.

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.

Rhythm is a biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic disorders. Their lead peptide product candidate, setmelanotide, is a potent, first-in-class melanocortin-4 receptor (MC4R) agonist for the treatment of rare genetic disorders of obesity. They believe that setmelanotide, for which they have exclusive worldwide rights, has the potential to serve as replacement therapy for the treatment of melanocortin 4, or MC4, pathway deficiencies. The MC4 pathway is a compelling target for treating these genetic disorders because of its critical role in regulating appetite and weight, and peptide therapeutics are uniquely suited to activating this target.

Neon Therapeutics is a clinical-stage biopharmaceutical company. It is focused in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. The company's pipeline products are NEO-PV-01, NEO-PTC-01, and NEO-SV-01.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing innovative therapies targeting challenging cancer pathways, particularly the RAS and mTOR signaling pathways. The company utilizes a unique approach that reconfigures natural substances into optimized drug candidates, building on the pioneering work of its founder, Martin D. Burke. Revolution Medicines has established an exclusive licensing agreement with the University of Illinois to utilize and further develop this transformative synthetic technology. Among its key product candidates are RMC-4630, a SHP2 inhibitor, and various programs within its RAS(ON) portfolio, along with SOS1 and 4EBP1/mTORC1 initiatives. The company's focus on harnessing complex natural products to create refined, effective therapies positions it to address significant unmet medical needs in oncology.

Goldfinch is a clinical-stage biotech company focused on discovering and developing precision therapies for patients with kidney disease. It is integrating breakthroughs in kidney genetics and biology to identify new therapeutic targets and advance first-in-class drug candidates to treat patients with kidney disease. Its Product Engine will industrialize the integration of genetics and kidney biology and confer a differentiated ability to identify, validate, and pursue novel therapeutic targets to treat progressive kidney disease. The company was founded in 2016 and is based in Cambridge, Massachusetts.

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 is a clinical-stage precision medicine company based in Cambridge, Massachusetts, focused on transforming the drug discovery process, particularly in targeted oncology. Utilizing its innovative Dynamo platform, the company combines advanced computational techniques with experimental methods to enhance small molecule therapeutic discovery. Relay Therapeutics is developing several product candidates, including RLY-1971, an oral inhibitor targeting the SHP2 protein, currently in Phase 1 trials for advanced solid tumors; RLY-4008, a selective inhibitor for patients with FGFR2 alterations; and RLY-PI3K1047, aimed at cancer-associated mutations of the PI3K alpha protein. Founded in 2015, Relay Therapeutics is dedicated to addressing complex diseases through its unique approach to drug discovery.

Element Science is a medical device and digital health company that develops solutions at the intersection of clinical-grade wearable devices, machine learning algorithms, and lifesaving therapies to address the needs of high-risk cardiovascular patients, primarily as they transition from the hospital to home. The company developing a proprietary next-generation wearable digital platform that unites patient-centric human factor engineering, sophisticated machine learning algorithm development principles, and rigorous electromechanical medical device development standards. The company was founded in 2011 and is headquartered in San Francisco, California.

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapies for patients with rare genetic diseases. The company is dedicated to addressing unmet medical needs, particularly in the realm of genetically defined conditions. Its lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle-wasting disorder. Additionally, Fulcrum is developing FTX-6058, aimed at increasing fetal hemoglobin levels in patients with sickle cell disease and beta-thalassemia. The company also engages in research to identify drug targets for various conditions, including Duchenne muscular dystrophy and neurological diseases. Fulcrum Therapeutics has established collaborations with Acceleron Pharma Inc. and MyoKardia, Inc. to explore new therapeutic avenues in pulmonary disease and genetic cardiomyopathies, respectively. Founded in 2015, Fulcrum aims to enhance the quality of life for patients suffering from these challenging disorders.

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 is a clinical stage biopharmaceutical company focused on discovering and developing novel therapies for the treatment of fibrosis. Pliant's lead product candidate, PLN-74809, is an oral small-molecule dual selective inhibitor of avß6 and avß1 integrins that it is developing for the treatment of idiopathic pulmonary fibrosis, or IPF, and primary sclerosing cholangitis, or PSC. PLN-74809 has received Orphan Drug Designation from the U.S. Food and Drug Administration for both IPF and PSC. Pliant is currently recruiting two Phase 2a trials of PLN-74809 for the treatment of IPF and plans to initiate a Phase 2a trial in PSC in the second half of 2020. Pliant's second product candidate, PLN-1474, is a small-molecule selective inhibitor of avß1 for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis, or NASH, which Pliant has partnered with Novartis. PLN-1474 is currently undergoing a Phase 1 trial. In addition to clinical stage programs, Pliant currently has two preclinical programs targeting oncology and muscular dystrophies.

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 developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.

Neon Therapeutics is a clinical-stage biopharmaceutical company. It is focused in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. The company's pipeline products are NEO-PV-01, NEO-PTC-01, and NEO-SV-01.

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, it is focused on discovering and developing innovative genome editing therapeutics to address a wide range of serious diseases. The company utilizes a proprietary platform based on CRISPR/Cas9 technology, which employs a protein-RNA complex to target and modify specific DNA sequences for therapeutic purposes. Editas Medicine aims to translate its advanced genome editing technologies into human therapeutics that can provide precise and corrective molecular modifications to treat genetic disorders. With a strong foundation of patent filings and intellectual property, the company is positioned to advance its early findings into viable treatments for various diseases at the genetic level.

Revolution Medicines is a clinical-stage precision oncology company dedicated to developing innovative therapies targeting challenging cancer pathways, particularly the RAS and mTOR signaling pathways. The company utilizes a unique approach that reconfigures natural substances into optimized drug candidates, building on the pioneering work of its founder, Martin D. Burke. Revolution Medicines has established an exclusive licensing agreement with the University of Illinois to utilize and further develop this transformative synthetic technology. Among its key product candidates are RMC-4630, a SHP2 inhibitor, and various programs within its RAS(ON) portfolio, along with SOS1 and 4EBP1/mTORC1 initiatives. The company's focus on harnessing complex natural products to create refined, effective therapies positions it to address significant unmet medical needs in oncology.

CytomX Therapeutics is an oncology-focused biopharmaceutical company based in South San Francisco, California. The company specializes in developing proteolytically-activated antibody therapeutics using its proprietary Probody technology platform. This innovative approach aims to create cancer immunotherapies that enhance targeting of tumor cells while minimizing effects on healthy tissues. CytomX's clinical-stage product candidates include CX-072, which targets programmed cell death ligand 1; CX-2009, a drug conjugate against CD166; and BMS-986249, a CTLA-4 Probody therapeutic for metastatic melanoma, among others. The company has established strategic collaborations with several major pharmaceutical companies, including AbbVie, Amgen, and Bristol-Myers Squibb, to further the development of its Probody therapeutics. Founded in 2008, CytomX is dedicated to providing patients with more effective and less toxic treatment options for cancer and inflammatory diseases.

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 is a biopharmaceutical company dedicated to developing targeted therapies for cancers and rare diseases driven by abnormal kinase activation. Founded in 2008 and based in Cambridge, Massachusetts, the company utilizes its proprietary Insights-to-Validation Platform and chemical library to create small molecule kinase inhibitors that address specific genomic drivers. Key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis, and pralsetinib for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. Additionally, fisogatinib is under investigation for advanced hepatocellular carcinoma, while BLU-782 targets fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with several pharmaceutical companies to enhance its research and development efforts.

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 headquartered in Watertown, Massachusetts, that specializes in developing therapies for diseases affecting mucosal tissues using its proprietary nanoparticle-based Mucus Penetrating Particles (MPP) technology. This innovative approach enhances drug distribution and pharmacokinetics by delivering biocompatible, drug-loaded particles that uniformly coat mucosal surfaces. The company's lead product candidate, KPI-121 0.25%, has successfully completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS, another product, has also completed two Phase III trials for treating inflammation and pain following ocular surgery. Kala is advancing its pipeline, including KPI-285, a receptor tyrosine kinase inhibitor for retinal diseases, which is currently in preclinical studies. The company aims to address significant clinical needs while exploring research collaborations to enhance its product offerings.

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 is a medical device and digital health company that develops solutions at the intersection of clinical-grade wearable devices, machine learning algorithms, and lifesaving therapies to address the needs of high-risk cardiovascular patients, primarily as they transition from the hospital to home. The company developing a proprietary next-generation wearable digital platform that unites patient-centric human factor engineering, sophisticated machine learning algorithm development principles, and rigorous electromechanical medical device development standards. The company was founded in 2011 and is headquartered in San Francisco, California.

NinePoint Medical, Inc. is a transformational medical device company developing innovative, real-time, in vivo pathology devices. The company's first goal is to develop a device that enables remote pathology, or telepathology, which would facilitate immediate review of images during diagnostic procedures. This capability could potentially provide physicians with immediately actionable information and eventually allow them to pursue therapeutic remedies at the time of diagnosis. This convergence of access, diagnosis and treatment during one procedure is expected to improve patient experiences and outcomes, improve the efficiency of care and provide important savings to the healthcare system. Headquartered in Cambridge, Mass., NinePoint is backed by Third Rock Ventures and Prospect Venture Partners.

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 is a biopharmaceutical company dedicated to developing targeted therapies for cancers and rare diseases driven by abnormal kinase activation. Founded in 2008 and based in Cambridge, Massachusetts, the company utilizes its proprietary Insights-to-Validation Platform and chemical library to create small molecule kinase inhibitors that address specific genomic drivers. Key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis, and pralsetinib for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. Additionally, fisogatinib is under investigation for advanced hepatocellular carcinoma, while BLU-782 targets fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with several pharmaceutical companies to enhance 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, it is focused on discovering and developing innovative genome editing therapeutics to address a wide range of serious diseases. The company utilizes a proprietary platform based on CRISPR/Cas9 technology, which employs a protein-RNA complex to target and modify specific DNA sequences for therapeutic purposes. Editas Medicine aims to translate its advanced genome editing technologies into human therapeutics that can provide precise and corrective molecular modifications to treat genetic disorders. With a strong foundation of patent filings and intellectual property, the company is positioned to advance its early findings into viable treatments for various diseases at the genetic level.

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.

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.

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.

Rhythm is a biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic disorders. Their lead peptide product candidate, setmelanotide, is a potent, first-in-class melanocortin-4 receptor (MC4R) agonist for the treatment of rare genetic disorders of obesity. They believe that setmelanotide, for which they have exclusive worldwide rights, has the potential to serve as replacement therapy for the treatment of melanocortin 4, or MC4, pathway deficiencies. The MC4 pathway is a compelling target for treating these genetic disorders because of its critical role in regulating appetite and weight, and peptide therapeutics are uniquely suited to activating this target.

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.