Third Rock Ventures

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D, for cerebral adrenoleukodystrophy. In oncology, Bluebird Bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.

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.

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.

Warp Drive Bio, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on developing genomic technology to discover drugs of natural origin. Founded in 2010, the company utilizes a proprietary genomic search engine and customized search queries to uncover hidden natural products by analyzing their unique genomic signatures. This innovative approach allows researchers to generate novel drug candidates and understand their mechanisms of action, facilitating the conversion of genomic data into potential therapeutic drugs. As of October 2018, Warp Drive Bio operates as a subsidiary of REVOLUTION Medicines, Inc.

Agios Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of medicines targeting cellular metabolism and related areas, including hematologic malignancies, solid tumors, and rare genetic diseases. The company offers TIBSOVO, an oral targeted inhibitor for treating relapsed or refractory acute myeloid leukemia (AML) in adult patients, and IDHIFA, which is also aimed at AML patients with specific mutations. Agios is advancing several drug candidates through various stages of clinical trials, including TIBSOVO for frontline AML and cholangiocarcinoma, mitapivat for pyruvate kinase deficiency and thalassemia, and vorasidenib for solid tumors. The company emphasizes a precision medicine approach, focusing on genetically or biomarker-defined patient populations to enhance the potential for early proof of concept and accelerated approval. Agios Pharmaceuticals was established in 2007 and has evolved from its original name, Cancer Metabolism Therapeutics, to its current identity.

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.

Foundation Medicine, Inc. specializes in providing molecular information products that enhance cancer treatment by utilizing advanced genomic profiling techniques. The company's platform analyzes various cancer specimens to deliver personalized genomic insights, which assist physicians in optimizing treatment plans and support biopharmaceutical companies in developing targeted therapies and immunotherapies. Key clinical products include FoundationOne, which focuses on solid tumors, and FoundationOne Heme for blood-based cancers. Additionally, Foundation Medicine offers diagnostic assays such as Foundation Assay for Circulating Tumor and FoundationFocus CDxBRCA, aimed at identifying ovarian cancer in women. The company maintains collaborative agreements with major pharmaceutical firms and organizations, such as F. Hoffmann-La Roche Ltd. and the European Organisation for Research and Treatment of Cancer, to advance precision medicine and develop companion diagnostics. Founded in 2009 and headquartered in Cambridge, Massachusetts, Foundation Medicine is a subsidiary of Roche Holdings, Inc.

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.

Lotus Tissue Repair is developing its proprietary recombinant human collagen Type VII (rC7) technology as a treatment for dermatologic conditions in which rC7 may play an important role in accelerating chronic wound healing, such as diabetic foot ulcers, venous stasis ulcers and similar conditions.

Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D, for cerebral adrenoleukodystrophy. In oncology, Bluebird Bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.

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.

Ablexis, LLC is a biotechnology company based in San Francisco, California, specializing in human antibody drug discovery and development. Founded in December 2009, the company offers the AlivaMab Mouse, a transgenic mouse platform designed to enhance the efficiency of discovering therapeutic antibodies for human diseases. This innovative platform significantly improves upon traditional antibody discovery methods, facilitating biotechnology and pharmaceutical companies in developing antibody and antibody-derivative drug candidates. Ablexis commercializes its technology through strategic partnerships, aiming to enable the next generation of breakthrough antibody drugs.

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.

Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D, for cerebral adrenoleukodystrophy. In oncology, Bluebird Bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.

Agios Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of medicines targeting cellular metabolism and related areas, including hematologic malignancies, solid tumors, and rare genetic diseases. The company offers TIBSOVO, an oral targeted inhibitor for treating relapsed or refractory acute myeloid leukemia (AML) in adult patients, and IDHIFA, which is also aimed at AML patients with specific mutations. Agios is advancing several drug candidates through various stages of clinical trials, including TIBSOVO for frontline AML and cholangiocarcinoma, mitapivat for pyruvate kinase deficiency and thalassemia, and vorasidenib for solid tumors. The company emphasizes a precision medicine approach, focusing on genetically or biomarker-defined patient populations to enhance the potential for early proof of concept and accelerated approval. Agios Pharmaceuticals was established in 2007 and has evolved from its original name, Cancer Metabolism Therapeutics, to its current identity.