Third Rock Ventures

Trace Neuroscience is a biopharmaceutical company dedicated to advancing genetic medicine for patients with neurodegenerative diseases. It specializes in developing targeted antisense oligonucleotide therapies, with its lead product aimed at restoring UNC13A protein function in individuals with Amyotrophic Lateral Sclerosis (ALS) caused by TDP-43 mislocalization. This therapy corrects UNC13A mRNA splicing, potentially improving muscle function and extending survival for these patients.

CARGO Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

Clasp Therapeutics develops precision oncology platforms to identify neoantigens from driver mutations in hard-to-treat cancers. Its technology enables the creation of targeted therapies that selectively bind to HLA-presented peptides, allowing for precise destruction of cancer cells across various tumor types.

Flare Therapeutics is a biotechnology company that develops small-molecule medicines by targeting transcription factors. It focuses on identifying switch sites—druggable regions that regulate transcription factor activity—to address disease-causing mutations. Leveraging insights into transcription factor biology, Flare aims to translate this understanding into precision medicines, building an early pipeline that is initially focused on precision oncology with potential expansions into neurology, rare genetic disorders, immunology, and inflammation. The company combines a drug-discovery platform with a biology-led approach to discover therapies that alter the trajectory of diseases driven by transcription factor dysregulation.

CARGO Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

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

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, focused on discovering and developing innovative therapies for severe liver diseases. Founded in 2016, Ambys aims to transform the lives of individuals suffering from liver conditions by creating advanced gene, drug, and cell therapies. The company is dedicated to restoring liver function, halting the progression of liver disease, and addressing the serious complications associated with liver failure. Through its regenerative medical therapies, Ambys seeks to provide relief to patients with chronic liver diseases, ultimately improving their quality of life.

Flare Therapeutics is a biotechnology company that develops small-molecule medicines by targeting transcription factors. It focuses on identifying switch sites—druggable regions that regulate transcription factor activity—to address disease-causing mutations. Leveraging insights into transcription factor biology, Flare aims to translate this understanding into precision medicines, building an early pipeline that is initially focused on precision oncology with potential expansions into neurology, rare genetic disorders, immunology, and inflammation. The company combines a drug-discovery platform with a biology-led approach to discover therapies that alter the trajectory of diseases driven by transcription factor dysregulation.

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

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

Nurix Therapeutics is a biopharmaceutical company focused on discovering, developing, and commercializing oral small-molecule therapies that modulate cellular protein levels to treat cancer and immune disorders. It operates DELigase, an integrated discovery platform that targets E3 ligases to alter protein abundance within cells. The company's product pipeline includes targeted protein degraders such as NX-2127, an oral Bruton's tyrosine kinase degrader for relapsed or refractory B-cell malignancies, and NX-1607, an oral Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology indications; these are in preclinical stages. Nurix also explores inhibitors of CBL-B, an E3 ligase that regulates T cell activation. The company collaborates with major industry partners, including Gilead Sciences and Sanofi, and was founded in 2009 and is headquartered in San Francisco.

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

Maze Therapeutics is a biotechnology company focused on developing medicines that mimic the effects of rare, protective genetic variants to address unmet medical needs. Utilizing its Compass platform, the company identifies genetic variants associated with diseases and maps them to the biological pathways driving these conditions in specific patient populations.

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, focused on discovering and developing innovative therapies for severe liver diseases. Founded in 2016, Ambys aims to transform the lives of individuals suffering from liver conditions by creating advanced gene, drug, and cell therapies. The company is dedicated to restoring liver function, halting the progression of liver disease, and addressing the serious complications associated with liver failure. Through its regenerative medical therapies, Ambys seeks to provide relief to patients with chronic liver diseases, ultimately improving their quality of life.

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

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

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

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

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

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

Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for treating rare genetic disorders that cause life-threatening metabolic issues. Its lead product candidate, setmelanotide, is a potent melanocortin-4 receptor agonist designed for precision medicine to treat hyperphagia and severe obesity resulting from rare MC4R pathway diseases.

Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at revolutionizing cancer treatment. By harnessing the immune system's ability to recognize and attack neoantigens, the company seeks to develop innovative therapies that improve patient outcomes. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to enhance the immune response against tumors. Through its focused research and development efforts, Neon Therapeutics is committed to advancing the field of immuno-oncology and providing new options for cancer patients.

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

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

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

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

Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at revolutionizing cancer treatment. By harnessing the immune system's ability to recognize and attack neoantigens, the company seeks to develop innovative therapies that improve patient outcomes. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to enhance the immune response against tumors. Through its focused research and development efforts, Neon Therapeutics is committed to advancing the field of immuno-oncology and providing new options for cancer patients.

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

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

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

MyoKardia, Inc. is a clinical-stage biopharmaceutical company based in Brisbane, California, specializing in the discovery, development, and commercialization of targeted therapies for rare cardiovascular diseases. Founded in 2012, the company focuses on heritable cardiomyopathies and genetically-driven heart failure stemming from biomechanical defects in cardiac muscle contraction. Its lead product candidate, Mavacamten, is an orally administered allosteric modulator of cardiac myosin currently undergoing Phase III clinical trials for hypertrophic cardiomyopathy (HCM). Additionally, MyoKardia is developing Danicamtiv, which has completed Phase I and IIa trials for dilated cardiomyopathy, alongside several preclinical programs targeting related cardiovascular conditions. As of November 2020, MyoKardia operates as a subsidiary of Bristol-Myers Squibb Company.

Nurix Therapeutics is a biopharmaceutical company focused on discovering, developing, and commercializing oral small-molecule therapies that modulate cellular protein levels to treat cancer and immune disorders. It operates DELigase, an integrated discovery platform that targets E3 ligases to alter protein abundance within cells. The company's product pipeline includes targeted protein degraders such as NX-2127, an oral Bruton's tyrosine kinase degrader for relapsed or refractory B-cell malignancies, and NX-1607, an oral Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology indications; these are in preclinical stages. Nurix also explores inhibitors of CBL-B, an E3 ligase that regulates T cell activation. The company collaborates with major industry partners, including Gilead Sciences and Sanofi, and was founded in 2009 and is headquartered in San Francisco.

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

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

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

Edimer Pharmaceuticals is a biotechnology company focused on developing innovative treatments for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder characterized by symptoms such as the absence of sweat glands, impaired temperature regulation, respiratory issues, and malformations of hair and teeth. The company's lead product candidate, EDI200, aims to provide significant and lasting improvements in health and quality of life for individuals affected by this condition. Edimer is guided by a team of experienced professionals in the biotechnology sector, supported by a network of distinguished clinical and scientific advisors, all committed to addressing the needs of families impacted by XLHED.

Nurix Therapeutics is a biopharmaceutical company focused on discovering, developing, and commercializing oral small-molecule therapies that modulate cellular protein levels to treat cancer and immune disorders. It operates DELigase, an integrated discovery platform that targets E3 ligases to alter protein abundance within cells. The company's product pipeline includes targeted protein degraders such as NX-2127, an oral Bruton's tyrosine kinase degrader for relapsed or refractory B-cell malignancies, and NX-1607, an oral Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology indications; these are in preclinical stages. Nurix also explores inhibitors of CBL-B, an E3 ligase that regulates T cell activation. The company collaborates with major industry partners, including Gilead Sciences and Sanofi, and was founded in 2009 and is headquartered in San Francisco.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for treating rare genetic disorders that cause life-threatening metabolic issues. Its lead product candidate, setmelanotide, is a potent melanocortin-4 receptor agonist designed for precision medicine to treat hyperphagia and severe obesity resulting from rare MC4R pathway diseases.

MyoKardia, Inc. is a clinical-stage biopharmaceutical company based in Brisbane, California, specializing in the discovery, development, and commercialization of targeted therapies for rare cardiovascular diseases. Founded in 2012, the company focuses on heritable cardiomyopathies and genetically-driven heart failure stemming from biomechanical defects in cardiac muscle contraction. Its lead product candidate, Mavacamten, is an orally administered allosteric modulator of cardiac myosin currently undergoing Phase III clinical trials for hypertrophic cardiomyopathy (HCM). Additionally, MyoKardia is developing Danicamtiv, which has completed Phase I and IIa trials for dilated cardiomyopathy, alongside several preclinical programs targeting related cardiovascular conditions. As of November 2020, MyoKardia operates as a subsidiary of Bristol-Myers Squibb Company.

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

Nurix Therapeutics is a biopharmaceutical company focused on discovering, developing, and commercializing oral small-molecule therapies that modulate cellular protein levels to treat cancer and immune disorders. It operates DELigase, an integrated discovery platform that targets E3 ligases to alter protein abundance within cells. The company's product pipeline includes targeted protein degraders such as NX-2127, an oral Bruton's tyrosine kinase degrader for relapsed or refractory B-cell malignancies, and NX-1607, an oral Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology indications; these are in preclinical stages. Nurix also explores inhibitors of CBL-B, an E3 ligase that regulates T cell activation. The company collaborates with major industry partners, including Gilead Sciences and Sanofi, and was founded in 2009 and is headquartered in San Francisco.

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

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

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

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

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

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

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

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

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

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

Edimer Pharmaceuticals is a biotechnology company focused on developing innovative treatments for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder characterized by symptoms such as the absence of sweat glands, impaired temperature regulation, respiratory issues, and malformations of hair and teeth. The company's lead product candidate, EDI200, aims to provide significant and lasting improvements in health and quality of life for individuals affected by this condition. Edimer is guided by a team of experienced professionals in the biotechnology sector, supported by a network of distinguished clinical and scientific advisors, all committed to addressing the needs of families impacted by XLHED.

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

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