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Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, established in 2011. The company specializes in the development and commercialization of Haduvio, an oral extended-release formulation of nalbuphine. Haduvio is currently undergoing a Phase IIb/III clinical trial, known as the PRISM trial, aimed at treating chronic pruritus associated with prurigo nodularis. Additionally, Trevi is exploring the use of nalbuphine ER for other serious neurologically mediated conditions, including chronic cough in patients with idiopathic pulmonary fibrosis and levodopa-induced dyskinesia in individuals with Parkinson’s disease. The treatment targets conditions that share a common pathophysiology mediated through opioid receptors in the central and peripheral nervous systems.

35Pharma is a biopharmaceutical company focused on designing and developing innovative biologics to address diseases with high unmet medical needs, particularly pulmonary hypertension and musculoskeletal disorders. The company leverages its expertise in protein engineering and its understanding of transforming growth factor-beta (TGF-beta) structure-function relationships to create highly potent ligand traps. These developments aim to enhance treatment options for patients suffering from cardio-pulmonary and metabolic diseases. By combining advanced scientific knowledge with a commitment to addressing critical health challenges, 35Pharma seeks to contribute significantly to the biopharmaceutical landscape.

ADCendo ApS is a biotech company focused on the development of innovative anti-cancer therapies, specifically through the creation of antibody-drug conjugates (ADCs). These conjugates are designed to target receptors that are overexpressed in various forms of cancer, addressing significant unmet medical needs in the treatment landscape. ADCendo's approach aims to enhance targeted therapy for patients suffering from conditions such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, among others. By developing new medicines that effectively target carcinomas—including breast, colon, pancreatic, prostate, ovarian, and renal cancers—ADCendo seeks to improve treatment outcomes for cancer patients.

Aclaris Therapeutics is a clinical-stage biopharmaceutical company based in the United States, specializing in the development of innovative therapies for dermatological and immuno-inflammatory conditions. Founded by the creators of Vicept Therapeutics, the company is dedicated to addressing significant unmet needs in these fields, focusing on areas where no FDA-approved treatments exist or where treatment options are limited. Aclaris operates primarily in two segments: therapeutics and contract research. The therapeutics segment is engaged in the identification and development of novel drug candidates, while the contract research segment generates revenue through laboratory services. Among its developmental candidates is Zunsemetinib, an oral MK2 inhibitor aimed at treating immuno-inflammatory diseases.

858 Therapeutics is a biotechnology company dedicated to the discovery of small molecule therapeutics aimed at treating cancers and other diseases, particularly those resistant to existing therapies. The company employs innovative strategies in areas such as synthetic lethality, innate immunity, and RNA modulation to develop its therapeutic candidates. Its lead program focuses on a PARG inhibitor, which is currently in early clinical development. By leveraging its platform to investigate the role of RNA modifying proteins in disease biology, 858 Therapeutics is generating a pipeline of targeted small molecules that address critical unmet medical needs in oncology and immunology.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

Enliven Therapeutics is a clinical-stage precision oncology company based in Boulder, Colorado, focused on developing innovative small molecule therapies to enhance patient outcomes. Established in July 2019 by Sam Kintz, Joe Lyssikatos, and Anish Patel, the company employs a unique discovery process that leverages clinically validated biological targets alongside advanced chemistry. This methodology aims to overcome the limitations of current therapies by creating potentially first-in-class treatments that address critical challenges such as tolerability, combinability, resistance, and disease progression, particularly in cases involving brain metastases. Enliven's product pipeline includes ELVN-001, a selective small molecule kinase inhibitor targeting the BCR-ABL gene fusion in chronic myeloid leukemia, and ELVN-002, an irreversible HER2 inhibitor designed to penetrate the central nervous system and act against various HER2 variants.

Cybin is a biopharmaceutical company dedicated to advancing research and development in psychedelic and medicinal mushrooms. It focuses on creating safe and effective therapeutics for mental health issues, with an emphasis on psilocybin-based products. The company is actively launching these products in jurisdictions where they are permitted and is engaged in clinical studies across North America and other regions. Through strategic partnerships with academic and institutional entities, Cybin seeks to develop novel compounds and delivery mechanisms, aiming to provide innovative treatments for various psychiatric and neurological conditions.

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for serious diseases that currently lack approved therapies. Established in 2016 and based in New York, the company is advancing a pipeline of novel product candidates that target high unmet medical needs, including cardiovascular diseases and metabolic disorders. Its lead candidate, AT-007, is in Phase I/II trials aimed at treating galactosemia. Additionally, AT-001 is undergoing Phase II trials for diabetic cardiomyopathy and Phase I trials for diabetic peripheral neuropathy, while AT-003 is in Phase I trials for diabetic retinopathy. The company also has preclinical candidates such as AT-104, intended for orphan hematological oncology conditions, demonstrating its commitment to addressing critical health challenges through targeted therapeutics.

enGene, Inc. is a biotechnology company based in Vancouver, Canada, specializing in mucosal immunotherapy to address inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables the localized delivery of immune-modulating proteins to mucosal tissues, targeting conditions affecting the gastrointestinal tract, lung, and bladder. This innovative platform allows for the systemic release of proteins from the gut, which can be beneficial for treating a variety of immune disorders, as well as other conditions such as diabetes, anemia, and hemophilia. Founded in 1999, enGene has formed a strategic alliance with Takeda Pharmaceutical Company to further advance its therapeutic offerings.

ProfoundBio is a clinical-stage biotechnology company dedicated to creating novel antibody-based therapeutics aimed at treating cancer. The company utilizes innovative proprietary technology platforms to develop a diverse pipeline of antibody-drug conjugate (ADC) candidates that target both solid tumors and hematological malignancies. These drug candidates are currently in various stages of discovery, preclinical, and clinical development. ProfoundBio's focus on advancing targeted therapeutics leverages the immune system's capabilities to potentially eradicate cancer, offering improved treatment options with curative potential for patients.

Adverum Biotechnologies is a clinical-stage gene therapy company focused on developing innovative treatments for ocular and rare diseases. Headquartered in Redwood City, California, the company aims to establish gene therapy as a new standard of care, aspiring to provide functional cures that restore vision and prevent blindness. Its pipeline includes several product candidates, notably ADVM-022, which targets wet age-related macular degeneration through a single intravitreal injection designed to deliver long-term therapeutic effects. Other candidates include ADVM-043 for alpha-1 antitrypsin deficiency and ADVM-053 for hereditary angioedema. Adverum collaborates with partners like Editas Medicine and Regeneron Pharmaceuticals to leverage advanced AAV vectors and develop treatments for various inherited retinal diseases and ocular therapeutic targets. Founded in 2006, the company was previously known as Avalanche Biotechnologies and rebranded in 2016.

Silence Therapeutics plc is a biotechnology company based in London, specializing in the discovery and development of innovative RNA therapeutics. The company focuses on utilizing short interfering RNA (siRNA) technology to modulate gene expression and address various medical conditions, including hematology, cardiovascular diseases, and rare metabolic disorders. Its pipeline includes several product candidates, such as SLN124, aimed at treating iron overload disorders by silencing the TMPRSS6 gene, and SLN360, which targets the LPA gene to reduce the risk of cardiovascular diseases. Silence Therapeutics has established strategic collaborations with major organizations, including AstraZeneca, to advance the development of siRNA therapeutics across multiple disease areas. The company leverages its proprietary genetic toolkit and delivery systems to optimize therapeutic outcomes, harnessing the body's natural biological mechanisms for effective treatment.

Dianthus Therapeutics is a clinical-stage biotechnology company focused on developing innovative treatments for severe autoimmune diseases through targeted therapies. The company's lead candidate, DNTH103, is a monoclonal antibody designed to selectively inhibit a specific component of the immune system, offering potential advantages over existing complement therapies. This treatment aims to improve safety and efficacy for patients suffering from serious autoimmune and inflammatory conditions. Dianthus is committed to advancing its pipeline of next-generation complement therapeutics, supported by a team of experienced professionals in the biotech and pharmaceutical sectors. The company envisions that DNTH103 could be administered as a self-injection for certain patients, enhancing convenience and accessibility in treatment.

Tourmaline Bio is a clinical biotechnology company focused on developing transformative medicines aimed at improving the lives of patients suffering from immune diseases. The company is in the late stages of clinical development and is centered on its lead product candidate, TOUR006, a fully human monoclonal antibody designed to selectively target interleukin-6 (IL-6), a significant proinflammatory cytokine implicated in various autoimmune and inflammatory disorders. The IL-6 antibody class has a well-established history, with over two decades of clinical and commercial experience, having successfully treated more than a million patients with diverse autoimmune and inflammatory conditions. Through its innovative research, Tourmaline Bio seeks to address the unmet medical needs of patients affected by life-altering immune diseases.

VistaGen Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for central nervous system disorders, particularly depressive and social anxiety disorders. The company's lead product candidate, AV-101, is an orally available treatment currently in Phase 2 development for major depressive disorder, targeting patients who have not adequately responded to standard antidepressants. Additionally, VistaGen is advancing PH94B, a neuroactive nasal spray, which is preparing for Phase III clinical trials for the acute treatment of anxiety in adults with social anxiety disorder. Another product in its pipeline, PH10, is being planned for Phase 2b development as a treatment for major depressive disorder. VistaGen has established licensing and collaboration agreements to facilitate the clinical development and commercialization of its products, including a strategic partnership for PH94B in Greater China, South Korea, and Southeast Asia. Founded in 1998, VistaGen is headquartered in South San Francisco, California.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing precision medicines for neurological disorders. The company focuses on creating transformational small-molecule therapies aimed at patients with central nervous system conditions. Leveraging foundational research in neuronal receptor biology, Rapport Therapeutics maps and targets specific neuronal receptor complexes, which are complex assemblies of proteins that include principal receptor subunits and receptor-associated proteins. These receptor-associated proteins are essential for regulating receptor expression and functionality, allowing the company to innovate in the treatment of neurological diseases.

COMPASS Pathways is a mental health care company based in Cheshire, United Kingdom, with operations in the United States. The company is focused on developing innovative therapies for treatment-resistant depression (TRD) using its proprietary psilocybin formulation, COMP360. Currently undergoing Phase IIb clinical trials, COMP360 aims to provide a new treatment option for patients who have not responded to traditional depression therapies. COMPASS Pathways emphasizes the combination of psilocybin-assisted therapy with psychological support to enhance treatment efficacy. The company is committed to addressing the significant unmet needs in mental health care by advancing evidence-based solutions for those suffering from mental health challenges.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines utilizing its proprietary virus-like particle (VLP) platform technology. This innovative approach allows for the multivalent, particle-based display of complex viral antigens, which is aimed at generating broad and durable immune responses. Icosavax focuses primarily on life-threatening respiratory diseases, with its lead vaccine candidate, IVX-121, targeting respiratory syncytial virus (RSV) in older adults. The company's pipeline also includes candidates for human metapneumovirus (hMPV) and SARS-CoV-2, reflecting its commitment to advancing vaccine solutions for infectious diseases.

Boundless Bio, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, focused on developing innovative therapies for the treatment of aggressive cancers. The company specializes in targeting extrachromosomal DNA (ecDNA), a key factor in the amplification of oncogenes that affects over 14% of cancer patients. By investigating the biology of ecDNA, Boundless Bio aims to create transformative treatments for previously untreatable cancers. Its lead therapeutic candidate, BBI-355, is an oral selective inhibitor of checkpoint kinase 1 (CHK1) designed to manage the replication and transcription of ecDNA in cancer cells. Founded in 2018, Boundless Bio is committed to addressing the significant unmet medical needs of patients with oncogene amplified tumors.

Delcath Systems, Inc. is an interventional oncology company based in New York that specializes in the treatment of primary and metastatic liver cancers. The company's primary product is Melphalan Hydrochloride for Injection, which is combined with the Delcath Hepatic Delivery System to deliver high-dose chemotherapy directly to the liver while minimizing systemic exposure and side effects. Delcath is currently conducting Phase III clinical trials, including the FOCUS Trial for patients with hepatic dominant ocular melanoma and the ALIGN Trial for intrahepatic cholangiocarcinoma. In addition to its clinical efforts, the company markets Melphalan Hydrochloride under the Delcath Hepatic CHEMOSAT Delivery System in Europe. Founded in 1988, Delcath Systems is dedicated to advancing treatment options for patients with liver cancer.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies to treat chronic inflammatory and autoimmune diseases. The company's pipeline includes three small molecule products. The lead program, IMU-838, is a selective immune modulator that blocks the enzyme DHODH, inhibiting the metabolism of activated immune cells and exhibiting antiviral effects. It is being developed for multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. IMU-935, a selective inverse agonist of the transcription factor RORγt, targets psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Additionally, IMU-856 aims to restore intestinal barrier function for diseases involving bowel barrier dysfunction.

Immunovant, headquartered in New York, is a clinical-stage biopharmaceutical company dedicated to improving the lives of patients with autoimmune diseases. Its primary focus is on developing monoclonal antibodies, notably IMVT-1401, a fully human antibody that targets the neonatal Fc receptor (FcRn). This investigational product is designed for subcutaneous injection and aims to address various IgG-mediated autoimmune conditions. IMVT-1401 is currently in Phase IIa clinical trials for myasthenia gravis and thyroid eye disease, with Phase II trials also initiated for warm autoimmune hemolytic anemia. Additionally, the company is developing batoclimab and IMVT-1402, both targeting FcRn.

Allakos Inc. is a clinical-stage biopharmaceutical company founded in 2012 and headquartered in Redwood City, California. The company focuses on developing antibody-based therapeutics aimed at treating diseases associated with dysregulation of the T-helper type 2 immune response, which plays a crucial role in various allergic and inflammatory conditions. Its lead product, antolimab (AK002), is being investigated for the treatment of eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets the Siglec-6 receptor on mast cells, designed to activate the receptor's inhibitory function and reduce mast cell activation. Through these innovative therapies, Allakos aims to address the underlying mechanisms of diseases characterized by excessive inflammatory responses.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing innovative therapies for autoimmune diseases and inflammatory disorders. Incorporated in 2018, Ventyx focuses on creating selective inhibitors targeting TYK2, a key mediator in various inflammatory conditions. The company's clinical pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, which aims to provide a safer alternative by avoiding the toxicities linked to broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator intended for ulcerative colitis treatment, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which plays a significant role in multiple inflammatory diseases.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

Triana Biomedicines is a pharmaceutical company focused on discovering and developing molecular glues, which are innovative therapeutic agents designed to regulate challenging disease targets. The company utilizes a sophisticated drug discovery platform that integrates high-resolution structural insights with advanced in silico tools and customized chemical libraries. This technology allows Triana to stabilize existing protein interactions or create new ones, thereby modifying the behavior or functionality of disease targets. The research team has successfully validated their platform and has initiated multiple drug development programs across various disease areas, aiming to provide healthcare professionals with effective treatment options for a broad spectrum of therapeutic applications.

Enliven Therapeutics is a clinical-stage precision oncology company based in Boulder, Colorado, focused on developing innovative small molecule therapies to enhance patient outcomes. Established in July 2019 by Sam Kintz, Joe Lyssikatos, and Anish Patel, the company employs a unique discovery process that leverages clinically validated biological targets alongside advanced chemistry. This methodology aims to overcome the limitations of current therapies by creating potentially first-in-class treatments that address critical challenges such as tolerability, combinability, resistance, and disease progression, particularly in cases involving brain metastases. Enliven's product pipeline includes ELVN-001, a selective small molecule kinase inhibitor targeting the BCR-ABL gene fusion in chronic myeloid leukemia, and ELVN-002, an irreversible HER2 inhibitor designed to penetrate the central nervous system and act against various HER2 variants.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

Neumora Therapeutics is a clinical-stage biotechnology company focused on developing precision medicines for brain diseases by combining data science with neuroscience. Founded to address the global brain disease crisis, Neumora takes an innovative approach to treatment development. The company has established a robust therapeutic pipeline that includes seven clinical and preclinical neuroscience programs targeting novel mechanisms of action for various underserved neuropsychiatric and neurodegenerative disorders. Neumora plans to advance its pipeline further by initiating multiple clinical trials across its programs in the upcoming 12 to 18 months.

858 Therapeutics is a biotechnology company dedicated to the discovery of small molecule therapeutics aimed at treating cancers and other diseases, particularly those resistant to existing therapies. The company employs innovative strategies in areas such as synthetic lethality, innate immunity, and RNA modulation to develop its therapeutic candidates. Its lead program focuses on a PARG inhibitor, which is currently in early clinical development. By leveraging its platform to investigate the role of RNA modifying proteins in disease biology, 858 Therapeutics is generating a pipeline of targeted small molecules that address critical unmet medical needs in oncology and immunology.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing innovative therapies for autoimmune diseases and inflammatory disorders. Incorporated in 2018, Ventyx focuses on creating selective inhibitors targeting TYK2, a key mediator in various inflammatory conditions. The company's clinical pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, which aims to provide a safer alternative by avoiding the toxicities linked to broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator intended for ulcerative colitis treatment, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which plays a significant role in multiple inflammatory diseases.

Alpine Immune Sciences, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, that specializes in the discovery and development of protein-based immunotherapies aimed at treating cancer, autoimmune, and inflammatory disorders. The company leverages a proprietary scientific platform to transform native immune system proteins into innovative therapeutic candidates. Its leading programs include ALPN-101, a dual antagonist targeting the inducible T cell costimulator and CD28 pathways, designed for autoimmune and inflammatory conditions, and ALPN-303, a dual antagonist of the B cell activating factor and proliferation-inducing ligand, which is vital for B cell activation and survival. Additionally, Alpine Immune Sciences has established a collaboration with Kite Pharma to explore immunotherapies targeting the immune synapse for cancer treatment.

Obsidian Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing next-generation cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Its innovative approach utilizes pharmacologic operating systems that enable precise control over protein activity within cells. This technology allows for the creation of adoptive cell therapies with novel functionalities, which can be regulated by treating physicians using simple and safe orally active medications. Through these advancements, Obsidian Therapeutics seeks to improve upon existing cell therapies and expand treatment options for a broader patient demographic.

RareCyte, Inc. is a life sciences company based in Seattle, Washington, founded in 2009. The company specializes in developing advanced platforms for liquid and tissue biopsy analysis, focusing on the detection and characterization of rare cells, particularly circulating tumor cells (CTCs) and circulating endothelial cells (CECs) in blood samples. RareCyte's AccuCyte system provides an open platform that facilitates the unbiased discovery and isolation of these rare cells, supporting various applications in cancer research, cardiovascular disease studies, and diagnostic development. The company offers a range of products, including sample preparation systems, staining kits, automated fluorescence microscopy instruments, and slide imaging systems. By enabling precision medicine approaches, RareCyte aims to provide new tools for medical professionals and researchers to address complex questions in the fields of cancer and other therapeutic 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.

Vigil Neuroscience Inc is a therapeutics company focused on developing treatments for both rare and common neurodegenerative diseases by targeting microglia, the brain's immune cells. The company aims to restore the vigilance of these cells to improve patient outcomes. Vigil is advancing its lead candidate, a monoclonal antibody TREM2 agonist, through Phase 1 clinical studies, while also working on VG-3927, an orally available small molecule TREM2 agonist. These efforts are part of a broader strategy to build a robust pipeline of therapies supported by the understanding of microglia biology as a critical therapeutic pathway. Vigil's clinical candidate, iluzanebart, is currently undergoing a Phase 2 proof-of-concept trial in patients with ALSP, marking a significant step in the company's commitment to precision-based neuroscience drug development.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Interius BioTherapeutics is a biopharmaceutical company that develops in vivo cell-specific gene medicines to treat B cell lymphomas. Intravenous in vivo CAR treatment, which treats B cell lymphomas, is the company's flagship program. Its unique engineering provides exceptional target tissue specificity. The business is working on a second initiative to address autoimmune conditions. With the help of Interius, a novel and distinct therapeutic modality for the precise administration of gene therapies may soon be made available to patients in expanded care settings, without the need for preconditioning chemotherapy.

Nuvalent is a clinical stage biotechnology company specializing in the development of targeted therapies aimed at clinically validated kinase targets in cancer. Focusing on small molecules, Nuvalent employs a structure-based design approach to create treatments that possess high selectivity, intending to address the limitations of existing therapies by overcoming resistance and minimizing side effects. The company is advancing a promising pipeline that includes lead programs targeting ROS1-positive and ALK-positive non-small cell lung cancer (NSCLC). Additionally, Nuvalent is engaged in various discovery-stage research initiatives that aim to expand its therapeutic offerings.

Gennao Bio is a privately held company focused on developing innovative targeted nucleic acid therapeutics, primarily aimed at treating oncology and rare monogenic skeletal muscle diseases. The company utilizes its proprietary gene monoclonal antibody (GMAB) platform technology, which features a novel, cell-penetrating antibody that facilitates the non-covalent binding and delivery of various nucleic acid payloads to specific cells. This non-viral delivery system stands out from traditional gene therapy methods by allowing for multiple types of nucleic acids, enabling repeat dosing, and employing established manufacturing processes. Gennao Bio's approach aims to address significant unmet medical needs and improve patient outcomes in the relevant therapeutic areas.

Boundless Bio, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, focused on developing innovative therapies for the treatment of aggressive cancers. The company specializes in targeting extrachromosomal DNA (ecDNA), a key factor in the amplification of oncogenes that affects over 14% of cancer patients. By investigating the biology of ecDNA, Boundless Bio aims to create transformative treatments for previously untreatable cancers. Its lead therapeutic candidate, BBI-355, is an oral selective inhibitor of checkpoint kinase 1 (CHK1) designed to manage the replication and transcription of ecDNA in cancer cells. Founded in 2018, Boundless Bio is committed to addressing the significant unmet medical needs of patients with oncogene amplified tumors.

Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at treating cancer. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux aims to generate targeted immune responses that effectively combat tumors while minimizing effects on healthy tissue. The company's approach seeks to enhance all three stages of the anti-tumor immune response, making it a promising player in the field of cancer treatment.

GH Research PLC is a clinical-stage biopharmaceutical company committed to advancing treatments for psychiatric and neurological disorders. The company's primary focus is on developing novel and proprietary therapies using Mebufotenin 5-Methoxy-N, N-Dimethyltryptamine, commonly known as 5-MeO-DMT, specifically targeting patients with Treatment-Resistant Depression.

Tyra Biosciences, Inc. is a clinical-stage biotechnology company based in Carlsbad, California, dedicated to developing precision oncology therapies targeted at overcoming drug resistance in cancer treatment. Founded in 2018, the company utilizes its proprietary SNÅP platform to facilitate rapid and precise drug design by generating iterative molecular snapshots. This innovative approach allows Tyra to create next-generation therapies specifically aimed at addressing acquired resistance in tumors. The company's primary focus is on developing selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. Tyra's lead product candidate, TYRA-300, is designed to selectively inhibit FGFR3, initially targeting patients with metastatic urothelial carcinoma of the bladder and urinary tract.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that focuses on targeted DNA integration to address severe genetic diseases. The firm employs advanced technology to enable the precise insertion of genetic sequences, effectively correcting mutations, replacing malfunctioning genes, and introducing therapeutic genetic materials into specific regions of the genome. By harnessing high-efficiency targeted gene integration, Graphite Bio aims to develop novel therapies that could potentially cure a wide array of serious and life-threatening conditions. Formerly known as Integral Medicines, Inc., the company rebranded in August 2020 and has operated since its incorporation in 2019. Graphite Bio is committed to the pioneering of a precision gene editing approach designed to fulfill the medical challenge of accurately "finding and replacing" genes.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing innovative therapies for autoimmune diseases and inflammatory disorders. Incorporated in 2018, Ventyx focuses on creating selective inhibitors targeting TYK2, a key mediator in various inflammatory conditions. The company's clinical pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, which aims to provide a safer alternative by avoiding the toxicities linked to broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator intended for ulcerative colitis treatment, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which plays a significant role in multiple inflammatory diseases.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that incorporates a proprietary dry-powder formulation of dihydroergotamine mesylate. Designed for self-administration, STS101 utilizes a pre-filled, single-use nasal delivery device, enhancing patient convenience and accessibility. The product is currently undergoing Phase III clinical trials, marking a significant step in its development. Founded in 2016, Satsuma is headquartered in South San Francisco, California.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Viracta Therapeutics, Inc. is a clinical-stage drug development company based in Cardiff, California, that specializes in precision oncology focused on virus-associated malignancies. The company utilizes a viral activation therapy platform to develop treatments for cancers linked to the Epstein-Barr Virus (EBV) and other virus-related diseases. Its lead investigational drug, nanatinostat, is being evaluated in combination with the antiviral valganciclovir as a potential oral therapy for EBV-positive lymphoma, including post-transplant lymphoproliferative disorder, plasmablastic lymphoma, and angioimmunoblastic T cell lymphoma. Viracta is also exploring the application of this approach to other EBV-associated conditions, such as nasopharyngeal carcinoma and gastric carcinoma. Founded in 2007 and rebranded from HemaQuest Pharmaceuticals in 2015, Viracta Therapeutics is actively conducting multiple clinical trials to advance its therapeutic candidates.

Centessa Pharmaceuticals is a clinical-stage biopharmaceutical company focused on innovating the drug development process through an asset-centric research and development model. The company manages a diverse portfolio of therapeutics across several therapeutic areas, including SerpinPC for Hemophilia A and B, LB101 for solid tumors, ORX750 for Narcolepsy Type 1 and other sleep disorders, and MGX292 for pulmonary arterial hypertension. Each therapeutic program is developed by a dedicated subsidiary, leveraging a centralized infrastructure and experienced management team to ensure efficient advancement of its pipeline assets.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, specializing in the development of vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. Founded in 2014, the company aims to enhance the protection against a range of infectious diseases that affect both children and adults globally. MAPS technology allows for the effective binding of protective polysaccharides and proteins within a single vaccine, inducing robust immune responses. Affinivax is advancing vaccine programs targeting significant pathogens, including Streptococcus pneumoniae, which contributes to considerable morbidity and mortality worldwide. The company leverages insights from leading experts in infectious diseases and has secured exclusive intellectual property rights related to its MAPS platform from Boston Children’s Hospital, with notable backing from the Bill & Melinda Gates Foundation.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

SomaLogic, Inc. is a commercial-stage company based in Boulder, Colorado, specializing in protein biomarker discovery and clinical diagnostics. Founded in 1999, the company has developed advanced technologies such as SOMAmers, slow off-rate modified aptamers that specifically bind to target proteins, and the SOMAscan platform, which facilitates comprehensive protein measurement. This innovative approach allows for the analysis of protein biomarker signatures, significantly enhancing the understanding of health and disease beyond traditional genomic methods. SomaLogic's products are utilized in a variety of applications, including diagnostics for conditions in oncology, neurology, and cardiovascular and metabolic diseases, as well as in drug discovery and development. The company also offers diagnostic tests and protein measurement solutions for life science and biopharma researchers, contributing to advancements in medical science.

Cullinan Therapeutics is a developer of an externally sourced cancer therapeutics intended to end a drug program quickly if the early research suggests it won't work. The company's therapeutics are developed via assets sourced internally through dry lab or externally through business, academic and pharma collaborations, enabling researchers to get a highly diversified portfolio of oncology therapeutics with a unique, cost-efficient business model.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing tumor-targeted small molecule medicines that leverage radioisotopes to improve cancer treatment outcomes. RayzeBio focuses on creating innovative radiopharmaceuticals, particularly utilizing alpha-emitting radioisotopes like Actinium-225, to target solid tumors effectively. With a robust pipeline of drug candidates, RayzeBio is committed to addressing significant market opportunities in oncology through late-stage clinical programs, development initiatives, and discovery efforts. The company's mission is to provide effective therapeutic solutions aimed at defeating cancer.

Edgewise Therapeutics, founded in 2017 and based in Boulder, Colorado, is a clinical-stage biopharmaceutical company dedicated to developing precision medicine therapies for severe, rare muscle disorders. The company's core expertise lies in muscle biology and small molecule engineering, which it employs through its proprietary drug discovery platform. This platform uses custom-built systems to measure integrated muscle function, enabling the identification of small molecule therapies targeting key proteins in muscle tissue. Edgewise's pipeline focuses on addressing genetically defined muscle disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophies.

Talaris Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies aimed at eliminating chronic immunosuppression for organ transplant recipients. The company's leading product, FCR001, is designed to enable living donor kidney transplant recipients to discontinue all chronic immunosuppression therapy within twelve months post-transplant. In addition to its applications in organ transplantation, Talaris’s technology facilitates the safe use of hematopoietic stem cells from unmatched and unrelated donors for bone marrow transplants, reducing the associated toxicity and risks of traditional methods. Founded in 1988 and originally named Regenerex, Inc., Talaris Therapeutics rebranded in March 2019. The company is headquartered in Louisville, Kentucky, with an additional office in Wellesley, Massachusetts.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Flame Biosciences is a clinical-stage biotechnology company based in New York, founded in 2017. The company specializes in developing innovative therapies for cancer and other inflammatory conditions. Its portfolio includes FL-101 therapy, designed to treat lung cancers driven by inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Flame Biosciences is dedicated to improving the lives of individuals suffering from debilitating and life-threatening diseases through its transformative therapeutic approaches.

Eledon Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Irvine, California, specializing in the development of innovative treatments for patients with disorders of the ear, nose, and throat, as well as for those undergoing organ or cellular transplantation and individuals with autoimmune and neurodegenerative diseases. The company’s lead product, OP0201, is a surfactant-based nasal aerosol designed for patients at risk for or suffering from otitis media. Additionally, Eledon has developed a foam-based drug delivery technology, OP0101 and OP0102, aimed at delivering medications to the ear, nose, and sinus cavities. The company also focuses on the CD40L pathway, with its main compound in development, tegoprubart, being an IgG1 anti-CD40L antibody that shows promise for treating autoimmune diseases and supporting organ transplants. Eledon Pharmaceuticals was formerly known as Novus Therapeutics, Inc. before its rebranding in January 2021.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing targeted therapies aimed at oncogenic drivers. The company employs a genetic selection strategy to identify patients who are most likely to benefit from its treatments, utilizing a tumor-agnostic approach that prioritizes the underlying genetics of tumors over traditional histological classifications. Its lead product candidate, milademetan, is a small molecule oral inhibitor of MDM2, which plays a role in various cancers. Additionally, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells by inhibiting RAD52, further diversifying its therapeutic pipeline.

Kinnate Biopharma Inc. is a biopharmaceutical company dedicated to the discovery and development of small molecule kinase inhibitors aimed at treating genomically defined cancers. Established in 2018 and headquartered in San Diego, California, the company focuses on precision oncology therapeutics, leveraging its expertise in structure-based drug discovery and translational research. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is advancing other preclinical candidates, including KIN003, which targets cancer-associated alterations in FGFR2 and FGFR3 genes, and a Cyclin-Dependent Kinase 12 inhibitor in the KIN004 program. Kinnate Biopharma is committed to developing targeted therapeutics for underserved patient populations, aiming to expand the reach of precision medicine in cancer treatment.

Dyne Therapeutics, Inc. is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral muscular dystrophy, as well as rare skeletal, cardiac, and metabolic muscle diseases. Utilizing its proprietary FORCE platform, Dyne Therapeutics delivers nucleic acids and other therapeutic molecules to muscle tissues with high precision, aiming to restore muscle strength and improve patients' quality of life. The company is currently advancing its product candidates, DYNE-101 and DYNE-251, which are in phase 1/2 clinical trials.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates aimed at targeting and eliminating disease-causing proteins for the treatment of cancer, neurodegenerative diseases, and other conditions. The company's lead product candidate, CFT7455, is an orally bioavailable degrader targeting IKZF1/3, designed to treat multiple myeloma and various lymphomas. Additionally, C4 Therapeutics is advancing CFT8634, which targets BRD9 for synovial sarcoma and SMARCB1-deleted solid tumors, as well as programs targeting BRAF V600E and RET for genetically defined resistant solid tumors. The company employs its proprietary Degronimid platform, which utilizes small molecule binders to flag harmful proteins for degradation by the cellular proteasome system, allowing for the potential treatment of previously undruggable targets. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics engages in strategic collaborations to enhance its research and development efforts.

Pliant Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, dedicated to discovering and developing innovative therapies for fibrotic diseases. Its lead product candidate, PLN-74809, is an oral small-molecule dual selective inhibitor of avß6 and avß1 integrins, targeting idiopathic pulmonary fibrosis and primary sclerosing cholangitis. PLN-74809 has received Orphan Drug Designation from the U.S. Food and Drug Administration for both indications and is currently undergoing Phase 2a clinical trials. The company is also developing PLN-1474, a small-molecule selective inhibitor of avß1, for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis, which is in Phase 1 trials. Founded in 2015, Pliant Therapeutics is also advancing preclinical programs aimed at addressing oncology and muscular dystrophies.

ALX Oncology is a clinical-stage immuno-oncology company based in Burlingame, California, established in 2015. The company specializes in developing innovative therapies aimed at treating various forms of cancer, with a particular focus on its lead product candidate, ALX148. This therapeutic agent is designed to block the CD47 protein, which cancer cells exploit to evade the immune system. ALX148 is currently being investigated for its efficacy in treating myelodysplastic syndromes, acute myeloid leukemia, and several solid tumor types, including head and neck squamous cell carcinoma and certain gastric cancers. The company leverages advanced protein engineering technologies to enhance the affinity of its CD47 blockers, aiming to minimize hematologic toxicities often associated with existing treatments. ALX Oncology's commitment to improving cancer therapies is rooted in pioneering research conducted by its founders at Stanford University.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for treating various cancers and autoimmune diseases. Unlike T-cell therapies, NK cells have an inherent ability to recognize and destroy abnormal cells without genetic alteration, offering promise for diverse tumor types with potentially better tolerated side effects. Nkarta's approach involves leveraging its NK expansion platform technology alongside proprietary cell engineering techniques to generate a substantial supply of enhanced NK cells that can persistently target and eliminate cancer cells.

Passage Bio, Inc. is a genetic medicines company based in Philadelphia, Pennsylvania, dedicated to developing transformative therapies for rare monogenic central nervous system (CNS) diseases. Established in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while taking responsibility for the clinical development, regulatory processes, manufacturing, and commercialization of its product candidates. The partnership also involves research and development collaborations that support the advancement of innovative treatments for these rare disorders.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, that specializes in discovering and developing targeted therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which integrates genome-wide analysis and CRISPR technology, Repare systematically identifies and advances cancer therapies aimed at genomic instability, particularly in DNA damage repair. The company's lead candidate, RP-3500, is an oral small molecule inhibitor designed for solid tumors with specific genomic alterations related to DNA damage repair. In addition, Repare is developing innovative programs such as a CCNE1-SL inhibitor for tumors with CCNE1 amplification and a Polymerase Theta program targeting BRCA mutations and other genomic changes. Founded in 2016, Repare Therapeutics is focused on addressing the unique vulnerabilities of tumor cells in genetically defined patient populations to enhance cancer treatment outcomes.