Surveyor Capital

Arbor Biotechnologies, Inc., founded in 2016 and headquartered in Cambridge, Massachusetts, operates as a bio-discovery company in the biotechnology sector. The company focuses on developing genomic tools that enhance human health and sustainability. Utilizing a combination of artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, Arbor's platform accelerates the discovery of proteins and facilitates human diagnostic development. The technology aggregates genomic sequence data through AI and machine learning, enabling drug developers to replace entire genes and precisely correct mutations. This approach aims to streamline the development of curative treatments for previously incurable genetic diseases, ultimately improving the lives of patients affected by such conditions.

35Pharma is a biopharmaceutical company focused on designing and developing innovative biologics to address diseases with significant unmet medical needs, particularly pulmonary hypertension and musculoskeletal disorders. The company leverages its expertise in protein engineering and a comprehensive understanding of transforming growth factor (TGF)-beta structure-function relationships to create highly effective ligand traps. These advancements aim to facilitate the development of targeted biologics for treating patients suffering from cardio-pulmonary and metabolic diseases.

ADCendo ApS is a biotechnology company focused on developing innovative anti-cancer drugs through the use of antibody-drug conjugates (ADCs). The company targets novel receptors that are overexpressed in various cancer types, addressing significant unmet medical needs in the field of oncology. Its ADCs aim to provide targeted therapy for patients suffering from aggressive cancers such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, as well as other carcinomas including those of the breast, colon, pancreas, prostate, ovary, and kidney. Through its research and development efforts, ADCendo strives to create effective treatment options for patients facing challenging cancer diagnoses.

Avenzo Therapeutics is a clinical-stage company focused on developing innovative oncology therapies aimed at treating cancer. The company’s platform is dedicated to creating novel treatments specifically for underserved cancers, including rare and genomically defined types. By advancing a pipeline of targeted drug candidates, Avenzo Therapeutics seeks to enhance treatment options for both researchers and patients, addressing significant gaps in current cancer care.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

Cartesian Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in the United States that specializes in developing innovative cell and gene immunotherapies for the treatment of cancer and autoimmune diseases. The company focuses on chimeric antigen receptor therapies to address multiple myeloma and other forms of cancer. Cartesian Therapeutics employs its proprietary ImmTOR immune tolerance platform, which aims to foster antigen-specific immunity while reducing unwanted immune responses. This approach utilizes a manufacturing platform to introduce mRNA molecules into cells, enhancing their functionality without the risk of integration into the cell's genetic material, as mRNA naturally degrades over time.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Obsidian Therapeutics develops innovative cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Founded in 2015 and based in Cambridge, Massachusetts, the company focuses on creating next-generation therapies that utilize pharmacologic operating systems to provide precise control over protein activity within cells. This technology allows for the development of adoptive cell therapies with advanced functionalities that can be managed by physicians using simple, safe, and orally active medications. By offering improved control over treatment, Obsidian Therapeutics aims to provide better outcomes for patients compared to existing cell therapy options.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

ProfoundBio is a clinical-stage biotechnology company dedicated to creating innovative antibody-based therapeutics aimed at treating cancer. Utilizing proprietary technology platforms, the company has developed a diverse pipeline of antibody-drug conjugate (ADC) candidates targeting both solid tumors and hematological malignancies. These drug candidates are currently in various stages of discovery, preclinical, and clinical development. ProfoundBio's focus is on harnessing the immune system to provide patients with more effective treatments that have the potential for curative outcomes.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of innovative therapies aimed at degrading disease-causing proteins. The company's lead product candidates include ARV-110, a proteolysis targeting chimera (PROTAC) currently in phase I clinical trials for metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. In addition to these, Arvinas is developing various other PROTACs aimed at degrading clinically relevant androgen receptor mutations and treatments for neurodegenerative diseases, including tauopathies. The company has established collaborations with prominent pharmaceutical firms such as Pfizer Inc., Genentech, and Bayer AG, enhancing its research capabilities and reach within the biopharmaceutical landscape. Founded in 2015, Arvinas aims to improve patient outcomes by eliminating harmful proteins from the body through its proprietary technology.

VYNE Therapeutics Inc. is a biopharmaceutical company specializing in the development and commercialization of innovative therapies for dermatological conditions. It offers AMZEEQ, a topical minocycline treatment for non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older. The company is advancing its pipeline with FMX103, currently in Phase III clinical trials for moderate-to-severe papulopustular rosacea in adults, and FCD105, a topical combination foam under investigation in Phase II trials for moderate-to-severe acne vulgaris. Additionally, VYNE is developing Serlopitant, an oral NK1 receptor antagonist aimed at addressing pruritus associated with prurigo nodularis, as well as exploring therapies for other immuno-inflammatory conditions. Formerly known as Menlo Therapeutics Inc., the company rebranded in September 2020 and is headquartered in Bridgewater, New Jersey.

Harpoon Therapeutics is a clinical-stage immunotherapy company focused on developing innovative T cell engagers to harness the body's immune system for treating cancer and other diseases. Utilizing its proprietary TriTAC platform, the company is working on a pipeline of engineered proteins designed to direct T cells to target and eliminate specific cancer cells. The lead product candidate, HPN424, is currently undergoing Phase I clinical trials for metastatic castration-resistant prostate cancer. Additionally, Harpoon is developing HPN536, which is in Phase I/IIa trials for ovarian cancer and other tumors expressing MSLN, as well as HPN217 for multiple myeloma and HPN328 for small cell lung cancer. Founded in 2015 and headquartered in South San Francisco, California, Harpoon Therapeutics has established a collaboration agreement with AbbVie Biotechnology Ltd to further its research efforts.

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.

Mariana Oncology is a biotechnology company focused on developing radiopharmaceuticals designed to target cancer cells using radioactive drugs. The company leverages expertise in ligand discovery, radiochemistry, and radiobiology, along with oncology translational and clinical research, to create a diverse pipeline aimed at treating various solid tumor types. By advancing the use of radiomedicines, Mariana Oncology seeks to enhance treatment options for medical professionals in their fight against cancer.

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

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.

AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.

ElevateBio, LLC is a holding company based in Cambridge, Massachusetts, founded in 2017. It focuses on developing cell and gene therapies for the treatment of cancer and viral infections. The company operates a portfolio of subsidiaries that aim to manufacture and commercialize innovative medicines. ElevateBio collaborates with leading scientists and inventors to create a robust portfolio of therapeutic solutions. Additionally, it has established a centralized facility designed to streamline the translation of cell and gene therapy research into viable medical products, ensuring that patients have access to treatments for severe diseases in a timely manner.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Karyopharm Therapeutics Inc., a pharmaceutical company based in Newton, Massachusetts, specializes in the discovery, development, and commercialization of drugs targeting nuclear transport and related pathways for cancer treatment. Its lead product, XPOVIO (selinexor), is an approved therapy for multiple myeloma, both as a standalone treatment and in combination with other drugs. The company's pipeline includes several clinical-stage compounds, such as BOSTON, STORM, STOMP, and SADAL, which are being evaluated in various blood cancers. Additionally, Karyopharm has collaborations with other organizations for COVID-19 and neurological disease research.

VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in the discovery, development, and commercialization of innovative treatments for severe rare diseases, particularly those with significant unmet medical needs. Its lead product candidate, Apraglutide, is a synthetic GLP-2 analog designed to enhance the intestine’s ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support in patients with short bowel syndrome. VectivBio is dedicated to developing best-in-disease therapies that aim to meaningfully improve the lives of patients and their families, providing more than just incremental advancements over existing standard-of-care options.

Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company focuses on developing antibody-based therapeutics aimed at treating various conditions linked to dysregulation of the T-helper type 2 immune response, such as allergic diseases and chronic inflammation. Its lead product, antolimab (AK002), targets conditions including eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets Siglec-6, an inhibitory receptor found on mast cells. By binding to Siglec-6, AK006 is designed to enhance the receptor's natural inhibitory function, thereby reducing mast cell activation and associated inflammatory responses.

Acelyrin is a late-stage clinical biopharmaceutical company dedicated to advancing new treatment options for patients. The company specializes in identifying, acquiring, and accelerating the development and commercialization of promising drug candidates. By leveraging its expertise, Acelyrin aims to rapidly bring transformative medicines to market, ultimately improving patient outcomes and quality of life.

Triana Biomedicines is focused on discovering and developing molecular glues, a novel therapeutic approach aimed at regulating challenging disease targets. The company employs a sophisticated drug discovery engine that combines high-resolution structural insights, advanced in silico tools, and custom chemical libraries. This platform is designed to stabilize existing protein interactions or create new ones, thereby influencing the functionality of disease targets. Triana has successfully validated its technology and is currently advancing multiple research programs across various disease areas, positioning itself to contribute significantly to therapeutic innovation.

MoonLake Immunotherapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for immunologic diseases. The company specializes in leveraging Nanobody technology to create new treatments aimed at addressing inflammatory conditions, particularly in the skin and joints. Its lead investigational product, Sonelokimab, represents a novel approach to managing inflammation, with the potential to significantly improve patient outcomes. Through its research and development efforts, MoonLake aims to advance the understanding and treatment of various inflammatory diseases, contributing to the evolving landscape of biopharmaceuticals.

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.

AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.

AmbAgon Therapeutics is a biotechnology company focused on developing small-molecule therapeutics for cancer treatment. The company specializes in research aimed at stabilizing the interactions of oncogenic proteins, thereby enhancing the activity of tumor-suppressing proteins. By targeting these proteins, AmbAgon Therapeutics seeks to address significant unmet clinical needs in oncology that conventional treatment methods have struggled to meet. Founded by Christian Ottmann, Lucas Brunsveld, and Michelle Arkin, the company aims to provide innovative solutions in cancer care through its unique approach to drug development.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Acelyrin is a late-stage clinical biopharmaceutical company dedicated to advancing new treatment options for patients. The company specializes in identifying, acquiring, and accelerating the development and commercialization of promising drug candidates. By leveraging its expertise, Acelyrin aims to rapidly bring transformative medicines to market, ultimately improving patient outcomes and quality of life.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Arbor Biotechnologies, Inc., founded in 2016 and headquartered in Cambridge, Massachusetts, operates as a bio-discovery company in the biotechnology sector. The company focuses on developing genomic tools that enhance human health and sustainability. Utilizing a combination of artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, Arbor's platform accelerates the discovery of proteins and facilitates human diagnostic development. The technology aggregates genomic sequence data through AI and machine learning, enabling drug developers to replace entire genes and precisely correct mutations. This approach aims to streamline the development of curative treatments for previously incurable genetic diseases, ultimately improving the lives of patients affected by such conditions.

Structure Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel oral therapeutics for chronic diseases with significant unmet medical needs. Utilizing advanced computational and structure-based technology, the company designs orally available small molecules that aim to overcome the limitations of traditional biologic and peptide drugs. Its lead product candidate, GSBR-1290, is a small molecule agonist targeting the glucagon-like peptide-1 receptor, which is relevant for treating type-2 diabetes and obesity. Additionally, Structure Therapeutics is advancing other oral therapeutics targeting G-protein-coupled receptors for conditions such as pulmonary and cardiovascular diseases, including candidates ANPA-0073 and LTSE-2578. The company combines expertise in drug design and development to create differentiated and effective treatments that can have a profound impact on patient care.

Neumora Therapeutics operates as a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience.

Science 37, Inc. is a technology-enabled clinical trial company that specializes in developing patient-centric models for clinical research, aimed at accelerating biomedical discovery. The company utilizes its innovative platform, NORA (Network Oriented Research Assistant), a cloud-based mobile research tool that facilitates communication between researchers, patients, and mobile nurses through videos, surveys, and photographs. By providing decentralized clinical trial services, Science 37 enables access to diverse patient populations that traditional site-based research may overlook. The company has established itself as a leader in conducting virtual trials, leveraging an extensive network of telemedicine investigators and home-health nurses to enhance patient enrollment and retention. Serving a wide range of clients, including pharmaceutical sponsors, biotech firms, and academic institutions, Science 37 aims to transform clinical research through its comprehensive, fully integrated trial platform. Founded in 2014 and headquartered in Los Angeles, California, Science 37 continues to innovate in the field of clinical research.

Jasper Therapeutics, Inc. is a clinical-stage biotechnology company based in Menlo Park, California, focused on developing therapeutic agents for hematopoietic stem cell transplants and gene therapies. The company aims to create safer conditioning agents that enable broader use of curative therapies, particularly through its novel engineered hematopoietic stem cells platform. Its lead compound, JSP191, is a humanized monoclonal antibody currently in clinical development. This agent functions as a conditioning treatment by targeting and clearing hematopoietic stem cells from bone marrow, facilitating successful stem cell transplants. By addressing the limitations of transplant grafts, Jasper Therapeutics seeks to increase the number of patients who can benefit from both allogeneic and autologous gene-edited hematopoietic stem cell therapies.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

Obsidian Therapeutics develops innovative cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Founded in 2015 and based in Cambridge, Massachusetts, the company focuses on creating next-generation therapies that utilize pharmacologic operating systems to provide precise control over protein activity within cells. This technology allows for the development of adoptive cell therapies with advanced functionalities that can be managed by physicians using simple, safe, and orally active medications. By offering improved control over treatment, Obsidian Therapeutics aims to provide better outcomes for patients compared to existing cell therapy options.

Vigil Neuroscience Inc. is a therapeutics company focused on microglia, the brain's immune sentinel cells, to address both rare and common neurodegenerative diseases. The company aims to restore the vigilance of microglia to improve patient outcomes. Vigil is advancing its lead candidate, a monoclonal antibody TREM2 agonist, through Phase 1 studies and is also developing an orally available small molecule TREM2 agonist, VG-3927, which is positioned to enter Investigational New Drug (IND) application. Additionally, Vigil is conducting the IGNITE trial, a Phase 2 proof-of-concept study that represents the first interventional trial for patients with adult-onset leukodystrophy. By leveraging modern neuroscience and various therapeutic modalities, Vigil seeks to deliver precision therapies that enhance the lives of patients and their families while expanding its pipeline and supporting the understanding of microglia biology as a critical therapeutic target.

Turnstone Biologics Inc. is a clinical-stage biotechnology company based in Ottawa, Canada, with an additional office in New York. Founded in 2015, the company is dedicated to advancing innovative viral immunotherapies aimed at improving cancer patient survival. Its lead product, RIVAL-01, utilizes a vaccinia virus backbone that encodes several immunomodulators, including Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine, which work together to enhance immune activity and optimize the tumor microenvironment for effective cancer treatment. Additionally, Turnstone Biologics is developing next-generation tumor-infiltrating lymphocyte (TIL) therapies by isolating and expanding the most potent and tumor-reactive T cells from patients' tumors, thereby harnessing the body's own immune system to combat solid tumors. Through these innovative approaches, Turnstone Biologics aims to deliver breakthrough cancer immunotherapies that significantly improve clinical outcomes for patients.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Point Biopharma Global Inc., established in 2019 and headquartered in Toronto, Canada, specializes in the development and commercialization of radioligand therapies for cancer treatment. The company leverages a portfolio of advanced radiopharmaceutical assets, expertise in radioisotopes like Actinium-225 and Lutetium-177, and innovative manufacturing technology. Point Biopharma Global's pipeline includes several promising candidates, such as PNT2002, PNT2004, PNT2003, and PNT2001, aiming to revolutionize theragnostic drug development and radioligand commercialization.

NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.

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

BioTheryX, Inc. is a clinical-stage biopharmaceutical company based in Chappaqua, New York, focused on developing innovative therapies that restore protein homeostasis through targeted protein degradation. The company specializes in Protein Homeostatic Modulators and small molecule targeted protein degraders (TPDs), which utilize a unique mechanism involving molecular glues to regulate protein levels within the body. Their proprietary PRODEGY platform is built on a library of Cereblon binders, enabling the design of bifunctional and hybrid degraders aimed at treating various diseases, particularly cancer. BioTheryX's lead candidate, BTX-A51, is an oral small molecule and multi-kinase inhibitor targeting specific leukemic stem cells and oncogenic transcription factors. Established in 2007, BioTheryX aims to address significant unmet medical needs by leveraging its expertise in protein modulation and degradation.

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 is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at generating immune responses to combat tumors while preserving healthy tissue. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux targets all three stages of the anti-tumor immune response. The company's approach is designed to enhance the efficacy of cancer treatments by specifically engaging the immune system to identify and eliminate cancer cells.

Arcellx is a clinical-stage biotechnology company focused on developing innovative cell therapies for patients with cancer and other incurable diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Its lead product candidate, CART-ddBCMA, targets relapsed or refractory multiple myeloma and is currently undergoing a Phase 1 clinical study. This therapy has received multiple designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is advancing its controllable CAR-T therapy, ARC-SparX, through two clinical programs aimed at treating relapsed or refractory multiple myeloma and acute myeloid leukemia.

GH Research PLC is a clinical-stage biopharmaceutical company that aims to transform the treatment of psychiatric and neurological disorders. The company is primarily focused on developing novel and proprietary therapies based on 5-MeO-DMT, a compound known as Mebufotenin, to address the needs of patients suffering from Treatment-Resistant Depression (TRD). Through its innovative approach, GH Research seeks to provide new therapeutic options for individuals who have not responded to conventional treatments.

ValenzaBio is focused on developing safe and effective treatment options for patients suffering from autoimmune and inflammatory diseases. The company utilizes biomarker-driven approaches to inform its research and clinical development. Its pipeline features several monoclonal antibodies designed to target surface antigens, aiming to reduce harmful subpopulations of autoreactive cells. ValenzaBio's most advanced program has successfully reached Phase 1 proof-of-concept in patients, while other preclinical programs are underway, exploring differentiated mechanisms of action. The company's mission is to address the needs of patients who lack effective treatment alternatives.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Pyxis Oncology, established in 2019 in Boston, Massachusetts, is a biotechnology company specializing in the development of antibody therapeutics for cancer treatment. The company focuses on promoting the body's immune response to cancer by analyzing tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and tumor cell signaling pathways within cold tumors. Pyxis aims to create novel antibody-based immunotherapies to directly kill tumor cells and address underlying pathologies that enable cancer proliferation and immune evasion, with the goal of improving patient outcomes for difficult-to-treat cancers.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

ElevateBio, LLC is a holding company based in Cambridge, Massachusetts, founded in 2017. It focuses on developing cell and gene therapies for the treatment of cancer and viral infections. The company operates a portfolio of subsidiaries that aim to manufacture and commercialize innovative medicines. ElevateBio collaborates with leading scientists and inventors to create a robust portfolio of therapeutic solutions. Additionally, it has established a centralized facility designed to streamline the translation of cell and gene therapy research into viable medical products, ensuring that patients have access to treatments for severe diseases in a timely manner.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

Satsuma Pharmaceuticals 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 features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

ASLAN Pharmaceuticals, established in 2010 and based in Singapore, is a clinical-stage biopharmaceutical company focused on immunology and oncology. It develops innovative treatments, including a monoclonal therapy for atopic dermatitis and a small molecule inhibitor for cancer, aiming to transform patients' lives. The company collaborates with prominent pharmaceutical partners and operates as a subsidiary of ASLAN Pharmaceuticals Limited.

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.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

Vera Therapeutics, a biotechnology company based in South San Francisco, California, specializes in developing innovative biologic therapeutics aimed at treating serious immunological diseases and genetic disorders. Founded in 2016 and renamed in March 2020, the company's research focuses on transformative treatments that can significantly improve patient outcomes. One of its key product candidates is atacicept, a fusion protein designed for self-administration via a weekly subcutaneous injection. Atacicept works by inhibiting both B lymphocyte stimulator and a proliferation-inducing ligand, which are involved in the production of autoantibodies that contribute to various autoimmune diseases. Additionally, Vera Therapeutics is exploring gene-editing technologies that hold the potential to address conditions such as sickle cell disease and cystic fibrosis.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, focused on developing innovative vaccines using its proprietary Multiple Antigen Presenting System (MAPS) technology. This approach allows for the combination of protective polysaccharides and proteins into a single vaccine, thereby enhancing immune responses against a variety of infectious diseases affecting both children and adults. Affinivax targets several significant pathogens, including Streptococcus pneumoniae, bacterial nosocomial infections, Salmonella typhi, and Staphylococcus aureus. The company aims to create vaccines that not only provide broader disease coverage compared to existing options but also address infectious diseases lacking effective immunization strategies. Founded in 2014, Affinivax benefits from the expertise of leading professionals in infectious diseases and vaccines, and it has received backing from the Bill & Melinda Gates Foundation, along with an exclusive license from Boston Children’s Hospital for its MAPS technology.

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.

Immuneering Corporation is a biopharmaceutical company specializing in genetic, genomic, and proteomic data analysis services for pharmaceutical companies. It focuses on generating biological insights that enhance the clinical and commercial success of medicines, particularly for patients with cancer, autoimmune diseases, and neurodegenerative conditions. Immuneering employs advanced methodologies such as microarray analysis, single-nucleotide polymorphism assessment, and next-generation sequencing to uncover difficult-to-find signals and formulate biological hypotheses. The company has over a decade of experience in translational bioinformatics, which informs its drug discovery programs and supports patient treatment response analysis. Immuneering's proprietary computational platform, known as Disease Cancelling Technology, underpins its capabilities, allowing for a comprehensive approach to drug development. Founded in 2008 and headquartered in Cambridge, Massachusetts, with an additional office in New York City, Immuneering operates as a subsidiary of Teva Pharmaceutical Industries Limited.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, that specializes in developing small molecule therapies for severe, rare musculoskeletal diseases. Founded in 2017, the company employs a precision medicine approach to create innovative treatments targeting various muscle disorders, including Duchenne and Becker muscular dystrophies, as well as limb girdle muscular dystrophies. Utilizing its proprietary muscle-focused drug discovery platform, Edgewise integrates expertise in muscle biology and small molecule engineering to identify and develop therapies that address genetically defined muscle disorders. The company emphasizes a holistic drug discovery method that assesses integrated muscle function, enabling it to create orally bioavailable therapies aimed at improving outcomes for patients with significant unmet medical needs in neuromuscular and cardiac diseases.

Nuvation Bio Inc. is a biopharmaceutical company dedicated to addressing unmet needs in oncology through the development of novel therapies for challenging cancers. Founded in 2018 and headquartered in New York, with an additional office in San Francisco, Nuvation Bio focuses on treating patients whose conditions have not responded to conventional treatments. The company's leading clinical-stage candidate, NUV-868, is a BD2-selective oral small molecule BET inhibitor that targets the BRD4 protein, which plays a crucial role in regulating tumor growth and differentiation. In addition to NUV-868, Nuvation Bio is advancing a proprietary small molecule Drug-Drug Conjugate platform aimed at enhancing therapeutic efficacy. The company’s comprehensive portfolio includes multiple drug development candidates designed to meet the specific needs of cancer patients.

VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in the discovery, development, and commercialization of innovative treatments for severe rare diseases, particularly those with significant unmet medical needs. Its lead product candidate, Apraglutide, is a synthetic GLP-2 analog designed to enhance the intestine’s ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support in patients with short bowel syndrome. VectivBio is dedicated to developing best-in-disease therapies that aim to meaningfully improve the lives of patients and their families, providing more than just incremental advancements over existing standard-of-care options.

Rezolute Inc., established in 2010 and headquartered in Redwood City, California, is a clinical-stage biopharmaceutical company focused on developing therapies for rare and metabolic diseases. The company's primary product candidate, RZ358, is a human monoclonal antibody currently in Phase 2b trials for treating congenital hyperinsulinism, an ultra-rare pediatric genetic disorder. Additionally, Rezolute is advancing RZ402, a small molecule plasma kallikrein inhibitor in preclinical stages for diabetic macular edema treatment. The company aims to improve existing standards of care by applying proprietary formulation and manufacturing capabilities to known molecules.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

Flame Biosciences, Inc. is a clinical-stage biotechnology company based in New York, dedicated to the research, development, and commercialization of innovative therapies targeting cancer and inflammatory diseases. The company is focused on creating transformative treatments, including FL-101, which addresses lung cancers associated with inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Established in 2017, Flame Biosciences seeks to improve the quality of life for individuals suffering from debilitating and life-threatening conditions through its advanced therapeutic solutions.

BioShin Limited is a clinical-stage biopharmaceutical company based in Pudong, China, focused on developing innovative medicines and therapies for neurological, neuropsychiatric diseases, and rare disorders. Established in 2018, it operates as a wholly owned subsidiary of Biohaven Pharmaceuticals. The company specializes in late-stage product candidates and utilizes clinical trial platforms to explore treatments such as calcitonin gene-related peptide (CGRP) receptor antagonism for migraine and pain, glutamate modulation for Alzheimer’s disease and anxiety disorder, and myeloperoxidase (MPO) inhibition for inflammation and neurodegeneration. BioShin aims to address unmet medical needs in the neurological field, particularly within the Asia-Pacific market.

Palleon Pharmaceuticals is a biotechnology company that develops therapeutics targeting glycan-mediated immune regulation for the treatment of cancer and inflammatory diseases. Founded in 2015 and based in Waltham, Massachusetts, the company specializes in creating glycoimmune checkpoint inhibitors, a novel class of drugs designed to enhance immune response against cancer. By integrating insights from global leaders in glycoscience and human immunology, Palleon aims to provide a broader array of combination therapies that not only address cancer but also combat resistance to traditional immuno-oncology treatments. Additionally, the company develops therapies for inflammatory diseases, including autoimmunity and fibrosis.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Kinnate Biopharma Inc. is a biopharmaceutical company based in San Diego, California, focused on developing small molecule kinase inhibitors for the treatment of genomically defined cancers. Founded in 2018, the company aims to create precision oncology therapeutics to address the needs of underserved patient populations. 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 working on KIN003, which targets specific alterations in the FGFR2 and FGFR3 genes, and has other small molecule research programs, including a Cyclin-Dependent Kinase 12 inhibitor. Kinnate Biopharma employs expertise in structure-based drug discovery and translational research to advance its pipeline of candidates aimed at treating cancers associated with specific oncogenic alterations.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Chinook Therapeutics, Inc. is a clinical-stage biotechnology company based in Seattle, Washington, that specializes in the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead program is atrasentan, an investigational endothelin receptor antagonist currently prepared for Phase III trials targeting IgA nephropathy and other primary glomerular diseases. In addition to atrasentan, Chinook is developing BION-1301, an investigational anti-APRIL monoclonal antibody evaluated in a Phase Ib trial for IgA nephropathy, and CHK-336, a preclinical candidate aimed at treating an undisclosed ultra-orphan kidney disease. Chinook Therapeutics is also engaged in research programs addressing other rare and severe chronic kidney conditions, including polycystic kidney disease.

Dyne Therapeutics 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 dystrophy, among others. Utilizing its innovative FORCE platform, Dyne Therapeutics aims to deliver disease-modifying therapies with enhanced precision to skeletal, cardiac, and smooth muscle tissues. The company's lead product candidates, DYNE-101 and DYNE-251, are currently undergoing phase 1/2 clinical trials, reflecting its commitment to addressing the unmet medical needs of patients with serious muscle disorders.

Aerovate Therapeutics is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative treatments for patients with rare cardiopulmonary diseases. The company's primary focus is on AV-101, a proprietary inhaled dry powder formulation of the drug imatinib, designed specifically for the treatment of pulmonary arterial hypertension (PAH). This formulation enables targeted delivery of the medication directly to affected lung tissues, thereby minimizing systemic side effects commonly associated with traditional treatments. Established in 2018, Aerovate aims to provide meaningful improvements in the lives of those suffering from these serious conditions.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Founded in 2018 and headquartered in Cambridge, Massachusetts, the company is advancing a diverse pipeline that includes five clinical-stage investigational therapies and several preclinical compounds targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Key products under development include CVL-231, a positive allosteric modulator for schizophrenia; CVL-865, which is being tested for drug-resistant focal onset epilepsy and acute anxiety; and Tavapadon, a selective dopamine D1/D5 partial agonist for Parkinson's disease. Cerevel Therapeutics employs a unique approach that leverages expertise in neurocircuitry and receptor selectivity to address unmet medical needs in the field of neuroscience.

Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders marked by neuronal imbalance. Established in 2015 and headquartered in Cambridge, Massachusetts, the company leverages genetic insights to create innovative treatments for neurological and psychiatric conditions. Its leading product candidates include PRAX-114, an allosteric modulator targeting GABAA receptors, currently undergoing Phase IIa trials for major depressive disorder and perimenopausal depression, and PRAX-944, a selective inhibitor of T-type calcium channels in Phase IIa trials for essential tremor. Additionally, Praxis is advancing PRAX-562, a sodium current blocker in Phase I trials for severe pediatric epilepsy and adult cephalgia, along with PRAX-222, an antisense oligonucleotide aimed at treating patients with SCN2A epilepsy, and a program focused on KCNT1-related epilepsy. The company has established collaborations and licensing agreements with various organizations to enhance its research and development efforts.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various forms of cancer, including hematological cancers and solid tumors. The company's primary area of research centers on targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), a cell-surface protein found in numerous tumors but absent in normal tissues, which makes it a promising target for cancer therapy. VelosBio's lead product, VLS-101, is a ROR1-directed ADC that has shown positive preclinical results and aims to provide new treatment options for patients. Founded in 2017, VelosBio operates under the umbrella of Merck & Co., Inc. as of December 2020, focusing on developing targeted therapies that can be administered alone or in combination with other treatments across a wide range of cancer types.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for patients suffering from classical complement-mediated disorders affecting the body, brain, and eye. The company's research is centered around its platform technology that targets classical complement-mediated autoimmune and neurodegenerative diseases associated with the abnormal activation of C1q, a key molecule in the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody intended for intravenous use in treating autoimmune and neurodegenerative disorders. Another candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration aimed at neurodegenerative ophthalmic conditions. The company employs a disciplined, biomarker-driven development strategy to ensure its therapies effectively engage the target at tolerable doses within the intended patient tissues, while also exploring additional indications within both orphan and larger market segments.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and addressing neurodegenerative diseases. The company's lead candidate, ATH-1017, is a small hepatocyte growth factor/MET activator designed to penetrate the blood-brain barrier and is currently undergoing various clinical trials for the treatment of Alzheimer's and Parkinson's diseases. Additionally, Athira is advancing other candidates, including ATH-1019, which is in preclinical development for depression, and ATH-1018, aimed at treating peripheral neuropathy. Founded in 2011 and originally named M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its commitment to innovative therapies that positively impact individuals affected by brain disorders.

Mereo Biopharma, established in London in 2015, specializes in acquiring and advancing late-stage drug candidates from larger pharmaceutical companies. These candidates, often overlooked due to resource constraints, target rare and specialized diseases with significant unmet medical needs. Mereo's strategy involves rapidly progressing these assets through clinical development, either by partnering or divesting them, or directly commercializing them, particularly in orphan disease indications. The company combines operational efficiency with financial resources, leveraging a unique partnership with a leading global CRO, ICON, to conduct comprehensive clinical studies. Mereo's focus is on transforming the lives of patients worldwide by developing innovative medicines that can significantly improve their quality of life.

ElevateBio, LLC is a holding company based in Cambridge, Massachusetts, founded in 2017. It focuses on developing cell and gene therapies for the treatment of cancer and viral infections. The company operates a portfolio of subsidiaries that aim to manufacture and commercialize innovative medicines. ElevateBio collaborates with leading scientists and inventors to create a robust portfolio of therapeutic solutions. Additionally, it has established a centralized facility designed to streamline the translation of cell and gene therapy research into viable medical products, ensuring that patients have access to treatments for severe diseases in a timely manner.

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

Spruce Biosciences, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for rare endocrine disorders, particularly those affecting the hypothalamic-pituitary-adrenal axis. Founded in 2014 and headquartered in Daly City, California, the company is advancing its lead product candidate, tildacerfont, which is currently undergoing Phase IIb clinical trials for adult patients and Phase II clinical trials for children with classic congenital adrenal hyperplasia (CAH). Tildacerfont is positioned as a potential first non-steroidal therapy that aims to improve disease control and reduce the reliance on steroids for patients with CAH. Additionally, Spruce is exploring the use of tildacerfont for women with polycystic ovary syndrome. The company has also entered into a licensing agreement with Eli Lilly and Company to research and develop compounds for various pharmaceutical applications.

Zentalis Pharmaceuticals is a clinical-stage biopharmaceutical company based in New York, dedicated to developing small molecule therapeutics aimed at treating various cancers. Founded in 2014, the company focuses on discovering and advancing drug candidates that target critical biological pathways in cancer. Its lead product candidate, ZN-c5, is an oral selective estrogen receptor degrader currently undergoing Phase I/II clinical trials for advanced or metastatic breast cancer. In addition, Zentalis is developing ZN-c3, an inhibitor of the WEE1 protein tyrosine kinase, for advanced solid tumors; ZN-d5, a selective B-cell lymphoma 2 inhibitor for hematological malignancies; and ZN-e4, an irreversible inhibitor of mutant epidermal growth factor receptor for advanced non-small cell lung cancer, all of which are also in Phase I/II clinical trials. The company utilizes an integrated discovery engine to identify new chemical entities with the potential for differentiated therapeutic profiles.

Jasper Therapeutics, Inc. is a clinical-stage biotechnology company based in Menlo Park, California, focused on developing therapeutic agents for hematopoietic stem cell transplants and gene therapies. The company aims to create safer conditioning agents that enable broader use of curative therapies, particularly through its novel engineered hematopoietic stem cells platform. Its lead compound, JSP191, is a humanized monoclonal antibody currently in clinical development. This agent functions as a conditioning treatment by targeting and clearing hematopoietic stem cells from bone marrow, facilitating successful stem cell transplants. By addressing the limitations of transplant grafts, Jasper Therapeutics seeks to increase the number of patients who can benefit from both allogeneic and autologous gene-edited hematopoietic stem cell therapies.

Pieris Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on the discovery and development of anticalin-based drugs. Anticalins are engineered proteins derived from naturally occurring lipocalins, which have the ability to bind and transport a variety of molecules. The company's clinical pipeline includes PRS-060, a drug candidate in Phase I trials for asthma and inflammatory diseases, and PRS-343, a bispecific protein also in Phase I trials for oncology. Additionally, Pieris is advancing PRS-344, a bispecific anticalin-antibody fusion protein in preclinical development, and PRS-080, which targets hepcidin for treating iron deficiency in patients with chronic kidney disease, currently in Phase IIa trials. Pieris has established strategic partnerships with several organizations, including Les Laboratoires Servier and AstraZeneca, and engages in research collaborations with academic institutions such as the University of Pittsburgh. The company was incorporated in 2000 and aims to address high unmet medical needs across various therapeutic areas, including cancer and severe asthma.

Satsuma Pharmaceuticals 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 features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.