Wellington Management

Atsena Therapeutics is a clinical-stage gene therapy company dedicated to developing innovative treatments for inherited retinal diseases. The company focuses on therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Utilizing an advanced adeno-associated virus (AAV) technology platform, including a novel spreading capsid, Atsena aims to target specific retinal conditions to prevent blindness resulting from genetic mutations. Their research is centered on providing effective ocular gene therapies that address significant challenges associated with inherited retinal diseases, with the goal of reversing or halting vision loss.

MedinCell S.A. is a pharmaceutical company focused on developing innovative therapeutic solutions through its proprietary BEPO technology, which facilitates long-acting injectable products utilizing biodegradable polymers and a biocompatible solvent. Based in Jacou, France, the company is engaged in ongoing research and development to optimize the efficiency and accessibility of medical treatments across various therapeutic areas. MedinCell's product pipeline includes several candidates, such as mdc-IRM, a subcutaneous injection for schizophrenia currently in Phase III trials, and mdc-CWM, an intra-articular injection aimed at treating pain and inflammation. Additionally, the company is exploring formulations like mdc-WWM for contraception and mdc-ANG for central nervous system disorders, alongside other candidates targeting pain management and organ transplant applications. Through its advancements in controlled, long-acting injectables, MedinCell aims to enhance treatment outcomes for patients.

Helicore Biopharma is a biopharmaceutical company dedicated to developing innovative treatments for obesity and related metabolic disorders. Its portfolio includes novel therapeutics based on glucose-dependent insulinotropic peptide (GIP) antagonism and monoclonal antibodies that bind circulating GIP ligands, forming a modular platform for anti-obesity medicines. The company aims to help patients with obesity and related conditions achieve significant weight loss.

Saluda Medical Pty Ltd. is a medical device company specializing in the development of neuromodulation technologies for chronic neurological conditions. Founded in 2010 and based in Artarmon, Australia, with additional offices in the United States and the United Kingdom, Saluda Medical focuses on its innovative Evoke system. This investigational device employs a closed-loop spinal cord stimulation approach that continuously monitors the spinal cord's response to stimulation and adjusts treatment in real-time to optimize patient outcomes. By utilizing a unique dose-control platform, Saluda Medical aims to customize therapy based on individual neural responses, thereby improving the management of chronic pain, particularly in patients suffering from conditions such as neuropathic pain and chronic back pain.

SiteOne Therapeutics is a San Francisco-based company focused on developing innovative therapeutics and diagnostics aimed at treating acute and chronic pain. The company specializes in creating products that address the limitations of existing pain management therapies, such as nonsteroidal anti-inflammatory drugs (NSAIDs) and opioids. Its lead therapeutic candidates are selective inhibitors of the voltage-gated sodium ion channel Naᵥ1.7, which is essential for the generation and conduction of pain signals. By targeting this specific channel, SiteOne Therapeutics aims to provide more effective and safer pain relief options for healthcare professionals and their patients, particularly in the treatment of neuropathic pain.

nChroma Bio is a biotechnology company specializing in targeted genetic medicine. It operates an integrated product engine for safe, accurate, and targeted in vivo administration, aiming to overcome limitations of current genetic medicine techniques. The company focuses on liver-targeted therapies, initially for chronic hepatitis B and D, leveraging epigenetics to enable precise and specific gene regulation. This approach allows for therapies with programmable tissue specificity, potentially offering durable and targeted cures for the liver and other organs.

Beta Bionics is a biotechnology company focused on developing advanced solutions for diabetes management. Its flagship product, the iLet Bionic Pancreas System, is an integrated device designed to alleviate the burden of diabetes care. This system encompasses a dual-chamber infusion pump that delivers insulin and glucagon with precision and accuracy, autonomously calculating and administering doses to help patients maintain optimal blood sugar levels. The iLet has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal, and it incorporates Zealand Pharma’s dasiglucagon, which is known for its stability in a ready-to-use aqueous solution. Through these innovations, Beta Bionics aims to improve the quality of life for individuals living with diabetes.

Crescent Biopharma is a biotechnology firm focused on developing novel precision-engineered medicines targeting verified biology to improve care for patients with solid tumors.

MBX Biosciences, Inc. is a biotechnology company founded in 2018 and based in Carmel, Indiana. The company specializes in developing therapeutics for rare endocrine disorders, focusing on conditions that currently lack effective treatments. MBX Biosciences aims to discover, develop, and commercialize first-in-class therapies, particularly peptides designed to address endocrine genetic diseases that affect glandular hormone production and the body’s chemical messaging systems.

Scorpion Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, focused on advancing precision oncology for cancer treatment. Founded in 2020, the company specializes in developing targeted small-molecule drugs that address validated cancer targets, as well as previously undruggable and novel targets. Scorpion Therapeutics utilizes an integrated approach that combines target discovery, medicinal chemistry, and translational medicine to create a diverse pipeline of optimized compounds. By leveraging its precision medicine platform, the company aims to enhance the efficacy of cancer therapies and expand treatment options for patients, ultimately striving to overcome the limitations of existing cancer treatments.

Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP, in Phase 2a development for nontuberculous mycobacteria, or NTM, lung infection, and in preparation for Phase 2a development in cystic fibrosis, or CF, affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in CF. Savara’s strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2 ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing innovative CAR T-cell therapies to improve outcomes for cancer patients. The company aims to address the challenges faced by existing autologous CAR therapies, which are only curative for less than half of cancer patients and hindered by issues such as manufacturing limitations, supply challenges, and reimbursement barriers. Cargo Therapeutics is advancing next-generation cell therapies designed to enhance effectiveness, safety, and supply reliability. One of its key products, CRG-022, is a novel CAR T-cell candidate targeting CD22, an antigen present in a majority of B-cell malignancies, specifically designed to overcome treatment resistance. By pursuing these novel solutions, Cargo Therapeutics seeks to facilitate broader access to curative therapies and significantly improve the treatment experience for cancer patients.

Bluejay Therapeutics is a private biopharmaceutical company dedicated to developing curative treatments for infectious diseases, with an initial focus on chronic Hepatitis B. This disease remains prevalent worldwide and has significant unmet medical needs. The company is pursuing two innovative approaches: fully human IgG1 anti-HBs monoclonal antibodies and first-in-class HBsAg oral small molecule inhibitors. These therapies aim to reduce hepatitis B surface antigen levels and restore adaptive immunity, potentially achieving a functional cure for patients.

Zenas BioPharma is a clinical-stage biopharmaceutical company headquartered in Florida, USA, that specializes in developing immune-based therapies for patients. The company’s lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to target both CD19 and FcγRIIb, which are found in B cell lineages. This innovative approach aims to inhibit the activity of cells involved in various autoimmune diseases while preserving their presence, thus avoiding depletion. Through its focus on these advanced therapies, Zenas BioPharma seeks to address unmet medical needs in the treatment of autoimmune conditions.

BridgeBio Oncology Therapeutics is a biopharmaceutical company specializing in the development of precision oncology therapeutics. It focuses on targeting RAS-dependent cancers, leveraging a profound understanding of RAS signaling biology to create drugs that selectively inhibit RAS-driven PI3K activation and a wide spectrum of KRAS mutants. The company aims to provide patients with tailored treatments that address both active and inactive cancer-driving states.

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.

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.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing innovative therapies for idiopathic pulmonary fibrosis (IPF) and other serious respiratory conditions. Headquartered in Seattle, Washington, with an additional office in San Diego, the company specializes in an inhaled formulation of pirfenidone, known as AP01. This formulation aims to enhance the amount of medication that reaches the lungs while minimizing side effects associated with oral treatments. Established in 2011, Avalyn Pharma is dedicated to improving care and outcomes for patients with severe respiratory diseases through its advanced therapeutic pipeline and novel inhaled therapeutics. Initially named Genoa Pharmaceuticals, the company rebranded to its current name in July 2017.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.

Upstream Bio Inc is a clinical-stage biotechnology company dedicated to developing treatments for inflammatory diseases, particularly severe respiratory disorders. The company's primary focus is on verekitug, an antibody therapy designed to inhibit the Thymic Stromal Lymphopoietin receptor. This receptor is a key target in the inflammatory response, influencing various immune cells and signaling pathways involved in both common and rare illnesses. By targeting TSLP, Upstream Bio aims to address the underlying mechanisms of severe asthma and related conditions, positioning itself as a significant player in the field of immunotherapy.

Alkeus Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston that specializes in developing treatments for serious ophthalmic conditions. The company's primary focus is on addressing untreatable diseases of the eye, with its lead drug candidate being an orally delivered compound aimed at treating Stargardt disease and age-related macular degeneration. Through its innovative approach, Alkeus aims to empower medical professionals to manage symptoms of irreversible vision loss at an earlier stage in affected patients.

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.

Saluda Medical Pty Ltd. is a medical device company specializing in the development of neuromodulation technologies for chronic neurological conditions. Founded in 2010 and based in Artarmon, Australia, with additional offices in the United States and the United Kingdom, Saluda Medical focuses on its innovative Evoke system. This investigational device employs a closed-loop spinal cord stimulation approach that continuously monitors the spinal cord's response to stimulation and adjusts treatment in real-time to optimize patient outcomes. By utilizing a unique dose-control platform, Saluda Medical aims to customize therapy based on individual neural responses, thereby improving the management of chronic pain, particularly in patients suffering from conditions such as neuropathic pain and chronic back pain.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing innovative CAR T-cell therapies to improve outcomes for cancer patients. The company aims to address the challenges faced by existing autologous CAR therapies, which are only curative for less than half of cancer patients and hindered by issues such as manufacturing limitations, supply challenges, and reimbursement barriers. Cargo Therapeutics is advancing next-generation cell therapies designed to enhance effectiveness, safety, and supply reliability. One of its key products, CRG-022, is a novel CAR T-cell candidate targeting CD22, an antigen present in a majority of B-cell malignancies, specifically designed to overcome treatment resistance. By pursuing these novel solutions, Cargo Therapeutics seeks to facilitate broader access to curative therapies and significantly improve the treatment experience for cancer patients.

Apogee Therapeutics is a San Francisco-based biotechnology company founded in 2022, specializing in the development of novel therapies for immunological and inflammatory disorders. The company focuses on creating differentiated biologics to treat conditions such as atopic dermatitis and chronic obstructive pulmonary disease, addressing significant unmet medical needs. Apogee's approach involves advancing antibody programs designed to target established mechanisms of action while utilizing advanced engineering techniques to enhance the therapeutic properties, including extended half-life. The company is actively developing two main programs: APG777 for atopic dermatitis and APG808 for chronic obstructive pulmonary disease.

MBX Biosciences, Inc. is a biotechnology company founded in 2018 and based in Carmel, Indiana. The company specializes in developing therapeutics for rare endocrine disorders, focusing on conditions that currently lack effective treatments. MBX Biosciences aims to discover, develop, and commercialize first-in-class therapies, particularly peptides designed to address endocrine genetic diseases that affect glandular hormone production and the body’s chemical messaging systems.

Zenas BioPharma is a clinical-stage biopharmaceutical company headquartered in Florida, USA, that specializes in developing immune-based therapies for patients. The company’s lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to target both CD19 and FcγRIIb, which are found in B cell lineages. This innovative approach aims to inhibit the activity of cells involved in various autoimmune diseases while preserving their presence, thus avoiding depletion. Through its focus on these advanced therapies, Zenas BioPharma seeks to address unmet medical needs in the treatment of autoimmune conditions.

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.

Synthego Corporation is a genome engineering company that specializes in accelerating life science research and development to enhance human health. Established in 2012 and headquartered in Redwood City, California, Synthego utilizes an innovative platform that integrates hardware, software, bioinformatics, chemistries, and molecular biology. This full-stack approach provides researchers with tools for gene editing, including engineered cells, CRISPR kits, and bioinformatics for guide RNA optimization and analysis. The products support various applications in basic research, therapeutic development, disease modeling, and diagnostics. Synthego serves a diverse range of customers, including biotechnology firms, universities, and therapeutic developers worldwide, distributing its offerings online and through partners in multiple countries. By leveraging automation and machine learning, Synthego enables genetic engineers and medical professionals to conduct research that is both rapid and cost-effective.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

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.

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.

Thriveworks is a provider of mental health services focused on addressing a range of psychological issues. The company offers counseling and psychiatric services for conditions such as depression, anxiety, relationship challenges, low self-esteem, and trauma. Thriveworks emphasizes the importance of personalized care, creating customized plans to meet the specific needs of each client. In addition to traditional therapy, the company provides life coaching and marriage counseling, ensuring a comprehensive approach to mental well-being. Through its diverse offerings, Thriveworks aims to support individuals in navigating life's challenges and achieving better mental health.

NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and headquartered in Wilmington, Delaware. The company specializes in the discovery and development of small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. Utilizing a discovery approach informed by target structure biology, NiKang employs structure-based drug design to facilitate the rapid and efficient development of proprietary drug candidates. This methodology enables the company to create innovative therapies with desirable pharmacological properties, ultimately aimed at improving the lives of patients through enhanced treatment options.

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.

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.

Entrada Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, focused on treating diseases through the intracellular delivery of biologics. Founded in 2016, the company has developed innovative technologies that facilitate the efficient delivery of proteins, peptides, and nucleic acids, targeting intracellular sites that have traditionally been difficult to access. Entrada's approach includes Intracellular Enzyme Replacement Therapy (IC-ERT) and Endosomal Escape Vehicle (EEV) therapeutics, which aim to improve the therapeutic index by enabling the delivery of a wide range of therapeutics across various organs and tissues. The company's pipeline addresses conditions related to neuromuscular diseases and expands into additional therapeutic areas, positioning Entrada at the forefront of advancing treatment options for patients with devastating illnesses.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapies utilizing engineered CRISPR technologies to address genetic disorders. Founded in 2018 and based in Berkeley, California, Scribe Therapeutics is known for its proprietary X-Editing (XE) molecules, which are advanced CRISPR enzymes designed to enhance efficacy, specificity, and deliverability compared to existing genome editing methods. The company's platform aims to overcome the limitations of current technologies by creating custom-engineered enzymes and delivery systems that facilitate precise genetic modifications. Scribe Therapeutics is committed to establishing CRISPR-based therapies as a new standard of clinical care, with the potential to significantly impact the treatment landscape for a variety of diseases, including those related to immuno-oncology, degenerative disorders, and enzyme replacement therapy.

Cityblock Health, Inc., established in 2017 and headquartered in Brooklyn, New York, operates a personalized health system tailored to meet the unmet health and social needs of Medicaid and Medicare populations. The company's online platform offers integrated primary care, behavioral health, and virtual care services, along with a tool called Commons for communication, information sharing, and task management. Cityblock's model focuses on upstream investment in highly personalized, prevention-oriented care, aiming to improve outcomes and reduce costs. It partners with community-based organizations to deliver care, enabling clinicians to provide personalized care for each member. During the COVID-19 pandemic, Cityblock developed a model to help identify high-risk members within organizations.

Zenas BioPharma is a clinical-stage biopharmaceutical company headquartered in Florida, USA, that specializes in developing immune-based therapies for patients. The company’s lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to target both CD19 and FcγRIIb, which are found in B cell lineages. This innovative approach aims to inhibit the activity of cells involved in various autoimmune diseases while preserving their presence, thus avoiding depletion. Through its focus on these advanced therapies, Zenas BioPharma seeks to address unmet medical needs in the treatment of autoimmune conditions.

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.

Artiva Biotherapeutics, Inc. is a San Diego-based biotechnology company focused on developing and manufacturing cellular immunotherapies for cancer treatment. Founded in 2019, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating both hematologic cancers and solid tumors. The company's product pipeline includes therapies that target CD20 and CD19 in B-cell lymphomas, as well as HER2 in various solid tumors. Artiva is committed to harnessing the therapeutic potential of NK cells while addressing challenges in their scalability and manufacturing. Its mission centers on delivering effective and safe cell therapies that are readily accessible to cancer patients in need.

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.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

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.

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

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.

Scorpion Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, focused on advancing precision oncology for cancer treatment. Founded in 2020, the company specializes in developing targeted small-molecule drugs that address validated cancer targets, as well as previously undruggable and novel targets. Scorpion Therapeutics utilizes an integrated approach that combines target discovery, medicinal chemistry, and translational medicine to create a diverse pipeline of optimized compounds. By leveraging its precision medicine platform, the company aims to enhance the efficacy of cancer therapies and expand treatment options for patients, ultimately striving to overcome the limitations of existing cancer treatments.

Cityblock Health, Inc., established in 2017 and headquartered in Brooklyn, New York, operates a personalized health system tailored to meet the unmet health and social needs of Medicaid and Medicare populations. The company's online platform offers integrated primary care, behavioral health, and virtual care services, along with a tool called Commons for communication, information sharing, and task management. Cityblock's model focuses on upstream investment in highly personalized, prevention-oriented care, aiming to improve outcomes and reduce costs. It partners with community-based organizations to deliver care, enabling clinicians to provide personalized care for each member. During the COVID-19 pandemic, Cityblock developed a model to help identify high-risk members within organizations.

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.

LianBio is a biopharmaceutical company founded in 2019, headquartered in Shanghai, China, with an additional office in Princeton, New Jersey. The company is dedicated to the discovery and development of therapeutic drugs aimed at treating oncology and cardiorenal diseases, as well as addressing other unmet medical needs in Greater China and major Asian markets. LianBio focuses on in-licensing assets and collaborates with leading partners to enhance its pipeline, which includes clinically validated product candidates across various therapeutic areas such as cardiovascular, oncology, ophthalmology, inflammatory diseases, and respiratory indications. The company aims to accelerate the availability of innovative medicines to improve patient outcomes through a science-driven approach.

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.

Novellus is a biotechnology company working to develop engineered cellular medicines. The company uses its non-immunogenic mRNA molecules to reprogram and gene-edit cells safely, and with high efficiency. They engineer precision medicines for patient segments and select from its technology toolbox for in vivo, ex vivo, allogeneic and autologous delivery. Novellus platform enables the high-level expression of gene-editing and cell-reprogramming proteins for high-specificity gene repair and both autologous and allogeneic cell therapies. The company is advancing its lead program, a breakthrough therapy for a rare dermatologic disease, through pre-clinical studies. Novellus was founded in 2015 and is headquartered in Cambridge, Massachusetts, United States.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.

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.

Synthego Corporation is a genome engineering company that specializes in accelerating life science research and development to enhance human health. Established in 2012 and headquartered in Redwood City, California, Synthego utilizes an innovative platform that integrates hardware, software, bioinformatics, chemistries, and molecular biology. This full-stack approach provides researchers with tools for gene editing, including engineered cells, CRISPR kits, and bioinformatics for guide RNA optimization and analysis. The products support various applications in basic research, therapeutic development, disease modeling, and diagnostics. Synthego serves a diverse range of customers, including biotechnology firms, universities, and therapeutic developers worldwide, distributing its offerings online and through partners in multiple countries. By leveraging automation and machine learning, Synthego enables genetic engineers and medical professionals to conduct research that is both rapid and cost-effective.

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.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on the discovery and development of small molecule therapies targeting p53 mutations in cancer. Its lead candidate, PC14586, aims to correct and restore the function of the p53 protein, which is critical in regulating cell growth and preventing tumor formation. In addition to PC14586, the company is developing therapies for various p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, PMV Pharmaceuticals leverages extensive expertise in p53 biology to advance its innovative treatment options for cancer patients.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various hematological cancers and solid tumors. Established in 2017, the company specializes in therapies that target receptor tyrosine kinase-like orphan receptor 1 (ROR1), a protein present on many cancerous cells but absent in normal tissues, making it a promising target for cancer treatment. VelosBio's lead product, VLS-101, is an ROR1-directed ADC designed for patients with both hematologic and solid tumor malignancies. The company's approach aims to provide novel targeted therapies that can be utilized as monotherapy or in combination with other treatments across a wide array of cancer types.

Freeline Therapeutics, established in 2015 and based in Stevenage, UK, is a clinical-stage biotechnology company specializing in the development of systemic adeno-associated virus (AAV) vector-mediated gene therapies. The company focuses on treating inherited, debilitating diseases by delivering functional copies of therapeutic genes into human liver cells, enabling the production of functional proteins in the bloodstream. Freeline's pipeline includes FLT180a for hemophilia B, FLT190 for Fabry disease, and FLT201 for Gaucher disease Type 1, all in various stages of clinical trials. Additionally, the company offers expertise in CMC process development, analytics, and AAV technologies.

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

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

Kymera Therapeutics, Inc. is a biopharmaceutical company based in Watertown, Massachusetts, specializing in targeted protein degradation. Founded in 2015, the company employs a proprietary platform that utilizes the body's natural protein degradation system to selectively degrade disease-causing proteins. Kymera is advancing several therapeutic programs, including its IRAK4 program aimed at treating immunology-inflammation diseases such as hidradenitis suppurativa, atopic dermatitis, and rheumatoid arthritis. Additionally, it is developing the IRAKIMiD program for MYD88-mutated diffuse large B cell lymphoma and the STAT3 program targeting hematologic malignancies, solid tumors, and autoimmune diseases. By focusing on previously untreatable conditions and using innovative small molecule modalities, Kymera Therapeutics aims to revolutionize drug discovery and create effective treatments for challenging diseases.

Nurix Therapeutics is a biopharmaceutical company dedicated to the discovery, development, and commercialization of small molecule therapies aimed at modulating cellular protein levels for the treatment of cancer and immune disorders. The company has developed an integrated discovery platform known as DELigase, which leverages its expertise in E3 ligases—enzymes capable of modulating proteins within cells. Nurix's pipeline includes promising candidates such as NX-2127, an orally available Bruton’s tyrosine kinase degrader targeting relapsed or refractory B-cell malignancies, and NX-1607, an orally available inhibitor of Casitas B-lineage lymphoma proto-oncogene B, intended for immuno-oncology applications. Founded in 2009 and headquartered in San Francisco, California, Nurix Therapeutics was formerly known as Nurix Inc. and rebranded in October 2018. The company has established strategic collaborations with major industry players, enhancing its research and development capabilities.

Generation Bio Co. is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies aimed at treating both rare and prevalent diseases. Founded in 2016 and formerly known as Torus Therapeutics, the company focuses on creating durable and redosable treatments for conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs a proprietary platform that utilizes closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP) to achieve targeted delivery of genetic materials across various tissues. This approach is designed to enhance treatment efficacy while expanding access to therapies for rare diseases and addressing more common conditions through scalable manufacturing processes.

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.

Forma Therapeutics, headquartered in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and various cancers. The company is advancing several key product candidates, including FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, targeting metastatic castration-resistant prostate cancer. Additionally, Forma is developing FT-2102, a selective small molecule designed to inhibit mutated IDH1 enzymes, which is being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and in exploratory trials for glioma. Other candidates include FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. The firm integrates advanced drug discovery technologies with deep oncology expertise to address challenging targets, contributing to a robust pipeline aimed at transforming patient outcomes in oncology.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

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.

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

Forty Seven, Inc. is a clinical-stage immuno-oncology company headquartered in Menlo Park, California, focused on developing innovative therapies to activate macrophages for cancer treatment. The company’s lead product, magrolimab, is a humanized monoclonal antibody targeting the CD47 receptor, which tumors exploit to evade immune detection. Magrolimab is currently undergoing Phase 1b/2 clinical trials for various cancers, including solid tumors and hematologic malignancies such as acute myeloid leukemia and non-Hodgkin's lymphoma. Additionally, Forty Seven is advancing other antibody-based therapies, including FSI-189, which targets SIRPa, and FSI-174, an anti-cKIT antibody. The company collaborates with Genentech and Acerta Pharma to explore further applications of its therapies in combination with other cancer treatments. Founded in 2014 and previously known as CD47 Sciences, Inc., Forty Seven operates as a subsidiary of Gilead Sciences, Inc.

Nightstar Therapeutics is a clinical-stage gene therapy company based in London, United Kingdom, specializing in the development of innovative one-time treatments for rare inherited retinal diseases. The company is advancing several product candidates, including its lead candidate NSR-REP1, which is in phase 3 clinical development for choroideremia. Additionally, Nightstar is working on NSR-RPGR, currently in phase 2/3 trials for X-linked retinitis pigmentosa, as well as NSR-BEST1. The company is also developing NSR-ABCA4, which is in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address significant unmet medical needs in the field of retinal disorders.

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

Mersana Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative antibody-drug conjugates (ADCs) for cancer patients with unmet medical needs. The company utilizes its proprietary Dolaflexin platform to create a pipeline of novel ADC candidates designed to improve treatment outcomes for those not responding to traditional therapies. Mersana's lead product candidate, XMT-1536, targets NaPi2b and is currently undergoing Phase I clinical trials for ovarian cancer, non-small cell lung cancer, and other orphan indications. In addition to XMT-1536, the company is advancing several early-stage programs, including XMT-1660, which targets B7-H4, and XMT-2056, a STING agonist ADC. Mersana has established strategic partnerships for research and development, notably with Merck KGaA and Asana BioSciences, to enhance its ADC product portfolio. Founded in 2005, Mersana Therapeutics continues to innovate in the field of oncology therapeutics.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Allena Pharmaceuticals, Inc. is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, focused on the development and commercialization of innovative oral enzyme therapeutics for rare and severe metabolic and kidney disorders. Founded in 2011, the company’s lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a condition characterized by elevated urinary oxalate levels that can lead to kidney stones and chronic kidney disease. Additionally, Allena is developing ALLN-346, a novel enzyme intended to address hyperuricemia in patients with moderate to severe chronic kidney diseases. By targeting metabolic disorders associated with the accumulation of harmful metabolites, Allena Pharmaceuticals aims to provide effective treatments for patients facing serious health challenges.

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

CytomX Therapeutics, Inc. is an oncology-focused biopharmaceutical company based in South San Francisco, California. It specializes in developing a novel class of antibody therapeutics utilizing its Probody technology platform, which aims to enhance the targeting of tumors while minimizing drug activity in healthy tissues. The company's clinical-stage product candidates include CX-072, a therapeutic targeting programmed cell death ligand 1; CX-2009, a drug conjugate targeting CD166; BMS-986249 and BMS-986288, both targeting CTLA-4 for metastatic melanoma and solid tumors; and CX-2029, a drug conjugate for CD71 in solid tumors. CytomX collaborates strategically with several industry leaders, including AbbVie, Amgen, and Bristol-Myers Squibb, to advance its Probody therapeutics. Founded in 2008, CytomX Therapeutics is committed to providing innovative and less toxic treatment options for patients with severe illnesses such as cancer.

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).

Jounce Therapeutics, Inc. is a clinical-stage immunotherapy company based in Cambridge, Massachusetts, focused on developing innovative therapies for cancer treatment. The company aims to harness the immune system to target and eliminate cancerous cells, thereby enhancing patient outcomes and quality of life. Among its key product candidates is vopratelimab, a monoclonal antibody currently undergoing Phase II clinical trials for patients with non-small cell lung cancer and urothelial cancer. Jounce is also advancing JTX-4014, an anti-PD-1 antibody for combination therapy, and JTX-1811, which is designed to deplete T regulatory cells in the tumor microenvironment. Additionally, JTX-8064 targets a specific receptor on macrophages to modulate the immune response. Founded in 2013 by experts in immunobiology and cancer biology, Jounce utilizes a proprietary Translational Science Platform to analyze the cellular and molecular characteristics of tumors, driving its commitment to developing first-in-class cancer immunotherapies.

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

Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for underserved patient communities, particularly those affected by blood disorders. The company is advancing its lead product candidate, Oxbryta (voxelotor), which is an oral, once-daily therapy designed for the treatment of sickle cell disease (SCD). Oxbryta has completed Phase III clinical trials in adult and adolescent patients and is currently being evaluated for safety and pharmacokinetics in a Phase IIa trial involving pediatric patients. In addition to its efforts in SCD, Global Blood Therapeutics is engaged in research and development for other blood-related conditions, including hereditary angioedema, and holds licensed rights to its product candidates in multiple regions, including the United States and Europe.

Moderna, Inc. is a biotechnology company established in 2010 and headquartered in Cambridge, Massachusetts. The company specializes in developing therapeutics and vaccines using messenger RNA (mRNA) technology, which provides instructions for cells to produce proteins essential for various biological functions. Moderna has a diverse pipeline, with numerous programs targeting infectious diseases, immuno-oncology, rare diseases, and cardiovascular conditions. As of September 2024, it has 40 mRNA development candidates in clinical trials across multiple therapeutic areas. The company's mRNA technology gained significant recognition with the authorization of its COVID-19 vaccine in December 2020. Moderna maintains strategic alliances with various organizations, including major pharmaceutical companies and research institutions, to enhance its research and development efforts.

Nivalis Therapeutics is a biotechnology company focused on developing innovative disease-modifying therapies aimed at cystic fibrosis (CF). The company is particularly dedicated to preserving intracellular S-nitrosoglutathione (GSNO), a naturally occurring molecule that plays a crucial role in cell signaling and is linked to the pathophysiology of CF. Its lead therapeutic candidate, N91115, specifically targets patients with the F508del mutation, which is the most prevalent mutation responsible for cystic fibrosis. Founded in 2007 and based in Boulder, Colorado, Nivalis Therapeutics aims to significantly improve the quality of life for individuals affected by cystic fibrosis and their families.

Blueprint Medicines Corporation is a biopharmaceutical company that specializes in developing small molecule kinase inhibitors aimed at treating genomically defined cancers and rare diseases. Founded in 2008 and headquartered in Cambridge, Massachusetts, the company focuses on personalized cancer therapies by leveraging its proprietary chemical library and advanced research capabilities. Its key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, and pralsetinib for RET-altered non-small cell lung cancer and medullary thyroid carcinoma. Additionally, Blueprint Medicines is advancing BLU-263, a KIT inhibitor for mast cell disorders, and fisogatinib for advanced hepatocellular carcinoma, both currently in clinical trials. The company’s innovative approach seeks to address the molecular drivers of cancer and resistance mechanisms, aiming to enhance treatment effectiveness and patient outcomes. Blueprint Medicines has established collaborations with several pharmaceutical companies to further its research and development efforts.

Adaptimmune Therapeutics, established in 2008, is a UK-based clinical-stage biopharmaceutical company specializing in novel cell therapies for solid tumor patients. Its core technology, the Specific Peptide Enhanced Affinity Receptor (SPEAR) T-cell platform, enables the identification of cancer targets and the development of affinity-enhanced T-cell receptors. Adaptimmune's pipeline includes several SPEAR T-cell therapies in phase I clinical trials, targeting various solid tumors such as urothelial, melanoma, and non-small cell lung cancer. The company has strategic collaborations with GSK, Noile-Immune Biotech, and Universal Cells, Inc. to advance its T-cell therapies.

Civitas Therapeutics is a biopharmaceutical company dedicated to the development and commercialization of innovative pulmonary delivery therapies aimed at treating central nervous system and respiratory disorders. Utilizing its proprietary ARCUS technology, Civitas aims to transform the therapeutic landscape for patients across the United States. The company's focus is on creating effective treatments that enhance patient outcomes and address unmet medical needs in these critical areas.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.