Novo Holdings

Glycomine, Inc. is a biotech company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, the company addresses the significant unmet medical needs of patients suffering from rare diseases, of which approximately 7,000 exist globally, with a majority lacking FDA-approved treatments. Glycomine specializes in creating replacement therapies that combine substrates, enzymes, and proteins with advanced bio-nano materials. These therapeutics are designed to facilitate the targeted delivery of treatment to clinically relevant organs, thereby enhancing the efficacy of care for conditions characterized by metabolic disorders and protein misfolding.

Hillstar Bio is a biopharmaceutical company focused on developing precision immunotherapies for treating autoimmune diseases. Its innovative approach selectively targets and depletes harmful immune cells, sparing healthy ones, to address specific disease mechanisms and reduce the risks associated with chronic broad immunosuppression. The company aims to provide durable relief and immune reset for patients with autoimmune conditions, addressing unmet medical needs.

Founded in 2021, Commit Biologics is a Danish biotechnology company dedicated to developing innovative immunotherapy platforms for cancer treatment. Its proprietary platform employs b-specific domain antibodies to engage the body's innate immune response, targeting and destroying cancer cells.

Windward Bio is a clinical-stage biopharmaceutical company dedicated to enhancing outcomes for patients with advanced immunological diseases, focusing on severe respiratory conditions. The company is advancing a fully human monoclonal antibody targeting thymic stromal lymphopoietin (TSLP) into phase 2 clinical trials. This antibody, administered subcutaneously, potently binds to the TSLP ligand, extending its half-life and silencing its effector functions. Windward Bio is also developing innovative bispecific programs for other immunological disorders, led by a team of experienced biopharmaceutical executives aiming to address significant unmet medical needs in this field.

Nuvig Therapeutics focuses on developing innovative therapies aimed at restoring immune homeostasis and rebalancing immune function. The company is dedicated to creating a pipeline of novel immune therapeutics specifically targeting chronic inflammatory and autoimmune diseases. By harnessing natural mechanisms to modulate immune responses, Nuvig seeks to enhance treatment options for patients, ultimately improving their health outcomes.

Adcendo is a biotechnology company focused on developing novel antibody-drug conjugates (ADCs) for cancer treatment. These ADCs target specific receptors overexpressed in various cancer forms with significant unmet needs, such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer.

Alentis Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for cancer and fibrotic diseases, including liver fibrosis, cirrhosis, and liver cancer. It pursues CLDN1-targeted approaches, developing anti-CLDN1 antibodies and antibody-drug conjugates to modify disease progression in CLDN1-positive cancers. Founded in 2019 and based in Basel, Switzerland, the company advances programs that aim to modulate the immune response and enhance anti-tumor activity while addressing liver-related conditions.

Kivu Biosciences, based in San Francisco, is a biotechnology company specializing in the development of next-generation antibody-drug conjugates (ADCs) for cancer treatment. The company aims to deliver superior therapeutics and rapidly advance transformative assets, operating discreetly while focusing on its innovative pipeline.

Based on our deep science and focused on patients in need.

Convergent Therapeutics is a biotechnology company developing radiopharmaceutical therapies for prostate cancer. Its proprietary platform links monoclonal antibodies targeting prostate-specific membrane antigen (PSMA) with alpha-emitting radionuclides, enabling precise delivery of radiation to cancer cells while minimizing exposure to healthy tissue.

Route 92 Medical, established in 2015 and headquartered in San Mateo, California, specializes in the development of innovative medical devices for stroke treatment. Their flagship product is a stealthy catheter designed for rapid and efficient removal of blood clots during acute ischemic stroke, aiming to improve patient outcomes and enhance recovery. Additionally, the company offers a rapid aspiration thrombectomy system and an intra-cerebral implant delivery system, further expanding their capabilities in stroke care.

Oxford Nanopore Technologies Limited is a biotechnology company specializing in the development and commercialization of nanopore-based sequencing technology for the analysis of DNA, RNA, proteins, and small molecules. Its innovative product lineup includes the portable MinION device, the benchtop PromethION, and the GridION system, all of which enable real-time data streaming and scalable sequencing for a variety of applications in scientific research, personalized medicine, environmental monitoring, and agriculture. The Flongle offers smaller, on-demand sequencing solutions, catering to users requiring rapid insights. Operating in over 80 countries, Oxford Nanopore's technology is designed for accessibility and ease of use, featuring straightforward library preparation and automated solutions. The company maintains a robust patent portfolio, supporting its ongoing research and development in both biological and solid-state nanopore technologies. Founded in 2005 and based in Oxford, United Kingdom, Oxford Nanopore Technologies also has offices in multiple countries, including the United States, China, and Germany.

Draupnir Bio is a biotechnology company focused on developing a new class of cholesterol-lowering medicines to prevent heart blood clots more effectively than existing therapies. The company pursues a platform that maps and leverages the glycome to build heparan sulfate glycomimetics, enabling synthesis, screening, and selection of potent, specific drug candidates for cardiovascular, inflammatory, and infectious diseases. Leveraging its technology, the firm aims to develop protein degraders that target extracellular disease proteins, potentially broadening therapeutic reach across unmet medical needs. The approach seeks to offer convenient treatment options that could reduce reliance on traditional injectables. Through its glycome-based platform, Draupnir Bio targets modalities beyond small molecules, with a focus on extracellular targets and biologics-compatible strategies to improve patient outcomes.

Adcendo is a biotechnology company focused on developing novel antibody-drug conjugates (ADCs) for cancer treatment. These ADCs target specific receptors overexpressed in various cancer forms with significant unmet needs, such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer.

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

Founded in 2021, Commit Biologics is a Danish biotechnology company dedicated to developing innovative immunotherapy platforms for cancer treatment. Its proprietary platform employs b-specific domain antibodies to engage the body's innate immune response, targeting and destroying cancer cells.

Bluejay Therapeutics is a private biopharmaceutical company focused on developing curative treatments for infectious and liver diseases. Its initial program targets chronic hepatitis B, a widespread infection with unmet medical need, using two approaches: fully human IgG1 monoclonal antibodies against hepatitis B surface antigen and oral small-molecule inhibitors of HBsAg. The aim is to reduce viral surface antigen and restore adaptive immunity, potentially achieving a functional cure for HBV and enabling patients to regain immune control of infection. The company’s work also contemplates extending its approach to other viral liver diseases, including chronic hepatitis D.

Reunion Neuroscience is a clinical-stage pharmaceutical company focused on developing therapeutic solutions for underserved mental health conditions. Its lead asset, RE104, is a proprietary psychedelic compound being developed as a potential fast-acting and durable antidepressant for patients suffering from postpartum depression and other mental health conditions.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Asgard Therapeutics is a private biotechnology company spun out from Lund University that develops in vivo direct cell reprogramming approaches for cancer immunotherapy. It advances a gene therapy platform called TrojanDC that reprograms cells to function as professional antigen-presenting cells, with the goal of initiating and shaping personalized anti-tumor immune responses. The company focuses on generating immune therapies capable of addressing cancers resistant to conventional treatments.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Catalent Pharma Solutions provides drug delivery technologies and outsourced development, manufacturing, and packaging services to the pharmaceutical, biotechnology, and consumer health industries worldwide. It operates in four segments: Development and Clinical Services, Oral Technologies, Sterile Technologies, and Packaging Services. Development and Clinical Services offers manufacturing, packaging, storage, and inventory management for drugs and biologics in clinical trials, along with analytical chemical and cell-based testing, respiratory product formulation and manufacturing, regulatory consulting, and biologics proprietary expression technology and product development. The Oral Technologies segment focuses on formulation, development, and manufacturing for oral dose forms, including conventional tablets and capsules, controlled-release formulations, and proprietary oral delivery technologies. The Sterile Technologies segment provides formulation and development for injectable drugs and fills products into vials, pre-filled syringes, bags, and other sterile formats, with blow-fill-seal technology for integrated dose form creation. Packaging Services delivers packaging for pharmaceuticals, biologics, consumer health, and veterinary products, including blisters, bottles, pouches, unit doses, and printed components.

OnCusp Therapeutics is a biopharmaceutical company dedicated to accelerating oncology medication innovation globally. It focuses on translating preclinical drug candidates into clinical proof-of-concept stage and advancing them for rapid global early clinical development.

NMD Pharma A/S is a clinical-stage biotech company based in Aarhus, Denmark, focused on developing small molecules to enhance neuromuscular transmission for the treatment of orphan motor neuron diseases, such as myasthenia gravis, amyotrophic lateral sclerosis, and spinal muscular atrophy. The company's lead candidate, NMD670, is currently undergoing clinical trials aimed at alleviating the symptoms of myasthenia gravis. Utilizing a translational muscle electrophysiology platform, NMD Pharma leverages its expertise in muscle physiology to design innovative therapeutics that address significant unmet medical needs in neuromuscular disorders. The company aims to improve the quality of life and survival rates for patients affected by these debilitating conditions.

Terremoto Biosciences is a biotechnology company that develops innovative drug delivery systems. Its platform enables the creation of optimized small-molecule medicines, aiming to enhance therapeutic benefits and potentially cure diseases.

MapLight Therapeutics is focused on discovering and developing innovative therapies for brain disorders. The company utilizes a unique platform that integrates single-cell transcriptomics with optogenetics, enabling the identification of novel drug targets. By leveraging these advanced technologies, MapLight aims to create effective treatments tailored to address complex neurological conditions. Founded in 2018 and based in San Francisco, California, the company is committed to advancing the understanding and treatment of brain disorders through its cutting-edge research and development efforts.

MinervaX is a biotechnology company focused on developing maternal vaccines to prevent infections caused by Group B streptococcus (GBS), which can lead to serious adverse pregnancy outcomes and life-threatening conditions for newborns. The company leverages a specific fusion protein that has shown promise in eliciting protective immunity against various clinically relevant GBS strains. By concentrating on this innovative vaccine approach, MinervaX aims to provide medical professionals with effective tools to safeguard newborns from bacterial infections, ultimately enhancing maternal and infant health outcomes.

Avalyn Pharma is a biopharmaceutical company focused on developing therapies for severe respiratory diseases, notably idiopathic pulmonary fibrosis (IPF). Established in 2011, the company is headquartered in Seattle, Washington.

Bactolife is a company focused on addressing the global challenge of antimicrobial resistance through innovative solutions. It develops proprietary Binding Protein ingredients aimed at fortifying the gut microbiome, thereby reducing the risk of gastrointestinal infections in both humans and animals. Bactolife's platform is designed to neutralize harmful pathogens and their toxins while preserving beneficial microbes, thus promoting overall gut health. By enhancing gut microbiome modulation technologies, the company seeks to diminish reliance on traditional antimicrobials, ultimately improving health outcomes and contributing to the fight against antimicrobial resistance.

Alentis Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for cancer and fibrotic diseases, including liver fibrosis, cirrhosis, and liver cancer. It pursues CLDN1-targeted approaches, developing anti-CLDN1 antibodies and antibody-drug conjugates to modify disease progression in CLDN1-positive cancers. Founded in 2019 and based in Basel, Switzerland, the company advances programs that aim to modulate the immune response and enhance anti-tumor activity while addressing liver-related conditions.

Adcendo is a biotechnology company focused on developing novel antibody-drug conjugates (ADCs) for cancer treatment. These ADCs target specific receptors overexpressed in various cancer forms with significant unmet needs, such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer.

Spruce Biosciences is a biopharmaceutical company focused on developing therapies for rare endocrine disorders. Its pipeline includes tildacerfont for classic congenital adrenal hyperplasia in adults and children, with ongoing clinical development, and Tralesinidase Alfa in its portfolio. The company collaborates with Eli Lilly to research, develop, and commercialize compounds. Founded in 2014, Spruce Biosciences is headquartered in Daly City, California, and aims to deliver meaningful therapies for patients with rare endocrine diseases.

Evosep is a developer of clinical proteomics solutions focused on making protein analysis faster and more robust for clinical and large-scale studies. Its products include EVOSEP ONE, a separation tool for clinical omics, and EVOTIP, disposable trap columns for sample loading. The company aims to enable reproducible plasma performance, protein identification with short gradients, rapid generation of data-independent acquisition libraries, and scalable screening for biomarkers in large cohorts. Built on extensive nano-UHPLC R&D experience, Evosep designs system architectures that improve throughput while maintaining reliability for clinical diagnostics. Founded in 2016 and based in Odense, Denmark, Evosep collaborates with leading scientists to advance proteomics-driven diagnostics.

Amolyt Pharma is a clinical-stage biotechnology company focused on developing peptide-based therapies for rare endocrine and metabolic disorders. The company is advancing several programs, including AZP-3601, a parathyroid hormone analog aimed at treating hypoparathyroidism by maintaining stable calcium levels in the blood. Additionally, Amolyt Pharma is developing AZP-3404, a peptide with a novel mechanism of action designed to restore fat and glucose metabolism. Through its innovative approaches, the company aims to provide effective solutions for patients suffering from critical and rare metabolic conditions.

iECURE is a gene editing company developing mutation-agnostic in vivo gene insertion therapies to treat rare, life-threatening liver disorders in children. Its approach seeks long-term, potentially curative treatment for metabolic liver diseases caused by loss-of-function mutations, with initial programs targeting ornithine transcarbamylase deficiency, citrullinemia type 1, and phenylketonuria.

Breye Therapeutics, a Danish-based biopharmaceutical firm in the clinical stages, is working to provide cutting-edge oral treatments for the management of retinal vascular diseases.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Inspirna is a clinical-stage biopharmaceutical company focused on developing first-in-class cancer therapies that target RNA-regulated drivers of tumor growth. Using RNA biology-based discovery platforms, including miRNA-DRIVEr and RNA-DRIVEr, it identifies novel cancer targets and advances them with oral small-molecule medicines and antibody-drug conjugates. Its lead programs include RGX-202 for RAS-mutant colorectal cancer and RGX-104 for small cell and non-small cell lung cancers, currently in early-phase trials (Phase 1b/2). The company aims to translate RNA-centric targets into treatments for patients with cancers that lack effective options.

TheraXyte BioScience is a biotechnology company specializing in extracellular vesicle (EV)-mediated drug delivery and novel EV-based therapeutics. The company has developed an advanced EV-based drug delivery platform called TAXY, which facilitates the mass production of modifiable EVs at a high manufacturing standard. This platform effectively packages payloads and targets specific organs or cells for therapeutic release. TheraXyte's goal is to address various therapeutic challenges by advancing EV research from laboratory settings to clinical applications, ultimately aiming to improve human health through innovative EV-based treatments.

Nuvig Therapeutics focuses on developing innovative therapies aimed at restoring immune homeostasis and rebalancing immune function. The company is dedicated to creating a pipeline of novel immune therapeutics specifically targeting chronic inflammatory and autoimmune diseases. By harnessing natural mechanisms to modulate immune responses, Nuvig seeks to enhance treatment options for patients, ultimately improving their health outcomes.

Developer of biotechnology research company designed for the next generation of T cell receptor therapies against cancer. The company engages in the development of TCR-based immunotherapies including T-cell therapies and biologics combining genome editing, functional high-throughput screening, deep sequencing, and machine learning, addressing key efficacy and safety challenges, enabling patients to get the treatment of serious diseases with high unmet medical need.

Precirix is a biotechnology company based in Brussels, Belgium, focused on the development of radio-immunotherapeutic drugs aimed at treating cancer patients. Established in 2014, the company utilizes the unique properties of the camelid immune system to create targeted therapies. Its innovative approach involves using antigen-binding fragments to deliver therapeutic radioisotopes directly to specific receptors on cancer cells. This method allows for a more personalized treatment option, enhancing the effectiveness of cancer therapies while minimizing damage to healthy tissue. Precirix is dedicated to advancing the field of oncology through its specialized drug development, contributing to improved outcomes for patients with various types of cancer.

NMD Pharma A/S is a clinical-stage biotech company based in Aarhus, Denmark, focused on developing small molecules to enhance neuromuscular transmission for the treatment of orphan motor neuron diseases, such as myasthenia gravis, amyotrophic lateral sclerosis, and spinal muscular atrophy. The company's lead candidate, NMD670, is currently undergoing clinical trials aimed at alleviating the symptoms of myasthenia gravis. Utilizing a translational muscle electrophysiology platform, NMD Pharma leverages its expertise in muscle physiology to design innovative therapeutics that address significant unmet medical needs in neuromuscular disorders. The company aims to improve the quality of life and survival rates for patients affected by these debilitating conditions.

Asgard Therapeutics is a private biotechnology company spun out from Lund University that develops in vivo direct cell reprogramming approaches for cancer immunotherapy. It advances a gene therapy platform called TrojanDC that reprograms cells to function as professional antigen-presenting cells, with the goal of initiating and shaping personalized anti-tumor immune responses. The company focuses on generating immune therapies capable of addressing cancers resistant to conventional treatments.

Amolyt Pharma is a clinical-stage biotechnology company focused on developing peptide-based therapies for rare endocrine and metabolic disorders. The company is advancing several programs, including AZP-3601, a parathyroid hormone analog aimed at treating hypoparathyroidism by maintaining stable calcium levels in the blood. Additionally, Amolyt Pharma is developing AZP-3404, a peptide with a novel mechanism of action designed to restore fat and glucose metabolism. Through its innovative approaches, the company aims to provide effective solutions for patients suffering from critical and rare metabolic conditions.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Aristea Therapeutics, established in 2018 and headquartered in San Diego, California, is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for serious inflammatory diseases. The company focuses on addressing unmet medical needs by creating drugs targeting conditions such as palmoplantar pustulosis, hidradenitis suppurativa, inflammatory bowel disease (IBD), familial Mediterranean fever, and Behçet's disease. Aristea Therapeutics operates with a diligent and agile approach to drug development, aiming to provide patients with access to innovative treatments for these conditions.

Glycomine, Inc. is a biotech company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, the company addresses the significant unmet medical needs of patients suffering from rare diseases, of which approximately 7,000 exist globally, with a majority lacking FDA-approved treatments. Glycomine specializes in creating replacement therapies that combine substrates, enzymes, and proteins with advanced bio-nano materials. These therapeutics are designed to facilitate the targeted delivery of treatment to clinically relevant organs, thereby enhancing the efficacy of care for conditions characterized by metabolic disorders and protein misfolding.

Adcendo is a biotechnology company focused on developing novel antibody-drug conjugates (ADCs) for cancer treatment. These ADCs target specific receptors overexpressed in various cancer forms with significant unmet needs, such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer.

Arcellx is a clinical-stage biotechnology company developing immunotherapies and cell therapies for cancer and other serious diseases. It seeks to make cell therapies safer, more effective, and more broadly accessible. Its lead candidate CART-ddBCMA is in a Phase 1 trial for relapsed or refractory multiple myeloma and has FDA Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations. The company is also advancing ARC-SparX, a dosable and controllable CAR-T platform, into clinical programs ACLX-001 for relapsed or refractory multiple myeloma and ACLX-002 for relapsed or refractory acute myeloid leukemia. Arcellx's pipeline covers multiple myeloma, acute myeloid leukemia, and other cancer indications, with research extending into additional oncology areas and other serious diseases.

Altasciences is a mid-sized contract research organization based in Laval, Quebec, specializing in preclinical and clinical pharmacology services for pharmaceutical and biotechnology companies. The company provides a comprehensive range of services, including preclinical safety testing, clinical pharmacology, drug formulation, manufacturing, bioanalysis, medical writing, biostatistics, and clinical monitoring. By offering integrated and customizable solutions, Altasciences supports sponsors in making informed decisions throughout early drug development. The company's focus is on accelerating the clinical research process to help bring effective drugs to market more rapidly, ultimately improving access for patients in need.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

IO Biotech is a clinical-stage biopharmaceutical company focused on developing innovative immune therapies for cancer treatment. Utilizing its unique T-win technology platform, the company creates first-in-class immune-modulating cancer vaccines that aim to activate T cells specific to immune-suppressive molecules. This approach allows for the simultaneous targeting and disruption of multiple pathways that tumors exploit to evade the immune response. IO Biotech has demonstrated a strong capability in advancing its preclinical and clinical candidates, with its two leading compounds targeting IDO and PD-L1 currently in clinical development, while several others are in the preclinical phase.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Reneo Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in San Diego, California, dedicated to developing therapies for patients with rare genetic mitochondrial diseases. These conditions are characterized by deficits in cellular metabolism and energy production, often leading to significant health challenges. The company is focused on creating treatments that improve mitochondrial function, preserve muscle integrity, and enhance the overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes critical to the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. Founded in 2014, Reneo aims to address the unmet needs of patients suffering from orphan metabolic diseases linked to mitochondrial dysfunction.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

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.

Evotec is a drug discovery partnership firm that collaborates with pharmaceutical and biotechnology companies, academic institutions, and nonprofit organizations to advance innovative therapeutic solutions. The company specializes in a range of therapeutic areas, including central nervous system disorders, diabetes, inflammation, oncology, infectious diseases, and women's health. Approximately 80% of its revenue comes from shared research and development services, which involve fee-for-service work and integrated drug discovery collaborations utilizing proprietary assets. The remaining 20% of sales derive from its Just-Evotec Biologics segment, which provides contract development and manufacturing organization services for biologics. Evotec's approach emphasizes collaboration with various stakeholders, including patient advocacy groups and venture capitalists, to enhance drug discovery and development processes.

Rappta Therapeutics Oy is a biopharmaceutical company focused on developing innovative anti-cancer drugs that target protein phosphatase 2A (PP2A), an essential enzyme involved in regulating protein de-phosphorylation and tumor growth. Established in 2019 and headquartered in Helsinki, Finland, with an additional office in the United States, the company is in the lead-optimization stage, working to create a series of first-in-class molecules that aim to reactivate this vital tumor suppressor. With proprietary development tools, Rappta Therapeutics is enhancing its capacity for rational drug design, thus providing therapeutic options for a variety of cancer types and certain important non-oncology conditions.

Syndesi Therapeutics SA is a biotechnology company based in Louvain-la-Neuve, Belgium, founded in 2018. The company specializes in developing drug molecules that modulate the synaptic vesicle protein SV2A, which is crucial for synaptic transmission—the process that enables communication between neurons in the brain. This innovative approach aims to address cognitive impairments associated with conditions such as Alzheimer’s Disease. Syndesi Therapeutics holds an exclusive license for its platform technology from UCB, a leader in SV2A research, positioning the company to advance therapeutic options for patients suffering from cognitive disorders.

Galecto is a clinical-stage biotechnology company developing small molecule therapeutics targeting galectin-3 and lysyl oxidase-like 2 for treating severe diseases like fibrosis and cancer. Its lead product candidate, GB0139, is an inhaled inhibitor of galectin-3 for treating severe fibrotic lung diseases such as idiopathic pulmonary fibrosis.

Founded in 2016 and headquartered in Utrecht, the Netherlands, Lava Therapeutics is a biotechnology company specializing in the development of proprietary Vγ9Vδ2 T-cell engagers for immune oncology. Its platform focuses on creating bispecific antibodies to engage gamma-delta T cells for treating hematological and solid cancers.

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.

ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, specializing in the discovery and development of antiviral drugs targeting respiratory syncytial virus (RSV). Founded in 2011, ReViral is focused on creating innovative first-in-class compounds, including a highly potent fusion inhibitor designed specifically for treating severe RSV infections, particularly in vulnerable populations such as neonates. By advancing these novel antiviral treatments, ReViral aims to address significant unmet medical needs and expand the antiviral market, ultimately improving patient outcomes for those affected by RSV.

Mission Bio develops and delivers tools for studying cellular heterogeneity in human health and life science research. Its flagship product is the Tapestri Platform, a single-cell platform that detects DNA and protein changes, enabling precise detection of heterogeneity in disease progression and treatment response. The company's solutions are primarily applied in oncology, including blood cancers and solid tumors.

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.

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing therapies for chronic respiratory diseases with unmet medical needs. Its lead candidate, ensifentrine, is an inhaled dual inhibitor of phosphodiesterases 3 and 4 that provides bronchodilation and anti-inflammatory effects in a single molecule. Development programs include formulations of ensifentrine for chronic obstructive pulmonary disease, with options such as nebulized administration, a dry powder inhaler, and a pressurized metered-dose inhaler. Verona Pharma is also pursuing development of ensifentrine for cystic fibrosis and asthma. The company was founded in 2005 and is headquartered in London, United Kingdom.

ISD-Immunotech develops a first-in-class therapy targeting the STING pathway to treat severe systemic lupus erythematosus (SLE), also known as lupus. Its therapy dampens the action of stimulator of interferon genes (STING) protein, reducing inflammatory proteins and cytokines that overactivate the immune system in SLE patients.

Freeline Therapeutics is a clinical-stage biotechnology company specializing in systemic adeno-associated virus (AAV)-based gene therapies. Its pipeline includes FLT180a for hemophilia B, FLT190 for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A, all in various stages of clinical trials.

Checkmate Pharmaceuticals is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing novel immunotherapies for cancer treatment, with a focus on CpG oligonucleotides. These oligonucleotides activate an anti-tumor T-cell response and are combined with checkpoint inhibition to enhance the immune system's ability to combat tumors. Checkmate Pharmaceuticals has formed strategic alliances with Merck KGaA and Pfizer to advance its clinical programs, aiming to increase the efficacy of existing immunotherapies and provide new treatment options for cancer patients.

IO Biotech is a clinical-stage biopharmaceutical company focused on developing innovative immune therapies for cancer treatment. Utilizing its unique T-win technology platform, the company creates first-in-class immune-modulating cancer vaccines that aim to activate T cells specific to immune-suppressive molecules. This approach allows for the simultaneous targeting and disruption of multiple pathways that tumors exploit to evade the immune response. IO Biotech has demonstrated a strong capability in advancing its preclinical and clinical candidates, with its two leading compounds targeting IDO and PD-L1 currently in clinical development, while several others are in the preclinical phase.

Avalyn Pharma is a biopharmaceutical company focused on developing therapies for severe respiratory diseases, notably idiopathic pulmonary fibrosis (IPF). Established in 2011, the company is headquartered in Seattle, Washington.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Spruce Biosciences is a biopharmaceutical company focused on developing therapies for rare endocrine disorders. Its pipeline includes tildacerfont for classic congenital adrenal hyperplasia in adults and children, with ongoing clinical development, and Tralesinidase Alfa in its portfolio. The company collaborates with Eli Lilly to research, develop, and commercialize compounds. Founded in 2014, Spruce Biosciences is headquartered in Daly City, California, and aims to deliver meaningful therapies for patients with rare endocrine diseases.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

Aligos Therapeutics is a biopharmaceutical company dedicated to developing innovative therapeutics targeting viral infections and liver diseases. Founded in 2018, the company focuses on chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH), leveraging its expertise in liver disease drug development.

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.

NorthSea Therapeutics B.V. is a biotechnology company based in Naarden, the Netherlands, focused on developing and manufacturing innovative therapeutics for metabolic, inflammatory, and fibrotic diseases. Founded in 2017, the company utilizes its proprietary Structurally Engineered Fatty Acid (SEFA) technology to create unique oral therapeutics, including icosabutate, which targets inflammatory and liver diseases. NorthSea Therapeutics aims to address various health conditions, such as dyslipidemia, insulin resistance, hepatic inflammation, and fibrosis, by delivering therapies that offer diverse biological effects. The company's pipeline reflects its commitment to advancing treatment options that enhance patient care and outcomes.

WCG is a company focused on accelerating the delivery of new treatments and therapies in the biopharmaceutical sector. It serves biopharmaceutical companies, contract research organizations (CROs), and health institutions by providing a range of services designed to enhance clinical and technological processes. These services include study planning, site optimization, patient engagement, and scientific and market insight solutions. By supporting organizations that develop products and therapies, WCG aims to improve human health and facilitate the efficient advancement of medical innovations.

Draupnir Bio is a biotechnology company focused on developing a new class of cholesterol-lowering medicines to prevent heart blood clots more effectively than existing therapies. The company pursues a platform that maps and leverages the glycome to build heparan sulfate glycomimetics, enabling synthesis, screening, and selection of potent, specific drug candidates for cardiovascular, inflammatory, and infectious diseases. Leveraging its technology, the firm aims to develop protein degraders that target extracellular disease proteins, potentially broadening therapeutic reach across unmet medical needs. The approach seeks to offer convenient treatment options that could reduce reliance on traditional injectables. Through its glycome-based platform, Draupnir Bio targets modalities beyond small molecules, with a focus on extracellular targets and biologics-compatible strategies to improve patient outcomes.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Allievex is a clinical-stage biotechnology company developing novel, non-opioid therapeutics for treating rare pediatric neurodegenerative diseases. Its lead product, tralesinidase alfa, is an investigational enzyme replacement therapy targeting Sanfilippo syndrome type B and mucopolysaccharidosis IIIB in children.

Clario specializes in endpoint technology solutions for clinical trials, serving pharmaceutical, biotech, and medical device partners. The company integrates scientific expertise with a comprehensive technology platform to facilitate various trial models, including decentralized, hybrid, and site-based studies. With a presence in nine countries and around 30 facilities across North America, Europe, and Asia Pacific, Clario has been providing critical clinical evidence for nearly 50 years. Its services include health outcomes research, cardiac safety, respiratory assessments, electronic clinical outcomes, and suicide risk evaluations. By leveraging software-enabled clinical research, Clario aims to enhance data quality, reduce costs, shorten study timelines, and improve trial oversight and patient engagement.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Arcellx is a clinical-stage biotechnology company developing immunotherapies and cell therapies for cancer and other serious diseases. It seeks to make cell therapies safer, more effective, and more broadly accessible. Its lead candidate CART-ddBCMA is in a Phase 1 trial for relapsed or refractory multiple myeloma and has FDA Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations. The company is also advancing ARC-SparX, a dosable and controllable CAR-T platform, into clinical programs ACLX-001 for relapsed or refractory multiple myeloma and ACLX-002 for relapsed or refractory acute myeloid leukemia. Arcellx's pipeline covers multiple myeloma, acute myeloid leukemia, and other cancer indications, with research extending into additional oncology areas and other serious diseases.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

Anokion is a biotechnology company focused on antigen-specific immune tolerance to reduce immunogenicity of therapeutic proteins and treat autoimmune and allergic diseases. A spin-off from EPFL, it uses a platform technology that can be applied to many proteins across multiple clinical indications, including autoimmune conditions such as celiac disease, multiple sclerosis, and diabetes. The company is based in Ecublens, Switzerland.

Nkarta Therapeutics is a clinical-stage biopharmaceutical company focused on developing allogeneic, off-the-shelf engineered natural killer (NK) cell therapies. The company aims to harness the inherent power of NK cells to target and destroy tumor cells, offering a potent and better-tolerated alternative to traditional T-cell therapies for a broad range of cancer indications.

Glycomine, Inc. is a biotech company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, the company addresses the significant unmet medical needs of patients suffering from rare diseases, of which approximately 7,000 exist globally, with a majority lacking FDA-approved treatments. Glycomine specializes in creating replacement therapies that combine substrates, enzymes, and proteins with advanced bio-nano materials. These therapeutics are designed to facilitate the targeted delivery of treatment to clinically relevant organs, thereby enhancing the efficacy of care for conditions characterized by metabolic disorders and protein misfolding.

Amolyt Pharma is a clinical-stage biotechnology company focused on developing peptide-based therapies for rare endocrine and metabolic disorders. The company is advancing several programs, including AZP-3601, a parathyroid hormone analog aimed at treating hypoparathyroidism by maintaining stable calcium levels in the blood. Additionally, Amolyt Pharma is developing AZP-3404, a peptide with a novel mechanism of action designed to restore fat and glucose metabolism. Through its innovative approaches, the company aims to provide effective solutions for patients suffering from critical and rare metabolic conditions.

AFYX Therapeutics A/S is a pharmaceutical company based in Copenhagen, Denmark, focused on developing and manufacturing treatments for mucosal diseases. Established in 2014, the company is known for its flagship product, the Rivelin® patch, which is designed for the treatment of oral lichen planus. This innovative patch is flexible, biodegradable, and adheres to mucosal surfaces, utilizing electrospinning technology to deliver medication directly to the affected area. By offering targeted therapies for conditions that currently lack approved treatment options, AFYX Therapeutics aims to enhance patient relief and support recovery without disrupting daily activities.

Oxford Biomedica is a biopharmaceutical company focused on gene and cell therapy, specializing in the development, manufacturing, and advancement of lentiviral vector-based medicines and related cell therapies. The company leverages its LentiVector platform to deliver therapeutic genes into non-dividing cells and collaborates with major pharmaceutical firms to progress both internal candidates and externally funded programs. Its internal pipeline targets diseases of the eye and the central nervous system, while ongoing partnerships span multiple therapeutic areas across Phase I/II and beyond. Oxford Biomedica also operates as a contract development and manufacturing organization, offering viral vector development and manufacturing services through OXB Solutions. Founded in 1995 and headquartered in Oxford, United Kingdom, the company maintains a global collaboration network with industry and academic partners.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Oxford Biomedica is a biopharmaceutical company focused on gene and cell therapy, specializing in the development, manufacturing, and advancement of lentiviral vector-based medicines and related cell therapies. The company leverages its LentiVector platform to deliver therapeutic genes into non-dividing cells and collaborates with major pharmaceutical firms to progress both internal candidates and externally funded programs. Its internal pipeline targets diseases of the eye and the central nervous system, while ongoing partnerships span multiple therapeutic areas across Phase I/II and beyond. Oxford Biomedica also operates as a contract development and manufacturing organization, offering viral vector development and manufacturing services through OXB Solutions. Founded in 1995 and headquartered in Oxford, United Kingdom, the company maintains a global collaboration network with industry and academic partners.

MinervaX is a biotechnology company focused on developing maternal vaccines to prevent infections caused by Group B streptococcus (GBS), which can lead to serious adverse pregnancy outcomes and life-threatening conditions for newborns. The company leverages a specific fusion protein that has shown promise in eliciting protective immunity against various clinically relevant GBS strains. By concentrating on this innovative vaccine approach, MinervaX aims to provide medical professionals with effective tools to safeguard newborns from bacterial infections, ultimately enhancing maternal and infant health outcomes.

Aristea Therapeutics, established in 2018 and headquartered in San Diego, California, is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for serious inflammatory diseases. The company focuses on addressing unmet medical needs by creating drugs targeting conditions such as palmoplantar pustulosis, hidradenitis suppurativa, inflammatory bowel disease (IBD), familial Mediterranean fever, and Behçet's disease. Aristea Therapeutics operates with a diligent and agile approach to drug development, aiming to provide patients with access to innovative treatments for these conditions.

Precirix is a biotechnology company based in Brussels, Belgium, focused on the development of radio-immunotherapeutic drugs aimed at treating cancer patients. Established in 2014, the company utilizes the unique properties of the camelid immune system to create targeted therapies. Its innovative approach involves using antigen-binding fragments to deliver therapeutic radioisotopes directly to specific receptors on cancer cells. This method allows for a more personalized treatment option, enhancing the effectiveness of cancer therapies while minimizing damage to healthy tissue. Precirix is dedicated to advancing the field of oncology through its specialized drug development, contributing to improved outcomes for patients with various types of cancer.

Mirum Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing therapies for debilitating liver diseases. Its primary focus is on Maralixibat, an investigational oral drug in Phase 3 trials for treating progressive familial intrahepatic cholestasis disease and Alagille syndrome.