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.

Commit Biologics is a biotechnology research company based in Aarhus, Denmark, established in 2021. The company specializes in developing an innovative immunotherapy platform aimed at treating various types of cancer. This platform utilizes bispecific domain antibodies to effectively target and eliminate tumor cells. Furthermore, it incorporates modular technology that enhances its ability to engage the body's innate immune response, thereby activating the complement component of the immune system. This approach not only focuses on cancer treatment but also has potential applications in addressing autoimmune diseases.

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

Alentis Therapeutics AG is a clinical-stage biopharmaceutical company based in Basel, Switzerland, specializing in the development of innovative therapies for advanced liver diseases and cancers associated with Claudin-1 (CLDN1) expression. Founded in 2019, Alentis focuses on addressing conditions such as liver fibrosis, cirrhosis, and liver cancer through the use of anti-CLDN1 antibody-drug conjugates and other targeted treatments. The company's unique approach combines clinically relevant read-outs with advanced techniques like single-cell RNA sequencing of patient liver tissues, aiming to reverse disease progression and improve patient outcomes. Alentis Therapeutics is recognized for its pioneering role in exploring anti-CLDN1 therapeutics, contributing to advancements in the treatment of fibrotic diseases and CLDN1-positive tumors.

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.

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Convergent Therapeutics is a clinical-stage biotechnology company specializing in the development of radiopharmaceutical therapies for prostate cancer. The company's lead product, CONV 01-α, is a monoclonal antibody conjugated with the radioactive alpha particle emitter 225Ac, designed to bind specifically to the prostate-specific membrane antigen (PSMA). This innovative therapy allows for the internalization of the radioactive payload directly into prostate cancer cells, enhancing treatment efficacy. Developed through proprietary technology by Dr. Neil Bander at Weill Cornell Medicine, CONV 01-α is protected by multiple U.S. and foreign patents. If it receives FDA approval, it will become the first antibody to target a radioisotope for prostate cancer treatment and the first drug to utilize 225Ac in this context. Convergent Therapeutics aims to leverage its therapeutic platform to explore targeted combination strategies against validated and novel cancer antigens, ultimately enhancing treatment options for patients.

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 focused on developing innovative cholesterol-lowering medications that aim to prevent heart blood clots more effectively than existing treatments. The company leverages a platform technology that explores the glycome to create novel therapeutics through advanced techniques in array technology, protein chemistry, and carbohydrate chemistry. Draupnir Bio specializes in synthesizing, screening, and selecting potent heparan sulfate glycomimetic drug candidates that address cardiovascular, inflammatory, and infectious diseases. Additionally, the company is working on protein degraders that target extracellular disease proteins, which could potentially address a wide range of unmet therapeutic needs across various medical conditions, providing more convenient treatment options compared to traditional injectable therapies.

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

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.

Commit Biologics is a biotechnology research company based in Aarhus, Denmark, established in 2021. The company specializes in developing an innovative immunotherapy platform aimed at treating various types of cancer. This platform utilizes bispecific domain antibodies to effectively target and eliminate tumor cells. Furthermore, it incorporates modular technology that enhances its ability to engage the body's innate immune response, thereby activating the complement component of the immune system. This approach not only focuses on cancer treatment but also has potential applications in addressing autoimmune diseases.

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.

Reunion Neuroscience is a clinical-stage pharmaceutical company dedicated to developing therapeutic solutions for underserved mental health conditions. The company’s primary focus is on its lead asset, RE104, a proprietary serotonergic psychedelic compound designed as a fast-acting and durable antidepressant for patients experiencing postpartum depression and other mental health issues. Additionally, Reunion is advancing the RE200 series, which comprises compounds aimed at achieving more selective serotonin receptor activity with reduced psychoactivity, thereby broadening the potential applications for chronic treatment scenarios. The company seeks to address significant gaps in mental health care through innovative pharmacological approaches.

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.

Asgard Therapeutics is a private biotechnology company focused on developing innovative cancer immunotherapies through direct cell reprogramming technologies. Founded as a spin-off from Lund University, the company employs its proprietary TrojanDC technology to initiate immune responses by leveraging the biological properties of professional antigen-presenting cells. With a commitment to creating personalized and efficient gene therapy products, Asgard Therapeutics aims to expand its pipeline of off-the-shelf cancer treatments, particularly for patients with cancers that are resistant to traditional therapies. By activating tailored anti-cancer immune defenses, the company seeks to offer new therapeutic options for individuals in need of effective cancer treatment alternatives.

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

Catalent Pharma Solutions, Inc. provides drug delivery technologies and outsourced development, manufacturing, and packaging services to the pharmaceutical, biotechnology, and consumer health industry worldwide. It operates in four segments: Development and Clinical Services, Oral Technologies, Sterile Technologies, and Packaging Services. The Development and Clinical Services segment provides manufacturing, packaging, storage, and inventory management for drugs and biologics in clinical trials. It also offers analytical chemical and cell-based testing and scientific services, respiratory products formulation and manufacturing, regulatory consulting, and biologics proprietary expression technology, and product development. The Oral Technologies segment provides formulation, development, and manufacturing services for the oral dose forms. Its oral drug delivery technologies include proprietary delivery technologies for drugs and consumer health products. This segment also offers formulation, development, and manufacturing services for conventional oral dose forms, including controlled release formulations, as well as immediate release tablets and capsules. The Sterile Technologies segment provides proprietary and traditional dose forms for separate routes of administration. It offers formulation and development for injectable drugs; and fills drugs or biologics into vials, pre-filled syringes, bags, and other sterile delivery formats. This segment's blow-fill-seal technology provides integrated dose form creation and filling of sterile liquids in a single process for respiratory, ophthalmic, and other routes of administration. The Packaging Services segment offers packaging services for pharmaceuticals, biologics, consumer health, and veterinary products, which include packaging drugs in blisters, bottles, pouches, and unit doses; and printed components, such as creating package inserts or folding cartons. The company is based in Somerset, New Jersey.

OnCusp Therapeutics is a biopharmaceutical company dedicated to transforming advanced research into innovative cancer treatments. Founded by Bing Yuan, Eric Slosberg, and Andy Fu, the company focuses on accelerating the development of oncology medications to provide help and hope to cancer patients globally. OnCusp Therapeutics specializes in translating preclinical drug candidates into clinical proof-of-concept stages, aiming to swiftly advance these assets into early global 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 focused on the development and discovery of small-molecule medicines through innovative drug delivery systems. By utilizing lysine-based covalency, the company aims to optimize clinical profiles and enhance therapeutic benefits for various diseases. Terremoto's platform is designed to improve existing medications while also facilitating the creation of new therapies aimed at addressing critical healthcare challenges. Through its research and technology, Terremoto Biosciences strives to provide effective solutions for the treatment of devastating diseases, contributing to advancements in the healthcare sector.

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, 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.

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 AG is a clinical-stage biopharmaceutical company based in Basel, Switzerland, specializing in the development of innovative therapies for advanced liver diseases and cancers associated with Claudin-1 (CLDN1) expression. Founded in 2019, Alentis focuses on addressing conditions such as liver fibrosis, cirrhosis, and liver cancer through the use of anti-CLDN1 antibody-drug conjugates and other targeted treatments. The company's unique approach combines clinically relevant read-outs with advanced techniques like single-cell RNA sequencing of patient liver tissues, aiming to reverse disease progression and improve patient outcomes. Alentis Therapeutics is recognized for its pioneering role in exploring anti-CLDN1 therapeutics, contributing to advancements in the treatment of fibrotic diseases and CLDN1-positive tumors.

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

Spruce Biosciences, Inc. is a biopharmaceutical company based in Daly City, California, specializing in the development and commercialization of innovative therapies for rare endocrine disorders. Founded in 2014, the company is primarily focused on its lead product candidate, tildacerfont, which is undergoing clinical trials for adult and pediatric patients with classic congenital adrenal hyperplasia (CAH). Tildacerfont is being developed as a non-steroidal therapy aimed at improving disease management and reducing reliance on steroids for affected patients. Additionally, the company is exploring the use of tildacerfont for females with polycystic ovary syndrome. With a commitment to addressing the unmet medical needs of patients with rare endocrine conditions, Spruce Biosciences aims to enhance the quality of life for those who have been historically underserved by existing treatment options. The company also has a partnership with Eli Lilly and Company to explore further pharmaceutical applications of its compounds.

Evosep ApS is a Danish company specializing in clinical proteomics, aiming to enhance the efficiency and reliability of protein-based diagnostics. Founded in 2016 and based in Odense, Evosep develops advanced solutions such as the EVOSEP ONE separation tool and EVOTIP disposable trap columns, which facilitate the rapid and reproducible analysis of biological samples. By leveraging years of expertise in nano-UHPLC research and development, Evosep focuses on addressing the growing demand for high-throughput proteomics. The company's innovative technologies are designed to make sample separation ten times faster and a hundred times more robust than existing methods, which is essential for accurately diagnosing health conditions through biomarker analysis. Collaborating with leading scientists, Evosep is committed to transforming clinical diagnostics to improve patient care and quality of life.

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 biotechnology company dedicated to developing innovative gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. Its approach employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted interventions in diseases characterized by metabolic "loss of function" disorders. By focusing on conditions with high unmet medical needs, iECURE aims to offer potential cures for devastating illnesses that currently lack effective treatment options. The company's commitment to advancing gene editing technology positions it as a key player in the field of genetic medicine.

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

Inspirna is a clinical-stage biopharmaceutical company focused on developing innovative drug candidates that target essential drivers of cancer. Utilizing a discovery platform centered on RNA biology, Inspirna identifies novel cancer targets that contribute to tumor growth and disease progression. The company is advancing several first-in-class oral small molecules, including RGX-202, aimed at treating patients with RAS mutant colorectal cancer, and RGX-104, which is in development for small cell lung cancer and non-small cell lung cancer. Through its microRNA-based target discovery approach, Inspirna seeks to provide effective treatment options for cancer patients by employing targeted therapies that leverage small molecules and biologics.

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.

Engimmune Therapeutics is a biotechnology company focused on developing innovative T cell receptor (TCR) therapies aimed at treating solid tumors. The company leverages advanced life sciences technologies, including genome editing, functional high-throughput screening, deep sequencing, and machine learning, to create highly potent and specific immunotherapies. By addressing critical efficacy and safety challenges in cancer treatment, Engimmune Therapeutics is dedicated to providing effective solutions for patients suffering from serious diseases with significant unmet medical needs.

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 focused on developing innovative cancer immunotherapies through direct cell reprogramming technologies. Founded as a spin-off from Lund University, the company employs its proprietary TrojanDC technology to initiate immune responses by leveraging the biological properties of professional antigen-presenting cells. With a commitment to creating personalized and efficient gene therapy products, Asgard Therapeutics aims to expand its pipeline of off-the-shelf cancer treatments, particularly for patients with cancers that are resistant to traditional therapies. By activating tailored anti-cancer immune defenses, the company seeks to offer new therapeutic options for individuals in need of effective cancer treatment alternatives.

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

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

Arcellx is a clinical-stage biotechnology company focused on developing innovative immunotherapies, particularly in the realm of cell therapy, for patients with cancer and other serious diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Arcellx's lead product candidate, CART-ddBCMA, is currently being investigated in a Phase 1 clinical trial for the treatment of relapsed or refractory multiple myeloma. This therapy has received several designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is progressing its dosable and controllable CAR-T therapy, known as ARC-SparX, through two clinical programs targeting relapsed or refractory multiple myeloma and acute myeloid leukemia. Through these efforts, Arcellx seeks to contribute significantly to the advancement of cell therapy in modern medicine.

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, 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.

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 focused on developing small-molecule therapeutics to address severe diseases, particularly fibrosis and cancer. Established in 2011, Galecto builds on over a decade of research into the roles of galectin-3 and lysyl oxidase-like 2 (LOXL2) in fibrotic diseases. The company's lead product candidate, GB0139, is an inhaled galectin-3 inhibitor specifically designed for treating severe fibrotic lung diseases, such as idiopathic pulmonary fibrosis (IPF), where there is a significant unmet medical need. Additionally, Galecto is advancing GB1211, an oral selective galectin-3 inhibitor aimed at addressing fibrosis related to non-alcoholic steatohepatitis. With a robust patent portfolio, Galecto leverages its unique therapeutic platform to target the underlying biological processes associated with fibrosis and related diseases.

Lava Therapeutics B.V. is a biotechnology company based in Utrecht, the Netherlands, focused on innovative cancer treatments. Founded in 2016, it specializes in the development of proprietary bispecific antibodies that engage gamma-delta T cells, aiming to harness the immune system's power to target and destroy tumor cells. The company's platform creates next-generation γδ T cell engaging antibodies designed for both hematological and solid tumors, emphasizing safety, potency, and cost-effectiveness in its biopharmaceuticals. By combining immune recruiting and activating functionalities, Lava Therapeutics seeks to transform cancer therapy and improve patient outcomes.

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, Inc. is a biotechnology company focused on advancing the study of cellular heterogeneity through its innovative Tapestri Platform, which is the first single-cell multi-omics platform. This platform allows for the simultaneous analysis of genotype and phenotype from individual cells, enabling precise detection of variations in disease progression and treatment responses. Utilizing proprietary microfluidic droplet technology, Mission Bio provides targeted solutions for high-impact applications in fields such as oncology, hematology, and genome editing. The company also offers Tapestri Designer, a cloud-based tool for creating custom single-cell DNA panels, enhancing its capabilities in research and clinical settings. Founded in 2011 and based in South San Francisco, California, Mission Bio was previously known as Torrent Bio, Inc. before rebranding in 2013.

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 plc is a clinical-stage biopharmaceutical company based in London, dedicated to developing and commercializing innovative therapies for respiratory diseases with significant unmet medical needs. Founded in 2005, the company primarily focuses on its lead product candidate, ensifentrine, an inhaled dual inhibitor of phosphodiesterase enzymes 3 and 4, which functions as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is currently in Phase 2b clinical development as a nebulized formulation for the maintenance treatment of chronic obstructive pulmonary disease (COPD), with additional formulations including a dry powder inhaler and a pressurized metered-dose inhaler also under development. In addition to COPD, Verona Pharma is exploring the use of ensifentrine for other respiratory conditions, including cystic fibrosis and asthma, thereby aiming to enhance the health and quality of life for patients suffering from these chronic disorders.

ISD-Immunotech is a biotechnology company focused on developing innovative therapies for patients suffering from severe systemic lupus erythematosus (SLE), a complex autoimmune disease. The company specializes in sting modulators that target the STING pathway, which is crucial in the immune response. Its therapy aims to mitigate the activity of the stimulator of interferon genes (STING) protein, which, when activated, leads to an increase in inflammatory proteins such as interferons and cytokines. By dampening this response, ISD-Immunotech's approach seeks to help SLE patients manage their condition more effectively and enhance their ability to combat pathogenic invaders, such as bacteria and viruses.

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.

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, 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.

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, Inc. is a biopharmaceutical company based in Daly City, California, specializing in the development and commercialization of innovative therapies for rare endocrine disorders. Founded in 2014, the company is primarily focused on its lead product candidate, tildacerfont, which is undergoing clinical trials for adult and pediatric patients with classic congenital adrenal hyperplasia (CAH). Tildacerfont is being developed as a non-steroidal therapy aimed at improving disease management and reducing reliance on steroids for affected patients. Additionally, the company is exploring the use of tildacerfont for females with polycystic ovary syndrome. With a commitment to addressing the unmet medical needs of patients with rare endocrine conditions, Spruce Biosciences aims to enhance the quality of life for those who have been historically underserved by existing treatment options. The company also has a partnership with Eli Lilly and Company to explore further pharmaceutical applications of its compounds.

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, 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.

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 focused on developing innovative cholesterol-lowering medications that aim to prevent heart blood clots more effectively than existing treatments. The company leverages a platform technology that explores the glycome to create novel therapeutics through advanced techniques in array technology, protein chemistry, and carbohydrate chemistry. Draupnir Bio specializes in synthesizing, screening, and selecting potent heparan sulfate glycomimetic drug candidates that address cardiovascular, inflammatory, and infectious diseases. Additionally, the company is working on protein degraders that target extracellular disease proteins, which could potentially address a wide range of unmet therapeutic needs across various medical conditions, providing more convenient treatment options compared to traditional injectable therapies.

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

Allievex Corporation is a clinical-stage biotechnology company based in Marblehead, Massachusetts, focused on developing and commercializing innovative, non-opioid therapeutics for rare pediatric neurodegenerative diseases. Founded in 2018, the company is primarily known for its investigational enzyme replacement therapy, tralesinidase alfa. This therapy combines recombinant human alpha-N-acetyglucosaminidase with a peptide derived from insulin-like growth factor 2, aiming to provide treatment options for children affected by Sanfilippo syndrome type B and mucopolysaccharidosis IIIB. Through its targeted approach, Allievex seeks to address the unmet medical needs of patients with these rare and debilitating conditions.

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 focused on developing innovative immunotherapies, particularly in the realm of cell therapy, for patients with cancer and other serious diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Arcellx's lead product candidate, CART-ddBCMA, is currently being investigated in a Phase 1 clinical trial for the treatment of relapsed or refractory multiple myeloma. This therapy has received several designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is progressing its dosable and controllable CAR-T therapy, known as ARC-SparX, through two clinical programs targeting relapsed or refractory multiple myeloma and acute myeloid leukemia. Through these efforts, Arcellx seeks to contribute significantly to the advancement of cell therapy in modern medicine.

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, 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.

Anokion SA is a biotechnology company based in Ecublens, Switzerland, specializing in antigen-specific immune tolerance solutions. As a spin-off from the Ecole Polytechnique Fédérale de Lausanne, Anokion develops innovative immunotherapy aimed at reducing the immunogenicity of therapeutic proteins. The company's platform technology can be applied across a wide range of clinical indications, targeting both prevalent and rare autoimmune diseases, including celiac disease, multiple sclerosis, and type 1 diabetes. By engineering proteins or antigens for precise therapy, Anokion aims to provide effective treatment options that minimize side effects, thereby improving patient outcomes in the management of autoimmune and allergic diseases.

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

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 based in Oxford, United Kingdom, specializing in the research, development, and manufacturing of lentiviral vector and cell therapy products for various diseases. The company operates through two main segments: Platform and Product. Its LentiVector platform technology enables the integration of therapeutic genes into non-dividing cells, making it suitable for treating conditions affecting the brain and eyes. Oxford BioMedica's product pipeline includes several candidates in various stages of development, targeting diseases such as hematological tumors, age-related macular degeneration, and central nervous system disorders. Additionally, the company has established partnerships with notable firms like Novartis and Bristol Myers Squibb to advance its therapeutic offerings. Oxford BioMedica also focuses on growing its bioprocessing services, leveraging its technologies to provide revenue-generating solutions for third-party clients, while continuing to innovate in gene and cell therapy. Founded in 1995, the company has positioned itself as a leader in the field of gene therapy, addressing unmet medical needs through its advanced therapeutic candidates.

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 based in Oxford, United Kingdom, specializing in the research, development, and manufacturing of lentiviral vector and cell therapy products for various diseases. The company operates through two main segments: Platform and Product. Its LentiVector platform technology enables the integration of therapeutic genes into non-dividing cells, making it suitable for treating conditions affecting the brain and eyes. Oxford BioMedica's product pipeline includes several candidates in various stages of development, targeting diseases such as hematological tumors, age-related macular degeneration, and central nervous system disorders. Additionally, the company has established partnerships with notable firms like Novartis and Bristol Myers Squibb to advance its therapeutic offerings. Oxford BioMedica also focuses on growing its bioprocessing services, leveraging its technologies to provide revenue-generating solutions for third-party clients, while continuing to innovate in gene and cell therapy. Founded in 1995, the company has positioned itself as a leader in the field of gene therapy, addressing unmet medical needs through its advanced therapeutic candidates.

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 innovative therapies for debilitating liver diseases. Founded in 2018 and headquartered in Foster City, California, Mirum's lead product candidate is maralixibat, an investigational oral drug currently in Phase 3 clinical trials targeting progressive familial intrahepatic cholestasis, Alagille syndrome, and biliary atresia. The company’s approved product, Livmarli, is aimed at alleviating cholestatic pruritus in patients with Alagille syndrome. In addition to maralixibat, Mirum is also advancing the development of volixibat, which is intended for treating intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. Through its focused pipeline, Mirum aims to address significant unmet medical needs in rare and orphan liver diseases.