Novo Holdings

Tribune Therapeutics is a preclinical biopharmaceutical company focused on developing medicines to treat fibrotic diseases by targeting extracellular matrix–associated proteins central to scar formation, with the aim of halting progression across diverse tissues and conditions.

Augustine Therapeutics N.V. is a biopharmaceutical company based in Gent, Belgium, focused on developing innovative therapies for patients suffering from Charcot-Marie-Tooth disease (CMT) and other neuromuscular disorders. Founded in 2019, the company is engaged in two drug discovery programs aimed at identifying novel selective HDAC6 inhibitors. These compounds are designed to prevent nerve degeneration and facilitate the repair of peripheral myelin and axons. Through its research and development efforts, Augustine Therapeutics seeks to enhance the quality of life for patients affected by these debilitating conditions.

Callio Therapeutics specializes in the development of advanced antibody-drug conjugates (ADCs) for cancer treatment. Their focus is on creating multi-payload ADCs, which allow for the targeted delivery of drug combinations to tumor cells, potentially maximizing therapeutic benefits and improving patient outcomes.

Newleos Therapeutics is a stealth-mode biotechnology company focused on developing safer and more effective treatments for neuropsychiatric disorders, aiming to improve mental health outcomes.

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.

Coave Therapeutics specializes in developing gene therapies for rare ocular and central nervous system (CNS) diseases. Its platform enables targeted delivery of therapeutic genes to the CNS, enhancing autophagy and lysosomal function, with a focus on improving patient outcomes for conditions like ALS.

Numab Therapeutics is an oncology-focused biopharmaceutical company that designs and develops antibody-based and multispecific therapeutics for cancer, inflammatory diseases, autoimmunity, and related severe conditions. It employs a plug-and-play platform that reduces the randomness of discovery and aims to predictably yield mono- or multispecific antibody fragments with tailored pharmacokinetic properties to engage multiple targets. Founded in 2011 and based in Wädenswil, Switzerland, Numab pursues immuno-oncology and inflammation programs through this multispecific approach, prioritizing therapies with improved selectivity and potential clinical efficacy.

AnaCardio is a clinical stage biopharmaceutical company focused on developing innovative drugs for the treatment of heart failure. The company's therapeutics aim to enhance the contractility of the heart muscle through a unique mechanism that utilizes a therapeutic peptide. This approach is designed to avoid common adverse effects associated with traditional treatments, such as tachycardia, arrhythmia, ischemia, or hypotension. By improving cardiac output safely, AnaCardio's drugs have the potential to enhance organ function, elevate quality of life, increase functional capacity, and lower the risks of hospitalization and mortality for patients suffering from heart failure.

FIRE1 is an Irish-based company specializing in the development of innovative therapeutic devices. Its flagship product is a remote monitoring system designed to enhance heart failure patients' lives by providing real-time data to healthcare providers, facilitating early detection and proactive interventions.

Orbis Medicines is a company that develops a macrocyclic chemistry and computational platform for oral macrocycle drug discovery. The platform generates macrocycle candidates, nCycles, optimized for oral bioavailability to advance therapeutics that historically faced development hurdles. Its pipeline centers on nCycle candidates targeting validated biologic targets that are typically addressed by injectable drugs, with the goal of enabling oral delivery of such therapies. The platform combines chemistry and computational methods to accelerate drug discovery toward challenging targets in unmet-need indications, supporting therapeutics designed to improve patient outcomes.

SiteOne Therapeutics is a San Francisco-based company developing innovative therapeutics and diagnostics for acute and chronic pain. Their lead candidates are selective inhibitors of the Naᵥ1.7 ion channel, aiming to treat pain safely and effectively without relying on NSAIDs or opioids.

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.

Antag Therapeutics ApS is a biotechnology company based in Copenhagen, Denmark, founded in 2017. The company specializes in developing peptide drugs aimed at treating dietary-related metabolic diseases, including obesity, diabetes, and non-alcoholic fatty liver disease. Antag Therapeutics focuses on creating peptide-based GIP receptor antagonists designed to manage these conditions by reducing blood flow and lipid uptake in adipose tissue. Through its innovative approach, the company seeks to address significant health challenges associated with metabolic disorders.

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.

Booster Therapeutics is a biotechnology company focused on developing drug modalities to regulate cellular homeostasis and eliminate toxic accumulations. Its primary goal is to reinstate balance within cells, enabling the prevention and treatment of diseases associated with aging.

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.

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.

Asceneuron is a biotechnology company dedicated to discovering and developing therapeutics for neurodegenerative disorders with high unmet medical needs. It focuses on orphan tauopathies, Alzheimer's disease, and Parkinson's disease.

HepaRegeniX GmbH is a biopharmaceutical company based in Ulm, Germany, founded in 2016. It specializes in developing innovative therapies for acute and chronic liver diseases, focusing on restoring the regenerative capacity of hepatocytes. The company’s therapeutic approach is based on research led by Professor Lars Zender at the University Hospital Tübingen, which identified mitogen-activated protein kinase kinase 4 (MKK4) as a critical regulator of liver regeneration. HepaRegeniX is developing small-molecule inhibitors of MKK4, aiming to enhance liver regeneration even in severely diseased conditions. The company’s potential treatments may address various liver disorders, including nonalcoholic steatohepatitis.

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.

Clasp Therapeutics develops precision oncology platforms to identify neoantigens from driver mutations in hard-to-treat cancers. Its technology enables the creation of targeted therapies that selectively bind to HLA-presented peptides, allowing for precise destruction of cancer cells across various tumor types.

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.

Orbis Medicines is a company that develops a macrocyclic chemistry and computational platform for oral macrocycle drug discovery. The platform generates macrocycle candidates, nCycles, optimized for oral bioavailability to advance therapeutics that historically faced development hurdles. Its pipeline centers on nCycle candidates targeting validated biologic targets that are typically addressed by injectable drugs, with the goal of enabling oral delivery of such therapies. The platform combines chemistry and computational methods to accelerate drug discovery toward challenging targets in unmet-need indications, supporting therapeutics designed to improve patient outcomes.

Claris Biotherapeutics develops a topical formulation intended to cure corneal wounds, ulcers, and scars. The formulation creates a recombinant hepatocyte growth factor which accelerates the healing process, providing healthcare providers noninvasive therapeutic treatment.

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.

Hoba Therapeutics is focused on developing innovative biopharmaceuticals aimed at treating neuropathic pain and central nervous system disorders. The company is advancing a novel therapeutic protein designed to target specific cells, providing medical practitioners with effective and safe options for managing chronic pain and hearing loss. Through its research and development efforts, Hoba Therapeutics seeks to deliver disease-modifying treatments that enhance the quality of life for patients suffering from neuropathic pain.

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.

Paratek Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of novel tetracycline-based therapies to treat infectious diseases and related public health threats. Its lead programs center on omadacycline, a broad-spectrum tetracycline available in oral and intravenous forms for community-acquired bacterial pneumonia, acute bacterial skin and skin structure infections, urinary tract infections, and other serious infections, and sarecycline for acne and rosacea. The company emphasizes expertise in advanced tetracycline chemistry to address antibiotic resistance. Paratek collaborates with industry and academic partners, including licenses with Zai Lab and Allergan, a license with Tufts University to develop products for bacterial diseases, and a cooperative R&D agreement with the U.S. Army Medical Research Institute of Infectious Diseases to study omadacycline. Founded in 1996 and headquartered in Boston, Paratek aims to bring therapies that improve patient outcomes and address public health challenges.

Octave Health Group Inc. is a behavioral health provider based in New York that focuses on addressing the mental health needs of young urban professionals. Established in 2017, Octave offers therapy and coaching for a wide range of clinical conditions, including anxiety, depression, trauma, and relationship issues, as well as general emotional well-being concerns such as stress management and self-esteem. The company aims to create a proactive approach to emotional health, akin to the emphasis placed on physical fitness. Its services are designed to be easily integrated into clients' lives and are delivered by a team of experienced psychologists, therapists, and coaches. Octave's innovative model emphasizes accessibility and sustainability, setting a new standard for mental health care through comprehensive and personalized support tailored to individual client needs.

Ray Therapeutics develops optogenetic gene therapies to restore vision in patients with blinding retinal diseases. The company designs therapies that use bioengineered light-sensitive proteins delivered to targeted retinal cells via intravitreal injections, with the goal of improving visual function across various forms of retinal blindness and inherited retinal diseases. By combining gene delivery with optogenetic control, Ray Therapeutics aims to provide durable, long-term benefits from a single treatment, addressing diseases that lead to severe retinal degeneration. The company focuses on advancing research and development in this niche field to enable patients with limited or no light perception to regain functional sight, supporting efforts to study rare retinal conditions and translate laboratory insights into potential clinical therapies.

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.

FIRE1 is an Irish-based company specializing in the development of innovative therapeutic devices. Its flagship product is a remote monitoring system designed to enhance heart failure patients' lives by providing real-time data to healthcare providers, facilitating early detection and proactive interventions.

Hemab is a company that creates preventative treatments for bleeding and thrombosis problems. The medicines developed by the business aggregate, modify, balance, and at times recruit endogenous clotting components already present in the blood to the site of injury, allowing patients to produce hemostatic plugs or clots to avert life-threatening complications. Hemab Therapeutics was established in 2017 in Copenhagen, Hovedstaden by Johan H. Faber and Mads Behrndt.

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.

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.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

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.

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.

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.

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.

Invetx, Inc. is a biotechnology company based in Boston, Massachusetts, specializing in the development of protein-based therapeutics for animal health care and veterinary medicine. The company employs a team of veterinary scientists and clinicians dedicated to the discovery and development of biotherapeutics for both pets and farm animals. Invetx aims to transform the animal health industry by leveraging biopharmaceutical technologies to improve health outcomes significantly. By combining deep expertise in veterinary science with innovative therapeutic solutions, Invetx seeks to advance the future of animal care, providing effective treatments that cater to the needs of various animal populations.

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.

Rondo Therapeutics is a biopharmaceutical company based in Hayward, California, specializing in the discovery and development of innovative bispecific antibodies aimed at activating the immune system to combat cancer. Founded in 2021, the company focuses on creating cancer immunotherapies specifically for solid tumor types that do not respond to existing immune checkpoint inhibitors. By addressing areas of unmet medical need, Rondo Therapeutics aims to advance novel therapeutic options for patients facing challenging cancer diagnoses.

Centauri Therapeutics is a biotechnology company that develops novel immunotherapeutics employing a dual‑mechanism platform. Its molecules combine a cell‑surface antigen‑binding moiety with a glycan effector domain that harnesses the body’s pre‑existing polyclonal antibodies. This design amplifies complement fixation, antibody‑dependent cellular cytotoxicity, and phagocytosis, enabling targeted cell death. The company’s pre‑clinical studies demonstrate potent enhancement of complement fixation and opsonophagocytosis for both antibacterial and oncology agents, and in vivo proof of concept in multiple infection models. Centauri also utilizes alphamer technology, chemically synthesizing molecules that redirect endogenous antibodies to specific pathogens, providing a new approach to treating infectious diseases. The company focuses on life‑threatening conditions, aiming to bring innovative therapies to patients.

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.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

Shanghai ZhenGe Biotechnology Co., Ltd. is a research and development company that specializes in the creation of drugs and protein therapeutics. Founded in 2017 and based in Shanghai, China, the company focuses on various aspects of drug development, including mammalian cell line development, upstream and downstream processing, and formulation development. Zhenge Biotech also offers contract development and manufacturing services, as well as assistance with regulatory filings such as IND and BLA. Additionally, the company provides a research platform for antibody development, transient infection processes, N-Glycosylation, and CAR-T technologies. Through these services, Zhenge Biotech supports the pharmaceutical industry in advancing innovative therapeutic solutions.

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.

Orbis Medicines is a company that develops a macrocyclic chemistry and computational platform for oral macrocycle drug discovery. The platform generates macrocycle candidates, nCycles, optimized for oral bioavailability to advance therapeutics that historically faced development hurdles. Its pipeline centers on nCycle candidates targeting validated biologic targets that are typically addressed by injectable drugs, with the goal of enabling oral delivery of such therapies. The platform combines chemistry and computational methods to accelerate drug discovery toward challenging targets in unmet-need indications, supporting therapeutics designed to improve patient 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.

Hemab is a company that creates preventative treatments for bleeding and thrombosis problems. The medicines developed by the business aggregate, modify, balance, and at times recruit endogenous clotting components already present in the blood to the site of injury, allowing patients to produce hemostatic plugs or clots to avert life-threatening complications. Hemab Therapeutics was established in 2017 in Copenhagen, Hovedstaden by Johan H. Faber and Mads Behrndt.

Muna Therapeutics is a biopharmaceutical company dedicated to discovering and developing therapies aimed at slowing or halting devastating neurodegenerative diseases such as Parkinson's, Alzheimer's, Frontotemporal Dementia, and Multiple Sclerosis.

Tribune Therapeutics is a preclinical biopharmaceutical company focused on developing medicines to treat fibrotic diseases by targeting extracellular matrix–associated proteins central to scar formation, with the aim of halting progression across diverse tissues and conditions.

Numab Therapeutics is an oncology-focused biopharmaceutical company that designs and develops antibody-based and multispecific therapeutics for cancer, inflammatory diseases, autoimmunity, and related severe conditions. It employs a plug-and-play platform that reduces the randomness of discovery and aims to predictably yield mono- or multispecific antibody fragments with tailored pharmacokinetic properties to engage multiple targets. Founded in 2011 and based in Wädenswil, Switzerland, Numab pursues immuno-oncology and inflammation programs through this multispecific approach, prioritizing therapies with improved selectivity and potential clinical efficacy.

Hummingbird Bioscience employs systems biology to uncover disease mechanisms and uses computational platforms for discovering and engineering innovative biotherapeutics. Its pipeline includes HMBD-001 (anti-HER3 antibody) and HMBD-002 (anti-VISTA antibody), both in cancer treatment. The company collaborates strategically with Cancer Research UK and Amgen.

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.

Exscientia is a biotechnology company that leverages artificial intelligence and machine learning to enhance the discovery and design of novel therapeutic compounds. By integrating human expertise with advanced computational techniques, Exscientia accelerates the development of small-molecule drug candidates, aiming to produce safe and effective treatments for clinical testing. The company's innovative approach seeks to revolutionize the pharmaceutical industry by improving the precision and efficiency of drug discovery, ultimately enhancing patient outcomes.

Exscientia is a biotechnology company that leverages artificial intelligence and machine learning to enhance the discovery and design of novel therapeutic compounds. By integrating human expertise with advanced computational techniques, Exscientia accelerates the development of small-molecule drug candidates, aiming to produce safe and effective treatments for clinical testing. The company's innovative approach seeks to revolutionize the pharmaceutical industry by improving the precision and efficiency of drug discovery, ultimately enhancing patient outcomes.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

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.

Hemab is a company that creates preventative treatments for bleeding and thrombosis problems. The medicines developed by the business aggregate, modify, balance, and at times recruit endogenous clotting components already present in the blood to the site of injury, allowing patients to produce hemostatic plugs or clots to avert life-threatening complications. Hemab Therapeutics was established in 2017 in Copenhagen, Hovedstaden by Johan H. Faber and Mads Behrndt.

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.

Stargazer Pharmaceuticals Inc. is a biopharmaceutical company based in Boston, Massachusetts, founded in 2018. It specializes in developing and manufacturing therapies specifically for rare eye diseases. The company is particularly focused on addressing Stargardt's disease, a juvenile retinal degeneration condition for which there are currently no available treatments. By concentrating on these rare diseases, Stargazer Pharmaceuticals aims to provide healthcare professionals with innovative therapeutic options for patients suffering from such conditions. The company is currently operating in stealth mode as it advances its research and development efforts.

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.

F2G Ltd is a biotechnology company focused on developing innovative therapies for serious fungal infections. Founded in 1998 and based in Manchester, United Kingdom, F2G primarily works on its F3 series of anti-mold compounds, specifically targeting antifungal agents against Aspergillus and other filamentous molds. Utilizing proprietary genomics technology known as MycoBank, the company identifies essential gene targets in fungi, which informs its drug development process. F2G has diversified its approach to include chemistry-driven discovery, leveraging a strong development team to advance its patented compounds. The company's efforts aim to address difficult-to-treat fungal pathogens that pose significant mortality risks, particularly for immunocompromised patients, thereby enhancing treatment options in the pharmaceutical industry.

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.

Bolt Biotherapeutics is a biotechnology company developing cancer immunotherapies based on the Boltbody platform, which links tumor-targeting antibodies to immune-stimulating payloads to activate innate and adaptive immune responses against solid tumors. The platform aims to transform non-responsive, or cold, tumors into immunologically active ones by delivering immune stimulants at the tumor site. The company’s pipeline includes BDC-1001 as a monotherapy for HER2-expressing solid tumors, with additional candidates such as BDC-3042 and Next-Gen ISAC designed to broaden its immuno-oncology program. Bolt Biotherapeutics focuses on myeloid biology and tumor-targeted therapies to leverage immune mechanisms for cancer treatment.

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.

Antag Therapeutics ApS is a biotechnology company based in Copenhagen, Denmark, founded in 2017. The company specializes in developing peptide drugs aimed at treating dietary-related metabolic diseases, including obesity, diabetes, and non-alcoholic fatty liver disease. Antag Therapeutics focuses on creating peptide-based GIP receptor antagonists designed to manage these conditions by reducing blood flow and lipid uptake in adipose tissue. Through its innovative approach, the company seeks to address significant health challenges associated with metabolic disorders.

NodThera is a UK-based biotechnology company focused on developing novel inhibitors of the NLRP3 inflammasome for treating inflammatory and neuroinflammatory diseases. Established in 2016, it operates laboratories in Little Chesterford (UK) and Seattle (US), with corporate offices in Lexington (US). The company's primary research involves inflammasomes, multi-protein complexes targeting conditions such as arthritis, atherosclerosis, Alzheimer's disease, diabetes, and certain cancers.

Exscientia is a biotechnology company that leverages artificial intelligence and machine learning to enhance the discovery and design of novel therapeutic compounds. By integrating human expertise with advanced computational techniques, Exscientia accelerates the development of small-molecule drug candidates, aiming to produce safe and effective treatments for clinical testing. The company's innovative approach seeks to revolutionize the pharmaceutical industry by improving the precision and efficiency of drug discovery, ultimately enhancing patient outcomes.