Forbion Capital Partners

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Pheon Therapeutics is an Antibody-Drug Conjugate (ADC) specialist that creates ADCs for a variety of difficult-to-treat cancers. Its lead program is a first-in-class ADC against a novel target that is highly expressed in solid tumors across a broad range of hard-to-treat cancer types. The company was founded in 2022 and is based in London, England.

SynOx Therapeutics is a clinical-stage biopharmaceutical company based in Dublin, Ireland, that specializes in the development of innovative therapies for cancer. The company's primary focus is on emactuzumab, a humanized monoclonal antibody targeting CSF-1R, aimed at depleting macrophages within tumor tissues. This therapy is being investigated for its potential in treating diffuse tenosynovial giant cell tumors and other macrophage-mediated diseases. Established in 2019, SynOx Therapeutics is backed by experienced investors in the biopharmaceutical field, emphasizing its commitment to addressing the unmet clinical needs of patients and improving their quality of life through advanced treatment options.

Engrail is forging a new direction to reduce the enormous burden of diseases that impact the nervous system. We unite biological insights with clinically meaningful solutions to build and catalyze a diversified portfolio of transformative medicines. Harnessing our rigorous scientific approach to identify the most promising therapies, we leverage our flexible transaction model to advance assets with validated mechanisms and efficiently move them through development to commercialization.

Enterprise Therapeutics Ltd. is a drug discovery company based in Brighton, United Kingdom, founded in 2014. It focuses on the research and development of innovative therapies for respiratory diseases, specifically targeting chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. The company aims to create disease-modifying therapies that address the underlying mechanisms of mucus congestion in the lungs, which can lead to breathing difficulties and increased susceptibility to lung infections. By developing these novel treatments, Enterprise Therapeutics seeks to enhance the quality of life for patients suffering from these chronic respiratory conditions.

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

NewAmsterdam Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for cardio-metabolic diseases. The company aims to enhance patient care for individuals with metabolic disorders by addressing the shortcomings of currently available treatments. Its leading product, obicetrapib, is a next-generation, oral, low-dose CETP inhibitor designed to effectively lower LDL cholesterol levels while potentially overcoming the limitations associated with existing therapies.

CatalYm GmbH, founded in 2016 and based in Munich, Germany, is a biotechnology company focused on developing innovative immunotherapies for cancer patients. The company has identified GDF-15 as a key regulator of the immune system within the tumor microenvironment. By neutralizing GDF-15, CatalYm aims to enhance the immune response against solid tumors, potentially leading to meaningful clinical outcomes. Its CTL-002 program is designed to demonstrate clinical proof-of-concept across multiple solid tumor indications, offering the prospect of expanding treatment options for cancer patients. Through its approach, CatalYm seeks to transform the tumor microenvironment, making tumors more accessible to the immune system and improving both survival rates and quality of life for patients.

Inversago Pharma Inc. is a preclinical-stage biotechnology company based in Montreal, Canada, focused on developing innovative therapies targeting metabolic and fibrotic disorders. The company specializes in peripherally-restricted CB1 receptor inverse agonists, which aim to offer new treatment options for conditions such as Prader-Willi syndrome, diabetes, and related metabolic disorders. Inversago Pharma's research includes addressing complications associated with diabetic kidney disease, non-alcoholic steatohepatitis, and various fibrotic diseases, including interstitial lung disease. Founded in 2015, the company is dedicated to enhancing patient outcomes through its pioneering approach to CB1 blockade.

VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in developing innovative treatments for severe rare diseases with significant unmet medical needs. Its primary product candidate, Apraglutide, is a long-acting synthetic analog of glucagon-like peptide-2 (GLP-2) designed to enhance the intestine's ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support. VectivBio aims to deliver transformational therapies that significantly improve the lives of patients and their families, rather than offering only incremental benefits over existing treatments.

Pheon Therapeutics is an Antibody-Drug Conjugate (ADC) specialist that creates ADCs for a variety of difficult-to-treat cancers. Its lead program is a first-in-class ADC against a novel target that is highly expressed in solid tumors across a broad range of hard-to-treat cancer types. The company was founded in 2022 and is based in London, England.

F2G Ltd is a biotechnology company based in Manchester, United Kingdom, that specializes in developing innovative therapies for serious fungal diseases. Established in 1998, F2G focuses on its F3 series of antifungal compounds, particularly targeting challenging fungi such as Aspergillus and other filamentous molds that pose significant health risks. The company leverages its proprietary genomics technology, MycoBank, to identify critical gene targets in fungi, facilitating the creation of effective antifungal agents. Since its initial funding in 2002, F2G has expanded its research and development capabilities, enabling it to advance its patented compounds and contribute to addressing life-threatening fungal infections that affect individuals with compromised immune systems.

Anaveon are developing IL-2 complexes which selectively promote effector T cell functions. Our compounds act as effective immune adjuvants with a broad therapeutic window and marked preclinical efficacy against cancer either as monotherapy or in combination with other therapies.

Rectify Pharmaceuticals is focused on creating disease-modifying precision therapies aimed at addressing the root causes of serious genetic diseases. The company specializes in developing positive functional modulators (PFMs) that restore the function of ABC transporters, which are critical for cellular processes. By targeting these underlying mechanisms, Rectify aims to provide innovative treatment solutions that improve the health outcomes of patients suffering from various genetic conditions. Their research and development efforts encompass multiple therapeutic areas, reflecting a commitment to advancing precision medicine in the field of genetic disorders.

Amphista Therapeutics Limited is a biopharmaceutical company based in Motherwell, United Kingdom, established in 2017. The company specializes in targeted protein degradation technology to develop innovative cancer therapeutics. Amphista's approach focuses on harnessing the body's natural processes to selectively and efficiently degrade disease-causing proteins, aiming to create first-in-class treatments. By advancing novel methodologies in targeted protein degradation, Amphista seeks to improve therapeutic efficacy and modulate protein abundance linked to disease progression, ultimately enhancing patient outcomes in oncology.

CatalYm GmbH, founded in 2016 and based in Munich, Germany, is a biotechnology company focused on developing innovative immunotherapies for cancer patients. The company has identified GDF-15 as a key regulator of the immune system within the tumor microenvironment. By neutralizing GDF-15, CatalYm aims to enhance the immune response against solid tumors, potentially leading to meaningful clinical outcomes. Its CTL-002 program is designed to demonstrate clinical proof-of-concept across multiple solid tumor indications, offering the prospect of expanding treatment options for cancer patients. Through its approach, CatalYm seeks to transform the tumor microenvironment, making tumors more accessible to the immune system and improving both survival rates and quality of life for patients.

SynOx Therapeutics is a clinical-stage biopharmaceutical company based in Dublin, Ireland, that specializes in the development of innovative therapies for cancer. The company's primary focus is on emactuzumab, a humanized monoclonal antibody targeting CSF-1R, aimed at depleting macrophages within tumor tissues. This therapy is being investigated for its potential in treating diffuse tenosynovial giant cell tumors and other macrophage-mediated diseases. Established in 2019, SynOx Therapeutics is backed by experienced investors in the biopharmaceutical field, emphasizing its commitment to addressing the unmet clinical needs of patients and improving their quality of life through advanced treatment options.

Inversago Pharma Inc. is a preclinical-stage biotechnology company based in Montreal, Canada, focused on developing innovative therapies targeting metabolic and fibrotic disorders. The company specializes in peripherally-restricted CB1 receptor inverse agonists, which aim to offer new treatment options for conditions such as Prader-Willi syndrome, diabetes, and related metabolic disorders. Inversago Pharma's research includes addressing complications associated with diabetic kidney disease, non-alcoholic steatohepatitis, and various fibrotic diseases, including interstitial lung disease. Founded in 2015, the company is dedicated to enhancing patient outcomes through its pioneering approach to CB1 blockade.

AM Pharma B.V. is a biopharmaceutical company based in Bunnik, the Netherlands, focused on developing and commercializing novel therapeutics for inflammatory and infectious diseases. Established in 2000, the company specializes in pre-clinical and clinical development using endogenous proteins and peptides derived from the human body. Its key products include alkaline phosphatase, aimed at treating acute kidney injury and ulcerative colitis, and the immune response amplifying peptide hLF1-11, which targets drug-resistant hospital-acquired infections. AM Pharma is committed to providing innovative therapies that enhance patient quality of life by addressing critical medical needs.

Azafaros B.V., based in Leiden, the Netherlands, is focused on developing therapeutic agents for rare metabolic disorders, particularly lysosomal storage disorders (LSDs). The company specializes in oral administration of aza-sugar compounds, which were discovered by Professor Hans Aerts at Leiden University and Amsterdam UMC and are exclusively licensed to Azafaros. These innovative agents aim to address key disease pathways by targeting the central nervous system and interfering with the metabolism of glycosphingolipids. Through a unique dual mode of action, Azafaros seeks to improve the lives of patients suffering from these severe and often life-threatening inherited diseases.

NorthSea Therapeutics B.V. is a biotechnology company focused on developing and manufacturing innovative therapeutics for metabolic, inflammatory, and fibrotic diseases. Established in 2017 and based in Naarden, the Netherlands, the company utilizes its proprietary Structurally Engineered Fatty Acid (SEFA) technology to create unique oral therapeutic candidates. One of its leading products, icosabutate, is designed to address inflammatory and liver diseases by offering a novel approach to treatment. The company's pipeline includes candidates that aim to improve conditions such as dyslipidemia, insulin resistance, hepatic inflammation, and fibrosis, providing healthcare providers with effective options for patient care.

AM Pharma B.V. is a biopharmaceutical company based in Bunnik, the Netherlands, focused on developing and commercializing novel therapeutics for inflammatory and infectious diseases. Established in 2000, the company specializes in pre-clinical and clinical development using endogenous proteins and peptides derived from the human body. Its key products include alkaline phosphatase, aimed at treating acute kidney injury and ulcerative colitis, and the immune response amplifying peptide hLF1-11, which targets drug-resistant hospital-acquired infections. AM Pharma is committed to providing innovative therapies that enhance patient quality of life by addressing critical medical needs.

SANIFIT is a biotechnology company that develops treatments for patients with calcification disorders.

Inflazome Ltd. is a biotechnology company based in Dublin, Ireland, founded in 2016. It specializes in developing orally available drugs aimed at addressing unmet clinical needs in inflammatory diseases by targeting the inflammasome, a component involved in the inflammatory process. The company’s innovative therapies are designed to block inflammasome signals, effectively tackling various inflammatory-driven conditions. These include orphan diseases, central nervous system disorders such as Alzheimer's and Parkinson's, systemic inflammatory diseases like cardiovascular and pulmonary conditions, and applications in ophthalmology and dermatology. By targeting the root causes of inflammation, Inflazome enables the development of precise treatments for a range of diseases, thereby enhancing therapeutic options for healthcare providers. As of September 2020, Inflazome operates as a subsidiary of Roche Holding AG.

OMEICOS Therapeutics GmbH is a Berlin-based company founded in 2013 that specializes in developing novel therapeutics for cardiovascular diseases and other health conditions. The company focuses on small molecule therapies derived from natural metabolites of omega-3 fatty acids, which exhibit a unique mode of action. These substances activate an endogenous cardio-protective signaling pathway, providing both anti-arrhythmic effects and curative benefits by stabilizing heart rhythm and preventing electrical and structural remodeling of the heart. In addition to cardiovascular applications, OMEICOS also explores treatments for inflammatory and ophthalmic diseases, aiming to deliver safe and effective therapeutic options for various medical indications.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

Gotham Therapeutics is a biotechnology company based in New York that focuses on developing a novel class of drugs targeting epitranscriptomics machinery. Founded in 2017, the company aims to provide new treatment options for patients with cancers, autoimmune disorders, and neurodegenerative diseases. Its approach involves creating small molecules that modify the activity of messenger ribonucleic acid (mRNA) to influence protein transcription. This innovative strategy allows Gotham Therapeutics to potentially optimize therapeutic outcomes for individuals suffering from serious health conditions.

Enterprise Therapeutics Ltd. is a drug discovery company based in Brighton, United Kingdom, founded in 2014. It focuses on the research and development of innovative therapies for respiratory diseases, specifically targeting chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. The company aims to create disease-modifying therapies that address the underlying mechanisms of mucus congestion in the lungs, which can lead to breathing difficulties and increased susceptibility to lung infections. By developing these novel treatments, Enterprise Therapeutics seeks to enhance the quality of life for patients suffering from these chronic respiratory conditions.

Escalier Biosciences B.V., established in 2016 and headquartered in Nijmegen, Netherlands, is a biopharmaceutical company focused on developing novel small molecule therapeutics for autoimmune disorders, particularly psoriasis. The company is advancing its lead drug candidates, which are designed to target ROR?t, through late-stage preclinical studies. Escalier's therapeutics enhance penetration in dermal tissues, optimizing target engagement at sites of inflammation while reducing the potential for systemic side effects and immune suppression. In addition to its operations in the Netherlands, Escalier also maintains an office in Encinitas, California.

NorthSea Therapeutics B.V. is a biotechnology company focused on developing and manufacturing innovative therapeutics for metabolic, inflammatory, and fibrotic diseases. Established in 2017 and based in Naarden, the Netherlands, the company utilizes its proprietary Structurally Engineered Fatty Acid (SEFA) technology to create unique oral therapeutic candidates. One of its leading products, icosabutate, is designed to address inflammatory and liver diseases by offering a novel approach to treatment. The company's pipeline includes candidates that aim to improve conditions such as dyslipidemia, insulin resistance, hepatic inflammation, and fibrosis, providing healthcare providers with effective options for patient care.

Hookipa Pharma Inc. is a clinical-stage biopharmaceutical company focused on developing immunotherapeutics for infectious diseases and cancers using its proprietary arenavirus platform. The company’s lead product candidate for infectious diseases, HB-101, is currently undergoing a randomized double-blinded Phase II clinical trial for patients awaiting kidney transplants from cytomegalovirus-positive donors. In oncology, Hookipa is advancing its candidates HB-201 and HB-202, which target human papillomavirus-positive cancers and are in preclinical studies. The company employs its innovative VaxWave and TheraT platforms to stimulate robust immune responses, generating high levels of antigen-specific killer T cells and antibodies, which are essential for effective treatment. Additionally, Hookipa has partnered with Gilead Sciences to develop therapies aimed at achieving functional cures for chronic hepatitis B and HIV infections. Founded in 2011 and based in New York, Hookipa Pharma is dedicated to transforming the landscape of immune therapies.

Replimune Group is a biotechnology company focused on developing oncolytic immune-gene therapies for cancer treatment. Utilizing its proprietary Immulytic platform, Replimune aims to create product candidates that activate the immune system against tumors. The company's lead candidate, RP1, is a modified herpes simplex virus currently undergoing Phase I/II clinical trials for various solid tumors and Phase II trials specifically for cutaneous squamous cell carcinoma. Additionally, Replimune is developing RP2, which targets CTLA-4 to enhance immune response, and RP3, designed to express immune-activating proteins that stimulate T cells. Founded in 2015 and based in Woburn, Massachusetts, Replimune seeks to advance its therapies through clinical trials and explore combinations with other immuno-oncology treatments to enhance efficacy.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

Hookipa Pharma Inc. is a clinical-stage biopharmaceutical company focused on developing immunotherapeutics for infectious diseases and cancers using its proprietary arenavirus platform. The company’s lead product candidate for infectious diseases, HB-101, is currently undergoing a randomized double-blinded Phase II clinical trial for patients awaiting kidney transplants from cytomegalovirus-positive donors. In oncology, Hookipa is advancing its candidates HB-201 and HB-202, which target human papillomavirus-positive cancers and are in preclinical studies. The company employs its innovative VaxWave and TheraT platforms to stimulate robust immune responses, generating high levels of antigen-specific killer T cells and antibodies, which are essential for effective treatment. Additionally, Hookipa has partnered with Gilead Sciences to develop therapies aimed at achieving functional cures for chronic hepatitis B and HIV infections. Founded in 2011 and based in New York, Hookipa Pharma is dedicated to transforming the landscape of immune therapies.

Rigontec is the leader in RIG-I targeting RNA therapeutics developing novel immuno-oncology treatment options.

CatalYm GmbH, founded in 2016 and based in Munich, Germany, is a biotechnology company focused on developing innovative immunotherapies for cancer patients. The company has identified GDF-15 as a key regulator of the immune system within the tumor microenvironment. By neutralizing GDF-15, CatalYm aims to enhance the immune response against solid tumors, potentially leading to meaningful clinical outcomes. Its CTL-002 program is designed to demonstrate clinical proof-of-concept across multiple solid tumor indications, offering the prospect of expanding treatment options for cancer patients. Through its approach, CatalYm seeks to transform the tumor microenvironment, making tumors more accessible to the immune system and improving both survival rates and quality of life for patients.

Allecra Therapeutics, a clinical stage biopharmaceutical company, was established in 2013 and is dedicated to the development of novel treatments to combat multi drug-resistant bacterial infections. Allecra has a novel β-lactamase inhibitor in Phase 2 clinical development. By inhibiting β-lactamases Allecra’s new medicine directly tackles emerging resistance mechanisms of certain hard-to-treat bacteria. These resistance mechanisms are often found in hospital-acquired Gram-negative bacterial infections, and are of particular concern to doctors, healthcare providers, to patients and their families and to governments around the world. Allecra Therapeutics is situated in the BioValley Life Sciences region in the Upper Rhein valley whichencompasses northwest Switzerland, southwestern Germany and the Alsace region of France

Akarna Therapeutics is a biopharmaceutical company with offices in San Diego and Cambridge (UK) that is developing novel small molecule therapeutics that target inflammatory and fibrotic diseases. Our lead program is a non-bile acid FXR agonist that is potentially a best-in-class therapeutic for the treatment of nonalcoholic steatohepatitis (NASH), a progressive form of fatty-liver disease for which there are no approved therapies. Akarna’s lead candidate is currently in preclinical, IND-enabling toxicology and safety pharmacology studies with first-in-human studies planned for early 2017.

Exosome Diagnostics, Inc. specializes in the development and commercialization of biofluid-based molecular diagnostic tests aimed at enhancing personalized precision healthcare. The company employs a proprietary exosome technology platform that extracts and analyzes molecular information from exosomes present in various biofluids, including blood, urine, and cerebrospinal fluid. This approach enables non-invasive diagnostics for serious diseases, significantly reducing the need for tissue biopsies. One of its key products is the MedOncAlyzer 170, a liquid biopsy pan-cancer panel that simultaneously analyzes exosomal RNA and circulating tumor DNA in a single assay to identify actionable mutations across multiple cancer types. By combining RNA, DNA, and protein analysis, Exosome Diagnostics provides comprehensive molecular insights that can transform the detection, diagnosis, treatment, and monitoring of cancer and other serious conditions. The company also collaborates with pharmaceutical firms to enhance research and development processes, including biomarker discovery and the creation of companion diagnostics. Founded in 2008 and based in Cambridge, Massachusetts, Exosome Diagnostics operates as a subsidiary of Bio-Techne Corporation.

Replimune Group is a biotechnology company focused on developing oncolytic immune-gene therapies for cancer treatment. Utilizing its proprietary Immulytic platform, Replimune aims to create product candidates that activate the immune system against tumors. The company's lead candidate, RP1, is a modified herpes simplex virus currently undergoing Phase I/II clinical trials for various solid tumors and Phase II trials specifically for cutaneous squamous cell carcinoma. Additionally, Replimune is developing RP2, which targets CTLA-4 to enhance immune response, and RP3, designed to express immune-activating proteins that stimulate T cells. Founded in 2015 and based in Woburn, Massachusetts, Replimune seeks to advance its therapies through clinical trials and explore combinations with other immuno-oncology treatments to enhance efficacy.

SANIFIT is a biotechnology company that develops treatments for patients with calcification disorders.

Exosome Diagnostics, Inc. specializes in the development and commercialization of biofluid-based molecular diagnostic tests aimed at enhancing personalized precision healthcare. The company employs a proprietary exosome technology platform that extracts and analyzes molecular information from exosomes present in various biofluids, including blood, urine, and cerebrospinal fluid. This approach enables non-invasive diagnostics for serious diseases, significantly reducing the need for tissue biopsies. One of its key products is the MedOncAlyzer 170, a liquid biopsy pan-cancer panel that simultaneously analyzes exosomal RNA and circulating tumor DNA in a single assay to identify actionable mutations across multiple cancer types. By combining RNA, DNA, and protein analysis, Exosome Diagnostics provides comprehensive molecular insights that can transform the detection, diagnosis, treatment, and monitoring of cancer and other serious conditions. The company also collaborates with pharmaceutical firms to enhance research and development processes, including biomarker discovery and the creation of companion diagnostics. Founded in 2008 and based in Cambridge, Massachusetts, Exosome Diagnostics operates as a subsidiary of Bio-Techne Corporation.

Replimune Group is a biotechnology company focused on developing oncolytic immune-gene therapies for cancer treatment. Utilizing its proprietary Immulytic platform, Replimune aims to create product candidates that activate the immune system against tumors. The company's lead candidate, RP1, is a modified herpes simplex virus currently undergoing Phase I/II clinical trials for various solid tumors and Phase II trials specifically for cutaneous squamous cell carcinoma. Additionally, Replimune is developing RP2, which targets CTLA-4 to enhance immune response, and RP3, designed to express immune-activating proteins that stimulate T cells. Founded in 2015 and based in Woburn, Massachusetts, Replimune seeks to advance its therapies through clinical trials and explore combinations with other immuno-oncology treatments to enhance efficacy.

Rigontec is the leader in RIG-I targeting RNA therapeutics developing novel immuno-oncology treatment options.

Insmed Incorporated is a global biopharmaceutical company dedicated to improving the lives of patients with serious and rare diseases. Its flagship product, ARIKAYCE (amikacin liposome inhalation suspension), is approved in the United States for treating Mycobacterium avium complex (MAC) lung disease, targeting adult patients who have limited or no alternative treatment options. MAC lung disease is a rare and potentially fatal infection that can lead to significant lung damage. In addition to ARIKAYCE, Insmed's clinical pipeline features Brensocatib, a novel oral reversible inhibitor of dipeptidyl peptidase 1, which shows promise for treating non-cystic fibrosis bronchiectasis and other inflammatory conditions. Another candidate in development is INS1009, an inhaled formulation of a treprostinil prodrug aimed at addressing rare pulmonary disorders, including pulmonary arterial hypertension.

Exosome Diagnostics, Inc. specializes in the development and commercialization of biofluid-based molecular diagnostic tests aimed at enhancing personalized precision healthcare. The company employs a proprietary exosome technology platform that extracts and analyzes molecular information from exosomes present in various biofluids, including blood, urine, and cerebrospinal fluid. This approach enables non-invasive diagnostics for serious diseases, significantly reducing the need for tissue biopsies. One of its key products is the MedOncAlyzer 170, a liquid biopsy pan-cancer panel that simultaneously analyzes exosomal RNA and circulating tumor DNA in a single assay to identify actionable mutations across multiple cancer types. By combining RNA, DNA, and protein analysis, Exosome Diagnostics provides comprehensive molecular insights that can transform the detection, diagnosis, treatment, and monitoring of cancer and other serious conditions. The company also collaborates with pharmaceutical firms to enhance research and development processes, including biomarker discovery and the creation of companion diagnostics. Founded in 2008 and based in Cambridge, Massachusetts, Exosome Diagnostics operates as a subsidiary of Bio-Techne Corporation.

Ario Pharma Ltd is focused on developing therapeutic drugs for respiratory conditions, particularly through the evaluation of XEN-D0501, a potential leading TRPV1 inhibitor. The company is conducting two Phase 2 clinical trials to assess the anti-tussive properties of this drug. In addition to its respiratory portfolio, Ario also works on small-molecule TRPV1 antagonists aimed at treating neuropathic and inflammatory pain. The firm is backed by a skilled development team and supported by leading experts in respiratory diseases, ensuring a robust approach to drug development.

Hookipa Pharma Inc. is a clinical-stage biopharmaceutical company focused on developing immunotherapeutics for infectious diseases and cancers using its proprietary arenavirus platform. The company’s lead product candidate for infectious diseases, HB-101, is currently undergoing a randomized double-blinded Phase II clinical trial for patients awaiting kidney transplants from cytomegalovirus-positive donors. In oncology, Hookipa is advancing its candidates HB-201 and HB-202, which target human papillomavirus-positive cancers and are in preclinical studies. The company employs its innovative VaxWave and TheraT platforms to stimulate robust immune responses, generating high levels of antigen-specific killer T cells and antibodies, which are essential for effective treatment. Additionally, Hookipa has partnered with Gilead Sciences to develop therapies aimed at achieving functional cures for chronic hepatitis B and HIV infections. Founded in 2011 and based in New York, Hookipa Pharma is dedicated to transforming the landscape of immune therapies.

uniQure is a pioneering gene therapy company recognized for developing the first gene therapy product to receive regulatory approval in the European Union. Its initial product, Glybera, was authorized in October 2012 for treating patients with lipoprotein lipase deficiency, a rare and severe metabolic disorder. uniQure specializes in creating innovative gene therapies that offer long-term and potentially curative benefits for genetic and acquired diseases. The company utilizes a modular technology platform and a proprietary manufacturing process to develop its treatments. Currently, uniQure is expanding its pipeline of adeno-associated virus (AAV)-based gene therapies, focusing on conditions such as hemophilia, Huntington's disease, and cardiovascular diseases through collaborations, including one with Bristol Myers Squibb. While the company initially targets orphan diseases, it also aims to address chronic and degenerative diseases affecting larger populations.

Allecra Therapeutics, a clinical stage biopharmaceutical company, was established in 2013 and is dedicated to the development of novel treatments to combat multi drug-resistant bacterial infections. Allecra has a novel β-lactamase inhibitor in Phase 2 clinical development. By inhibiting β-lactamases Allecra’s new medicine directly tackles emerging resistance mechanisms of certain hard-to-treat bacteria. These resistance mechanisms are often found in hospital-acquired Gram-negative bacterial infections, and are of particular concern to doctors, healthcare providers, to patients and their families and to governments around the world. Allecra Therapeutics is situated in the BioValley Life Sciences region in the Upper Rhein valley whichencompasses northwest Switzerland, southwestern Germany and the Alsace region of France

Dezima Pharma B.V., founded in 2012 and based in Naarden, the Netherlands, focuses on developing protein-based compounds aimed at treating cardiovascular diseases associated with dyslipidemia. The company is known for its innovative approach in creating a cholesteryl ester transfer protein inhibitor, which serves as a pharmacological therapy to reduce low-density lipoprotein cholesterol levels. By lowering these cholesterol levels, Dezima Pharma aims to provide clinicians with effective treatment options for patients suffering from dyslipidemia and related cardiovascular conditions.

Promedior, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing therapeutics for rare fibrotic and inflammatory diseases. The company’s primary targets include conditions such as myelofibrosis, idiopathic pulmonary fibrosis, age-related macular degeneration, and diabetic retinopathy. Promedior's drug portfolio features PRM-151, a recombinant form of human pentaxin-2 protein, and PRM-167, a variant designed for intravitreal delivery. These therapeutics are aimed at regulating monocyte-derived cell populations, which are critical in fibrotic, inflammatory, and autoimmune diseases. By specifically addressing these cells at injury sites, Promedior seeks to promote tissue healing and minimize systemic side effects commonly associated with existing therapies. The company was incorporated in 2006 and was previously known as Fibrotix, Inc.

Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D, for cerebral adrenoleukodystrophy. In oncology, Bluebird Bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.

Santaris Pharma A/S is a privately held clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies. The LNA Drug Platform and Drug Discovery Engine developed by Santaris Pharma A/S combine the company’s proprietary LNA chemistry with its highly specialized and targeted drug development capabilities to rapidly deliver potent single-stranded LNA-based drug candidates across a multitude of disease states. The company’s research and development activities focus on infectious diseases and cardiometabolic disorders, while partnerships with major pharmaceutical companies include a range of therapeutic areas including cancer, cardiovascular disease, infectious and inflammatory diseases, and rare genetic disorders. The company has strategic partnerships with miRagen Therapeutics, Shire PLC, Pfizer, GlaxoSmithKline, and Enzon Pharmaceuticals. As part of its broad patent estate, the company holds exclusive worldwide rights to manufacture, have manufactured and sell products that contain LNA as an active ingredient for studies performed with a view to obtaining marketing approval. Santaris Pharma A/S, founded in 2003, is headquartered in Denmark with operations in the United States.

Promedior, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing therapeutics for rare fibrotic and inflammatory diseases. The company’s primary targets include conditions such as myelofibrosis, idiopathic pulmonary fibrosis, age-related macular degeneration, and diabetic retinopathy. Promedior's drug portfolio features PRM-151, a recombinant form of human pentaxin-2 protein, and PRM-167, a variant designed for intravitreal delivery. These therapeutics are aimed at regulating monocyte-derived cell populations, which are critical in fibrotic, inflammatory, and autoimmune diseases. By specifically addressing these cells at injury sites, Promedior seeks to promote tissue healing and minimize systemic side effects commonly associated with existing therapies. The company was incorporated in 2006 and was previously known as Fibrotix, Inc.

Argenx is a global immunology company based in the Netherlands, dedicated to enhancing the lives of individuals affected by severe autoimmune diseases and cancer. The company employs advanced antibody engineering technology to develop treatments for rare autoimmune conditions. Its notable product, Vyvgart (efgartigimod), received initial approval in the United States in December 2021 for the treatment of generalized myasthenia gravis, followed by approvals in Europe and Japan in 2022. Through its innovative approaches, Argenx aims to address unmet medical needs in the field of immunology.

Amakem is a kinase platform company focusing on ophthalmology and is developing new treatments for serious eye conditions. Amakem's product pipeline is based on its unique ‘Localized Drug Action' platform which is designed to generate safe and effective novel kinase inhibitors that minimize systemic exposure with the aim of reducing side effects.

Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D, for cerebral adrenoleukodystrophy. In oncology, Bluebird Bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.

Cardoz AB manufactures anti-inflammatory pharmaceuticals to combat heart and vascular disorders. The company was founded in 2006 and is based in Stockholm, Sweden.

Cytheris is a privately held clinical-stage biopharmaceutical company focused on research and development of therapies for immune modulation. These drugs aim at reconstituting and enhancing the immune system of patients suffering from cancer, chronic viral or bacterial infections such as HIV, HCV, and HBV or lympho-depleting treatments such as chemotherapy, radiotherapy, bone marrow transplantation (BMT) and hematopoietic cell transplantation (HCT).

Exosome Diagnostics, Inc. specializes in the development and commercialization of biofluid-based molecular diagnostic tests aimed at enhancing personalized precision healthcare. The company employs a proprietary exosome technology platform that extracts and analyzes molecular information from exosomes present in various biofluids, including blood, urine, and cerebrospinal fluid. This approach enables non-invasive diagnostics for serious diseases, significantly reducing the need for tissue biopsies. One of its key products is the MedOncAlyzer 170, a liquid biopsy pan-cancer panel that simultaneously analyzes exosomal RNA and circulating tumor DNA in a single assay to identify actionable mutations across multiple cancer types. By combining RNA, DNA, and protein analysis, Exosome Diagnostics provides comprehensive molecular insights that can transform the detection, diagnosis, treatment, and monitoring of cancer and other serious conditions. The company also collaborates with pharmaceutical firms to enhance research and development processes, including biomarker discovery and the creation of companion diagnostics. Founded in 2008 and based in Cambridge, Massachusetts, Exosome Diagnostics operates as a subsidiary of Bio-Techne Corporation.

Promedior, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing therapeutics for rare fibrotic and inflammatory diseases. The company’s primary targets include conditions such as myelofibrosis, idiopathic pulmonary fibrosis, age-related macular degeneration, and diabetic retinopathy. Promedior's drug portfolio features PRM-151, a recombinant form of human pentaxin-2 protein, and PRM-167, a variant designed for intravitreal delivery. These therapeutics are aimed at regulating monocyte-derived cell populations, which are critical in fibrotic, inflammatory, and autoimmune diseases. By specifically addressing these cells at injury sites, Promedior seeks to promote tissue healing and minimize systemic side effects commonly associated with existing therapies. The company was incorporated in 2006 and was previously known as Fibrotix, Inc.

Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D, for cerebral adrenoleukodystrophy. In oncology, Bluebird Bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.

Transave, Inc., a biotechnology company, develops inhaled pharmaceuticals for the treatment of lung diseases. The company’s liposomal technology allows the release of drug in the lung's microenvironment. Its products also include Arikace for the treatment of gram-negative lung infections and pseudomonas infections in cystic fibrosis patients; and cisplatin lipid complex for the treatment of cancers affecting the lungs. Transave, Inc. was founded in 1997 and is headquartered in Monmouth Junction, New Jersey. As of December 1, 2010, Transave, Inc. operates as a subsidiary of Insmed Incorporated.

Santaris Pharma A/S is a privately held clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies. The LNA Drug Platform and Drug Discovery Engine developed by Santaris Pharma A/S combine the company’s proprietary LNA chemistry with its highly specialized and targeted drug development capabilities to rapidly deliver potent single-stranded LNA-based drug candidates across a multitude of disease states. The company’s research and development activities focus on infectious diseases and cardiometabolic disorders, while partnerships with major pharmaceutical companies include a range of therapeutic areas including cancer, cardiovascular disease, infectious and inflammatory diseases, and rare genetic disorders. The company has strategic partnerships with miRagen Therapeutics, Shire PLC, Pfizer, GlaxoSmithKline, and Enzon Pharmaceuticals. As part of its broad patent estate, the company holds exclusive worldwide rights to manufacture, have manufactured and sell products that contain LNA as an active ingredient for studies performed with a view to obtaining marketing approval. Santaris Pharma A/S, founded in 2003, is headquartered in Denmark with operations in the United States.

Fovea Pharmaceuticals is a French biopharmaceutical company. It discovers and develops drugs for the treatment of ocular diseases. The company offers FOV1101 Prednisporin for the treatment of allergic conjunctivitis; FOV2302, a proprietary plasma kallikrein-kinin inhibitor for the systemic treatment of hereditary angioedema; FOV2304 for the treatment of diabetic macular edema; and FOV2501, an intravitreal formulation of RdCVF designed to treat retinitis pigmentosa with extension to dry age-related macular degeneration. Fovea Pharmaceuticals was founded in 2005 and is based in Paris, France. As of October 30, 2009, it operates as a subsidiary of Sanofi.

AM Pharma B.V. is a biopharmaceutical company based in Bunnik, the Netherlands, focused on developing and commercializing novel therapeutics for inflammatory and infectious diseases. Established in 2000, the company specializes in pre-clinical and clinical development using endogenous proteins and peptides derived from the human body. Its key products include alkaline phosphatase, aimed at treating acute kidney injury and ulcerative colitis, and the immune response amplifying peptide hLF1-11, which targets drug-resistant hospital-acquired infections. AM Pharma is committed to providing innovative therapies that enhance patient quality of life by addressing critical medical needs.

Pieris Pharmaceuticals is a clinical-stage biotechnology company that develops drugs to fight cancer, severe asthma, anemia, and other medical conditions with unmet medical needs. The company applies its proprietary Anticalin® technology to the drug development processes. Pieris Pharmaceuticals was founded in 2001 and is based in Freising, Bayern.

Xention Limited is a biopharmaceutical company based in Cambridge, United Kingdom, that focuses on discovering and developing innovative ion channel-modulating drugs. Founded in 2002, the company aims to address significant unmet medical needs by creating therapies for conditions such as atrial fibrillation, overactive bladder, pain, and autoimmune diseases. Xention develops drug candidates that target two novel yet well-characterized ion channels, striving to produce breakthrough therapies that enhance treatment options in these areas. The company, originally known as Xention Discovery Ltd., rebranded to Xention Limited in October 2006.

Borean Pharma ApS operates as a drug discovery and development company. The company's technology platform enables to design, develop, and produce pharmaceutical protein products. The company focuses on recombinant protein technology, as well as on antibody biopharmaceuticals. It offers protein products, antibody analogues, and CTLD library, as well as TNF antagonist for rheumatoid arthritis. The company was founded in 1997 and is based in Aarhus, Denmark.

Roche Glycart develops antibody drug candidates, which include GA101, a humanized anti-CD20 monoclonal antibody to increase direct- and immune-mediated target cell death; and GA201, a humanized glycoengineered antibody for solid tumors.

Acorda Therapeutics is a biopharmaceutical company focused on developing and commercializing therapies for neurological disorders in the United States and internationally. The company’s primary product, Ampyra (dalfampridine), is an oral medication approved to enhance walking in patients with multiple sclerosis. Additionally, Acorda markets Inbrija, an inhalation powder for the treatment of OFF periods in individuals with Parkinson's disease. The company's pipeline includes rHIgM22, which has completed Phase I trials for multiple sclerosis, and Cimaglermin alfa, currently in Phase Ib trials for heart failure. Acorda has established a collaboration with Biogen for the development and commercialization of Ampyra. Founded in 1995 and based in Ardsley, New York, Acorda Therapeutics aims to improve the quality of life for those affected by neurological conditions.

Acadia Pharmaceuticals Inc. is a biopharmaceutical company dedicated to developing and commercializing small molecule drugs for central nervous system disorders. Founded in 1993 and headquartered in San Diego, California, the company is best known for its product NUPLAZID (pimavanserin), which is approved for treating hallucinations and delusions associated with Parkinson’s disease psychosis. Acadia is actively advancing pimavanserin through Phase III clinical trials for dementia-related psychosis and as an adjunctive treatment for schizophrenia, as well as exploring its use for major depressive disorder. The company operates two subsidiaries in Sweden and Denmark and is committed to discovering novel treatments for conditions such as Alzheimer's and schizophrenia, while also seeking to in-license or acquire complementary products. Acadia holds several patent applications related to its proprietary technology, including innovative methods for drug discovery and development.

Transave, Inc., a biotechnology company, develops inhaled pharmaceuticals for the treatment of lung diseases. The company’s liposomal technology allows the release of drug in the lung's microenvironment. Its products also include Arikace for the treatment of gram-negative lung infections and pseudomonas infections in cystic fibrosis patients; and cisplatin lipid complex for the treatment of cancers affecting the lungs. Transave, Inc. was founded in 1997 and is headquartered in Monmouth Junction, New Jersey. As of December 1, 2010, Transave, Inc. operates as a subsidiary of Insmed Incorporated.