Sofinnova Investments

Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, that specializes in developing small molecule therapies for severe, rare musculoskeletal diseases. Founded in 2017, the company employs a precision medicine approach to create innovative treatments targeting various muscle disorders, including Duchenne and Becker muscular dystrophies, as well as limb girdle muscular dystrophies. Utilizing its proprietary muscle-focused drug discovery platform, Edgewise integrates expertise in muscle biology and small molecule engineering to identify and develop therapies that address genetically defined muscle disorders. The company emphasizes a holistic drug discovery method that assesses integrated muscle function, enabling it to create orally bioavailable therapies aimed at improving outcomes for patients with significant unmet medical needs in neuromuscular and cardiac diseases.

Atsena Therapeutics is a clinical-stage gene therapy company focused on developing treatments for inherited retinal diseases that can lead to blindness. Their clinical pipeline includes therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Atsena employs an adeno-associated virus (AAV) technology platform, including a novel spreading capsid, to specifically target these retinal disorders. The company aims to prevent vision loss caused by genetic mutations through innovative ocular gene therapy solutions. With a commitment to advancing treatments for some of the most challenging forms of inherited retinal disease, Atsena Therapeutics seeks to improve the lives of patients affected by these conditions.

Avenzo Therapeutics is a clinical-stage company focused on developing innovative oncology therapies aimed at treating cancer. The company’s platform is dedicated to creating novel treatments specifically for underserved cancers, including rare and genomically defined types. By advancing a pipeline of targeted drug candidates, Avenzo Therapeutics seeks to enhance treatment options for both researchers and patients, addressing significant gaps in current cancer care.

Seaport Therapeutics is a clinical stage biopharmaceutical company focused on developing innovative neuropsychiatric medicines for patients with significant unmet needs. The company's approach emphasizes the creation of treatments that offer oral bioavailability, which allows the medications to bypass first-pass metabolism. This method aims to reduce hepatotoxicity and other adverse side effects commonly associated with neuropsychiatric treatments. By addressing these critical issues, Seaport Therapeutics seeks to improve the quality of life for individuals living with neuropsychiatric disorders.

Seaport Therapeutics is a clinical stage biopharmaceutical company focused on developing innovative neuropsychiatric medicines for patients with significant unmet needs. The company's approach emphasizes the creation of treatments that offer oral bioavailability, which allows the medications to bypass first-pass metabolism. This method aims to reduce hepatotoxicity and other adverse side effects commonly associated with neuropsychiatric treatments. By addressing these critical issues, Seaport Therapeutics seeks to improve the quality of life for individuals living with neuropsychiatric disorders.

Avenzo Therapeutics is a clinical-stage company focused on developing innovative oncology therapies aimed at treating cancer. The company’s platform is dedicated to creating novel treatments specifically for underserved cancers, including rare and genomically defined types. By advancing a pipeline of targeted drug candidates, Avenzo Therapeutics seeks to enhance treatment options for both researchers and patients, addressing significant gaps in current cancer care.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing innovative small-molecule therapies for neurological disorders. The company focuses on understanding the intricate biology of neuronal receptor complexes, which are essential for proper neuronal function and signaling. By mapping and targeting these receptor systems, Rapport Therapeutics aims to create precision medicines that can effectively address the unmet needs of patients suffering from central nervous system disorders. Through its foundational research, the company seeks to transform treatment options and improve outcomes for individuals with these challenging conditions.

Alkeus Pharmaceuticals is a Boston-based clinical-stage biopharmaceutical company dedicated to developing treatments for serious and untreatable ophthalmic conditions. The company's primary focus is on its lead drug candidate, an orally delivered compound aimed at addressing Stargardt disease and age-related macular degeneration. This innovative treatment has the potential to help medical professionals manage symptoms of irreversible vision loss early in life, offering hope to patients affected by these debilitating eye diseases.

Antiva Biosciences is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral drugs to treat human papillomavirus (HPV) infections. Founded in 2012 and previously known as Hera Therapeutics, the company aims to prevent cancer by addressing precancerous lesions associated with HPV. Antiva's innovative approach involves localized therapeutics and small molecule antivirals designed to improve health outcomes for patients suffering from diseases linked to HPV and other viruses. The company's clinical programs target high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias, utilizing a medicinal chemistry platform that emphasizes the development and commercialization of direct-acting antivirals.

ArriVent Biopharma is a clinical-stage biopharmaceutical company focused on developing innovative treatments for currently untreatable cancers. The company is committed to addressing unmet medical needs in oncology by leveraging its team's extensive drug development experience. ArriVent aims to maximize the potential of its lead candidate, furmonertinib, while advancing a pipeline of novel therapeutics, including next-generation antibody-drug conjugates. With an initial emphasis on solid tumors, ArriVent seeks to bring differentiated medicines from development through to commercialization, ultimately improving patient outcomes in cancer treatment.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.

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.

Antiva Biosciences is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral drugs to treat human papillomavirus (HPV) infections. Founded in 2012 and previously known as Hera Therapeutics, the company aims to prevent cancer by addressing precancerous lesions associated with HPV. Antiva's innovative approach involves localized therapeutics and small molecule antivirals designed to improve health outcomes for patients suffering from diseases linked to HPV and other viruses. The company's clinical programs target high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias, utilizing a medicinal chemistry platform that emphasizes the development and commercialization of direct-acting antivirals.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, established in 2018. The company is dedicated to developing clinical candidates targeting cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at treating resistant hypertension and primary aldosteronism. CinCor Pharma's mission is to advance its promising clinical candidates toward marketing approval, emphasizing the development of innovative therapies for patients suffering from cardio-renal conditions.

Gyroscope is a clinical-stage gene therapy company that specializes in developing treatments for retinal diseases, particularly focusing on Age-related Macular Degeneration (AMD), a major cause of blindness. The company leverages advancements in understanding the complement system's role in eye diseases and uses gene therapy as a therapeutic approach. Gyroscope's lead investigational therapy, GT005, is designed to be administered as a one-time treatment under the retina, aiming to restore balance to an overactive complement system by enhancing the production of the Complement Factor I protein. Currently, GT005 is undergoing evaluation in multiple clinical trials, including a Phase I/II trial named FOCUS and two Phase II trials called EXPLORE and HORIZON. Founded in 2016 in Stevenage, Herefordshire, by Andrew Lotery, David Kavanagh, and Peter Lachmann, Gyroscope seeks to provide innovative solutions for patients suffering from retinal diseases.

XyloCor Therapeutics, based in Newtown Square, Pennsylvania, is a biopharmaceutical company dedicated to developing gene therapies for the treatment of cardiovascular diseases, particularly advanced coronary artery disease. Its lead product candidate, XC001, is currently in clinical development for patients suffering from refractory angina, addressing critical unmet medical needs for those who have exhausted conventional treatment options. Additionally, the company is developing XC002, aimed at regenerating cardiac tissue in patients with compromised heart function due to previous heart attacks and resultant heart failure. XyloCor's innovative gene therapy technologies focus on stimulating the formation of new coronary blood vessels, thereby improving blood supply to areas of the heart that are insufficiently perfused. The company collaborates with Weill Cornell Medical College to enhance its therapeutic approaches and outcomes for patients.

Vera Therapeutics, a biotechnology company based in South San Francisco, California, specializes in developing innovative biologic therapeutics aimed at treating serious immunological diseases and genetic disorders. Founded in 2016 and renamed in March 2020, the company's research focuses on transformative treatments that can significantly improve patient outcomes. One of its key product candidates is atacicept, a fusion protein designed for self-administration via a weekly subcutaneous injection. Atacicept works by inhibiting both B lymphocyte stimulator and a proliferation-inducing ligand, which are involved in the production of autoantibodies that contribute to various autoimmune diseases. Additionally, Vera Therapeutics is exploring gene-editing technologies that hold the potential to address conditions such as sickle cell disease and cystic fibrosis.

Atsena Therapeutics is a clinical-stage gene therapy company focused on developing treatments for inherited retinal diseases that can lead to blindness. Their clinical pipeline includes therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Atsena employs an adeno-associated virus (AAV) technology platform, including a novel spreading capsid, to specifically target these retinal disorders. The company aims to prevent vision loss caused by genetic mutations through innovative ocular gene therapy solutions. With a commitment to advancing treatments for some of the most challenging forms of inherited retinal disease, Atsena Therapeutics seeks to improve the lives of patients affected by these conditions.

Aerovate Therapeutics is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative treatments for patients with rare cardiopulmonary diseases. The company's primary focus is on AV-101, a proprietary inhaled dry powder formulation of the drug imatinib, designed specifically for the treatment of pulmonary arterial hypertension (PAH). This formulation enables targeted delivery of the medication directly to affected lung tissues, thereby minimizing systemic side effects commonly associated with traditional treatments. Established in 2018, Aerovate aims to provide meaningful improvements in the lives of those suffering from these serious conditions.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various forms of cancer, including hematological cancers and solid tumors. The company's primary area of research centers on targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), a cell-surface protein found in numerous tumors but absent in normal tissues, which makes it a promising target for cancer therapy. VelosBio's lead product, VLS-101, is a ROR1-directed ADC that has shown positive preclinical results and aims to provide new treatment options for patients. Founded in 2017, VelosBio operates under the umbrella of Merck & Co., Inc. as of December 2020, focusing on developing targeted therapies that can be administered alone or in combination with other treatments across a wide range of cancer types.

Checkmate Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing novel immunotherapies for cancer treatment. The company specializes in CpG oligonucleotides, which are designed to stimulate an anti-tumor T-cell response while counteracting the mechanisms that tumors use to evade the immune response. Checkmate aims to enhance the effectiveness of existing immunotherapies and provide new treatment options for patients through its innovative approaches. Since its incorporation in 2015, Checkmate has established strategic partnerships with major pharmaceutical companies, including Merck KGaA and Pfizer, to advance its clinical programs and broaden the impact of its research in the field of cancer immunotherapy.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and addressing neurodegenerative diseases. The company's lead candidate, ATH-1017, is a small hepatocyte growth factor/MET activator designed to penetrate the blood-brain barrier and is currently undergoing various clinical trials for the treatment of Alzheimer's and Parkinson's diseases. Additionally, Athira is advancing other candidates, including ATH-1019, which is in preclinical development for depression, and ATH-1018, aimed at treating peripheral neuropathy. Founded in 2011 and originally named M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its commitment to innovative therapies that positively impact individuals affected by brain disorders.

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.

Vera Therapeutics, a biotechnology company based in South San Francisco, California, specializes in developing innovative biologic therapeutics aimed at treating serious immunological diseases and genetic disorders. Founded in 2016 and renamed in March 2020, the company's research focuses on transformative treatments that can significantly improve patient outcomes. One of its key product candidates is atacicept, a fusion protein designed for self-administration via a weekly subcutaneous injection. Atacicept works by inhibiting both B lymphocyte stimulator and a proliferation-inducing ligand, which are involved in the production of autoantibodies that contribute to various autoimmune diseases. Additionally, Vera Therapeutics is exploring gene-editing technologies that hold the potential to address conditions such as sickle cell disease and cystic fibrosis.

Intrepida Bio, Inc. is a biotechnology company focused on developing innovative cancer therapies by targeting the body's innate immune system. Incorporated in 2016 and based in San Diego, California, the company aims to create medicines that modulate innate immunity to combat cancer and other diseases. Utilizing proprietary monoclonal antibodies, Intrepida Bio works on blocking novel targets, specifically BAG3 and its receptor IFITM-2, to disrupt the tumor-supportive environment. Through this approach, the company seeks to provide patients with effective treatment options that may improve outcomes in cancer care.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, established in 2018. The company is dedicated to developing clinical candidates targeting cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at treating resistant hypertension and primary aldosteronism. CinCor Pharma's mission is to advance its promising clinical candidates toward marketing approval, emphasizing the development of innovative therapies for patients suffering from cardio-renal conditions.

Karuna Therapeutics is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, dedicated to developing innovative therapies for neuropsychiatric disorders and pain. Its lead product candidate, KarXT, is an oral modulator that has completed a Phase II clinical trial for treating acute psychosis in schizophrenia patients and is undergoing a Phase Ib clinical trial for various central nervous system disorders, including cognitive and negative symptoms of schizophrenia, Alzheimer’s, and dementia-related psychosis. The company is also developing additional muscarinic-targeted drug candidates to address unmet medical needs in these areas. Founded in 2009, Karuna Therapeutics has established partnerships with organizations such as Eli Lilly and Company and PureTech Health LLC, focusing on advancing its drug discovery efforts through collaborations with Charles River Laboratories and PsychoGenics Inc.

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.

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.

XyloCor Therapeutics, based in Newtown Square, Pennsylvania, is a biopharmaceutical company dedicated to developing gene therapies for the treatment of cardiovascular diseases, particularly advanced coronary artery disease. Its lead product candidate, XC001, is currently in clinical development for patients suffering from refractory angina, addressing critical unmet medical needs for those who have exhausted conventional treatment options. Additionally, the company is developing XC002, aimed at regenerating cardiac tissue in patients with compromised heart function due to previous heart attacks and resultant heart failure. XyloCor's innovative gene therapy technologies focus on stimulating the formation of new coronary blood vessels, thereby improving blood supply to areas of the heart that are insufficiently perfused. The company collaborates with Weill Cornell Medical College to enhance its therapeutic approaches and outcomes for patients.

NextCure, Inc. is a clinical-stage biopharmaceutical company based in Beltsville, Maryland, that specializes in the discovery and development of novel immunomedicines aimed at treating cancer and other immune-related diseases. The company focuses on restoring normal immune function through its lead product candidate, NC318, currently in Phase 2 clinical trials for advanced or metastatic solid tumors. In addition, NextCure is developing NC410, an immunomedicine designed to inhibit immune suppression mediated by Leukocyte-Associated Immunoglobulin-like Receptor 1. The company's research also includes preclinical programs targeting various immunomodulatory proteins associated with tumors and inflamed tissues. NextCure has established collaborations, including a licensing agreement with Yale University and a research partnership with Eli Lilly and Company, to further its innovative therapeutic approaches. Founded in 2015, NextCure is dedicated to advancing first-in-class immunotherapy solutions.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various forms of cancer, including hematological cancers and solid tumors. The company's primary area of research centers on targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), a cell-surface protein found in numerous tumors but absent in normal tissues, which makes it a promising target for cancer therapy. VelosBio's lead product, VLS-101, is a ROR1-directed ADC that has shown positive preclinical results and aims to provide new treatment options for patients. Founded in 2017, VelosBio operates under the umbrella of Merck & Co., Inc. as of December 2020, focusing on developing targeted therapies that can be administered alone or in combination with other treatments across a wide range of cancer types.

Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Malvern, Pennsylvania, dedicated to developing innovative therapeutics aimed at enhancing the effectiveness of radiotherapy for cancer patients. The company's lead product candidate, avasopasem manganese (GC4419), is a small molecule dismutase mimetic currently undergoing Phase III clinical trials for the treatment of radiation-induced severe oral mucositis in patients with head and neck cancer. Additionally, GC4419 is being evaluated in Phase IIa trials for the treatment of radiotherapy-induced esophagitis in lung cancer patients. Galera is also advancing another candidate, GC4711, which is in Phase I/II trials for pancreatic and lung cancers. The company's research is focused on targeting oxygen metabolic pathways to improve patient outcomes in radiation therapy, and GC4419 has received Fast Track and Breakthrough Therapy designations from the U.S. Food and Drug Administration.

Antiva Biosciences is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral drugs to treat human papillomavirus (HPV) infections. Founded in 2012 and previously known as Hera Therapeutics, the company aims to prevent cancer by addressing precancerous lesions associated with HPV. Antiva's innovative approach involves localized therapeutics and small molecule antivirals designed to improve health outcomes for patients suffering from diseases linked to HPV and other viruses. The company's clinical programs target high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias, utilizing a medicinal chemistry platform that emphasizes the development and commercialization of direct-acting antivirals.

ENYO Pharma SA is a clinical-stage biopharmaceutical company based in Lyon, France, that focuses on the discovery and development of innovative treatments for acute and chronic viral infections. Established in 2014 by a team of scientists from the Infectiology Research Center, the company utilizes a proprietary platform to identify intracellular therapeutic targets and develop small molecule therapeutics. ENYO Pharma's research primarily targets significant human infecting viruses, with current programs aimed at treating chronic hepatitis B and severe influenza. The company's pipeline includes a lead compound, EYP001, which modulates FXR to reduce the cccDNA reservoir and inhibit the expression of viral proteins, along with EYP002, which is in preclinical studies. With an emphasis on addressing unmet medical needs in infectious diseases and metabolic disorders, ENYO Pharma has established collaborations with prominent research institutions to enhance its drug development efforts.

Neurana Pharmaceuticals, Inc. is a pharmaceutical company based in San Diego, California, focusing on the development of treatments for neuromuscular conditions. Founded in 2013, the company specializes in a novel therapeutic compound, Tolperisone, which addresses acute and painful muscle spasms in the neck and back as well as other musculoskeletal issues. Unlike traditional muscle relaxants, Tolperisone provides relief without causing sedation, offering a unique solution for patients experiencing spasticity and muscle discomfort.

Merus B.V. is a clinical-stage biomedical company focused on the discovery and development of antibody-based biopharmaceuticals, specifically in the field of immuno-oncology. The company utilizes its proprietary Oligoclonics technology to produce a class of human antibodies, which allows for the creation of a mixture of therapeutic antibodies targeting a common antigen from a single cell clone. Additionally, Merus develops bispecific antibody therapeutics known as Biclonics, which are designed to engage multiple targets simultaneously. The company's pipeline includes several product candidates such as MCLA-128, MCLA-117, and MCLA-158, among others, all aimed at addressing various cancer indications.

Kiniksa Pharmaceuticals, Ltd. is a clinical-stage biopharmaceutical company dedicated to discovering, acquiring, developing, and commercializing therapeutic medicines for patients with debilitating diseases that have significant unmet medical needs. The company has a diverse pipeline of clinical-stage product candidates, including Rilonacept, which is undergoing Phase III trials for recurrent pericarditis; Mavrilimumab, a monoclonal antibody in Phase II trials for giant cell arteritis; and KPL-716, currently in Phase 2a trials for prurigo nodularis and Phase 1a trials for atopic dermatitis. Kiniksa also has preclinical candidates, such as KPL-404, which targets the CD40/CD40L interaction in immune responses. Additionally, the company is collaborating with Kite Pharma to explore the combination of Yescarta and Mavrilimumab in patients with relapsed or refractory Large B-Cell lymphoma. Founded in 2015 and headquartered in Hamilton, Bermuda, Kiniksa Pharmaceuticals aims to address multiple autoimmune and autoinflammatory conditions through its innovative approaches.

Y-mAbs Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New York, dedicated to developing and commercializing innovative antibody-based therapies for cancer treatment. The company is advancing several product candidates, including naxitamab, currently in Phase 2 clinical trials for pediatric patients with relapsed osteosarcoma and high-risk neuroblastoma, as well as GD2-GD3 Vaccine, also in Phase 2 for high-risk neuroblastoma. Additionally, omburtamab is under development for desmoplastic small round cell tumors and pediatric central nervous system cancers. Y-mAbs is also exploring huB7-H3 for B7-H3 positive adult solid tumors and huCD33 BsAb for hematological cancers. The company collaborates with Memorial Sloan Kettering Cancer Center and Massachusetts Institute of Technology to enhance its antibody constructs through the SADA-BiDE pre-targeted radioimmunotherapy platform. Y-mAbs aims to bring multiple cancer therapies to FDA approval, with a focus on reducing long-term toxicities associated with traditional chemotherapies. Its first FDA-approved product, DANYELZA, targets GD2, a marker in various neuroectoderm-derived tumors and sarcomas.

Entasis Therapeutics is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts. It focuses on developing novel antibacterial products to treat serious infections caused by multi-drug resistant Gram-negative bacteria. The company's pipeline includes Sulbactam-durlobactam (SUL-DUR) for Acinetobacter infections, Zoliflodacin for gonorrhea, ETX0282CPDP for urinary tract infections, and ETX0462 for multidrug-resistant Gram-negative infections. Entasis has collaboration agreements with Zai Lab for the development of SUL-DUR.

Checkmate Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing novel immunotherapies for cancer treatment. The company specializes in CpG oligonucleotides, which are designed to stimulate an anti-tumor T-cell response while counteracting the mechanisms that tumors use to evade the immune response. Checkmate aims to enhance the effectiveness of existing immunotherapies and provide new treatment options for patients through its innovative approaches. Since its incorporation in 2015, Checkmate has established strategic partnerships with major pharmaceutical companies, including Merck KGaA and Pfizer, to advance its clinical programs and broaden the impact of its research in the field of cancer immunotherapy.

Iterum Therapeutics plc is a clinical-stage pharmaceutical company focused on developing anti-infective therapies to address multi-drug resistant pathogens. Headquartered in Dublin, Ireland, the company is advancing its lead product, sulopenem, a novel penem compound available in both oral and intravenous formulations. Currently in Phase III clinical trials, sulopenem is being evaluated for the treatment of uncomplicated urinary tract infections, complicated urinary tract infections, and complicated intra-abdominal infections. Iterum aims to provide effective solutions to combat antibiotic resistance, particularly against a range of gram-negative, gram-positive, and anaerobic bacteria that are resistant to existing antibiotics. Founded in 2015, Iterum Therapeutics seeks to improve the lives of patients affected by serious and life-threatening infections globally.

Antiva Biosciences is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral drugs to treat human papillomavirus (HPV) infections. Founded in 2012 and previously known as Hera Therapeutics, the company aims to prevent cancer by addressing precancerous lesions associated with HPV. Antiva's innovative approach involves localized therapeutics and small molecule antivirals designed to improve health outcomes for patients suffering from diseases linked to HPV and other viruses. The company's clinical programs target high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias, utilizing a medicinal chemistry platform that emphasizes the development and commercialization of direct-acting antivirals.

Ascendis Pharma A/S is a biopharmaceutical company based in Hellerup, Denmark, focused on developing prodrug therapies to address unmet medical needs. Leveraging its innovative TransCon technology platform, the company aims to create superior prodrugs across various drug classes, including proteins, peptides, and small molecules. Its pipeline includes TransCon growth hormone, which has completed Phase III clinical trials for treating growth hormone deficiency in children, and TransCon parathyroid hormone, currently in Phase II trials for hypoparathyroidism. Additionally, TransCon CNP is in Phase II development for achondroplasia. Ascendis Pharma also explores preclinical studies in oncology and employs strategic collaborations to enhance its product development, aiming for systemic and localized drug delivery. The company primarily operates in North America, with additional activities in Europe and China.

InCarda Therapeutics is a clinical-stage biopharmaceutical company based in Newark, California, focused on developing innovative therapies for cardiovascular diseases. The company specializes in an inhaled drug delivery system aimed at treating acute paroxysmal atrial fibrillation, allowing patients to administer treatment quickly during the onset of symptoms. Its lead product, InRhythm, is an inhaled formulation of an antiarrhythmic medication designed to provide rapid relief from arrhythmic events. This method allows for effective medication delivery directly to cardiac tissue, which enhances treatment efficacy and simplifies the management of atrial arrhythmias. InCarda Therapeutics has completed Phase 1 clinical trials and is progressing through Phase 2 with its lead product.

Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Malvern, Pennsylvania, dedicated to developing innovative therapeutics aimed at enhancing the effectiveness of radiotherapy for cancer patients. The company's lead product candidate, avasopasem manganese (GC4419), is a small molecule dismutase mimetic currently undergoing Phase III clinical trials for the treatment of radiation-induced severe oral mucositis in patients with head and neck cancer. Additionally, GC4419 is being evaluated in Phase IIa trials for the treatment of radiotherapy-induced esophagitis in lung cancer patients. Galera is also advancing another candidate, GC4711, which is in Phase I/II trials for pancreatic and lung cancers. The company's research is focused on targeting oxygen metabolic pathways to improve patient outcomes in radiation therapy, and GC4419 has received Fast Track and Breakthrough Therapy designations from the U.S. Food and Drug Administration.

Iterum Therapeutics plc is a clinical-stage pharmaceutical company focused on developing anti-infective therapies to address multi-drug resistant pathogens. Headquartered in Dublin, Ireland, the company is advancing its lead product, sulopenem, a novel penem compound available in both oral and intravenous formulations. Currently in Phase III clinical trials, sulopenem is being evaluated for the treatment of uncomplicated urinary tract infections, complicated urinary tract infections, and complicated intra-abdominal infections. Iterum aims to provide effective solutions to combat antibiotic resistance, particularly against a range of gram-negative, gram-positive, and anaerobic bacteria that are resistant to existing antibiotics. Founded in 2015, Iterum Therapeutics seeks to improve the lives of patients affected by serious and life-threatening infections globally.

NextCure, Inc. is a clinical-stage biopharmaceutical company based in Beltsville, Maryland, that specializes in the discovery and development of novel immunomedicines aimed at treating cancer and other immune-related diseases. The company focuses on restoring normal immune function through its lead product candidate, NC318, currently in Phase 2 clinical trials for advanced or metastatic solid tumors. In addition, NextCure is developing NC410, an immunomedicine designed to inhibit immune suppression mediated by Leukocyte-Associated Immunoglobulin-like Receptor 1. The company's research also includes preclinical programs targeting various immunomodulatory proteins associated with tumors and inflamed tissues. NextCure has established collaborations, including a licensing agreement with Yale University and a research partnership with Eli Lilly and Company, to further its innovative therapeutic approaches. Founded in 2015, NextCure is dedicated to advancing first-in-class immunotherapy solutions.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Checkmate Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing novel immunotherapies for cancer treatment. The company specializes in CpG oligonucleotides, which are designed to stimulate an anti-tumor T-cell response while counteracting the mechanisms that tumors use to evade the immune response. Checkmate aims to enhance the effectiveness of existing immunotherapies and provide new treatment options for patients through its innovative approaches. Since its incorporation in 2015, Checkmate has established strategic partnerships with major pharmaceutical companies, including Merck KGaA and Pfizer, to advance its clinical programs and broaden the impact of its research in the field of cancer immunotherapy.

Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.

Antiva Biosciences is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral drugs to treat human papillomavirus (HPV) infections. Founded in 2012 and previously known as Hera Therapeutics, the company aims to prevent cancer by addressing precancerous lesions associated with HPV. Antiva's innovative approach involves localized therapeutics and small molecule antivirals designed to improve health outcomes for patients suffering from diseases linked to HPV and other viruses. The company's clinical programs target high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias, utilizing a medicinal chemistry platform that emphasizes the development and commercialization of direct-acting antivirals.

Merus B.V. is a clinical-stage biomedical company focused on the discovery and development of antibody-based biopharmaceuticals, specifically in the field of immuno-oncology. The company utilizes its proprietary Oligoclonics technology to produce a class of human antibodies, which allows for the creation of a mixture of therapeutic antibodies targeting a common antigen from a single cell clone. Additionally, Merus develops bispecific antibody therapeutics known as Biclonics, which are designed to engage multiple targets simultaneously. The company's pipeline includes several product candidates such as MCLA-128, MCLA-117, and MCLA-158, among others, all aimed at addressing various cancer indications.

Edge Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapies for acute neurological conditions, particularly in hospital settings. Founded in 2009 and based in Berkeley Heights, New Jersey, the company aims to transform treatment paradigms for serious medical conditions with significant unmet needs. Its lead product candidate, EG-1962, is currently being evaluated in the Phase 3 NEWTON 2 study for the treatment of aneurysmal subarachnoid hemorrhage, a condition resulting from bleeding around the brain due to a ruptured aneurysm. Additionally, Edge is developing EG-1964 as a potential prophylactic treatment for chronic subdural hematoma, aiming to prevent recurrent bleeding on the brain's surface. The company employs its proprietary Precisa platform to create polymer-based therapeutics that deliver treatment directly to the site of injury, minimizing impact on surrounding tissues.

aTyr Pharma is a biotherapeutics company based in San Diego, California, specializing in the discovery and development of innovative medicines utilizing novel immunological pathways. The company focuses primarily on ATYR1923, a clinical-stage fusion protein designed to down-regulate immune engagement in interstitial lung diseases by binding to the neuropilin-2 receptor. This candidate is currently undergoing Phase 1b/2a clinical trials. aTyr Pharma has established a strong intellectual property portfolio centered around Physiocrine-based compositions and therapeutic applications, particularly in immunomodulation disorders related to inflammation and immunity. Additionally, the company has formed collaborations with several institutions, including the University of Nebraska Medical Center, CSL Behring, Boston Children’s Hospital, and the Medical University of South Carolina, to advance its research and development efforts. Founded in 2005 by a prominent scientist from The Scripps Research Institute, aTyr is supported by leading life sciences investors.

Chiasma, Inc. is a late-stage biopharmaceutical company based in Needham, Massachusetts, that specializes in developing oral medications for rare and serious chronic diseases. Utilizing its proprietary Transient Permeability Enhancer technology, Chiasma aims to transform injectable drugs into oral formulations, enhancing their absorption and potentially enabling new therapeutic indications. The company is currently focused on MYCAPSSA, an oral octreotide capsule designed for adult patients with acromegaly, a condition characterized by excessive growth hormone production. MYCAPSSA is undergoing Phase III clinical trials in the United States and is also in the process of seeking regulatory approval in the European Union. Founded in 2001, Chiasma is dedicated to improving treatment options for patients with debilitating conditions through innovative drug delivery methods.

NextCure, Inc. is a clinical-stage biopharmaceutical company based in Beltsville, Maryland, that specializes in the discovery and development of novel immunomedicines aimed at treating cancer and other immune-related diseases. The company focuses on restoring normal immune function through its lead product candidate, NC318, currently in Phase 2 clinical trials for advanced or metastatic solid tumors. In addition, NextCure is developing NC410, an immunomedicine designed to inhibit immune suppression mediated by Leukocyte-Associated Immunoglobulin-like Receptor 1. The company's research also includes preclinical programs targeting various immunomodulatory proteins associated with tumors and inflamed tissues. NextCure has established collaborations, including a licensing agreement with Yale University and a research partnership with Eli Lilly and Company, to further its innovative therapeutic approaches. Founded in 2015, NextCure is dedicated to advancing first-in-class immunotherapy solutions.

Iterum Therapeutics plc is a clinical-stage pharmaceutical company focused on developing anti-infective therapies to address multi-drug resistant pathogens. Headquartered in Dublin, Ireland, the company is advancing its lead product, sulopenem, a novel penem compound available in both oral and intravenous formulations. Currently in Phase III clinical trials, sulopenem is being evaluated for the treatment of uncomplicated urinary tract infections, complicated urinary tract infections, and complicated intra-abdominal infections. Iterum aims to provide effective solutions to combat antibiotic resistance, particularly against a range of gram-negative, gram-positive, and anaerobic bacteria that are resistant to existing antibiotics. Founded in 2015, Iterum Therapeutics seeks to improve the lives of patients affected by serious and life-threatening infections globally.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Ascendis Pharma A/S is a biopharmaceutical company based in Hellerup, Denmark, focused on developing prodrug therapies to address unmet medical needs. Leveraging its innovative TransCon technology platform, the company aims to create superior prodrugs across various drug classes, including proteins, peptides, and small molecules. Its pipeline includes TransCon growth hormone, which has completed Phase III clinical trials for treating growth hormone deficiency in children, and TransCon parathyroid hormone, currently in Phase II trials for hypoparathyroidism. Additionally, TransCon CNP is in Phase II development for achondroplasia. Ascendis Pharma also explores preclinical studies in oncology and employs strategic collaborations to enhance its product development, aiming for systemic and localized drug delivery. The company primarily operates in North America, with additional activities in Europe and China.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Civitas Therapeutics is a biopharmaceutical company dedicated to developing and commercializing innovative pulmonary delivery therapies. Utilizing its proprietary ARCUS technology, the company creates therapeutics aimed at treating central nervous system and respiratory disorders. Civitas Therapeutics serves patients across the United States, focusing on delivering transformative treatment options to improve health outcomes.

Spark Therapeutics, Inc. is a biotechnology company focused on developing gene therapy products aimed at treating debilitating genetic diseases. Headquartered in Philadelphia, Pennsylvania, the company was founded in 2013 and has established itself as a leader in the field of gene therapy, with a robust pipeline that includes LUXTURNA for retinal dystrophy, as well as several candidates for hemophilia and choroideremia. Spark is also exploring therapies for neurodegenerative diseases and inherited retinal diseases. The company holds a collaboration agreement with Pfizer for the development of hemophilia B treatments and has partnered with Novartis for the commercialization of its lead product outside the United States. Spark Therapeutics leverages a proprietary manufacturing platform and a team with extensive expertise in research, clinical development, and regulatory affairs, building on a legacy of innovation in gene therapy from its origins at The Children’s Hospital of Philadelphia. As of December 2019, Spark operates as a subsidiary of Roche Holding AG.

Lysogene S.A. is a biotechnology company based in Neuilly-sur-Seine, France, focused on the research and clinical development of gene therapy for neurodegenerative disorders. Founded in 2009, the company is advancing several clinical programs, including LYS-SAF302, currently in Phase II/III trials for the treatment of mucopolysaccharidosis type IIIA, and LYS-GM101, which is in Phase I/II trials for GM1 gangliosidosis. Lysogene is also engaged in a collaborative research effort with the Weizmann Institute of Science to develop a novel AAV gene therapy approach targeting neuronopathic Gaucher disease, Parkinson's disease, and other conditions. The company aims to enhance the health outcomes of patients suffering from life-threatening, incurable disorders by utilizing AAV vectors that effectively deliver genetic material to the central nervous system.

Coherus Biosciences is a biopharmaceutical company that specializes in the development, manufacture, and commercialization of biologic therapeutics, with a strong emphasis on oncology and inflammatory diseases. The company is actively engaged in research and development, focusing on process science, analytical characterization, and protein production. Coherus's portfolio includes FDA-approved products such as UDENYCA, a biosimilar of Neulasta, and it plans to launch YUSIMRY, a biosimilar of Humira, in the United States. Additionally, the company is advancing a pipeline of biosimilar candidates across areas such as immunology and ophthalmology, as well as anti-tumor necrosis factor treatments. With a commitment to building an immuno-oncology franchise, Coherus aims to leverage its diversified portfolio to generate revenue and enhance patient access to biologic therapies.

Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.

NuCana plc is a UK-based, clinical-stage biopharmaceutical company dedicated to developing innovative cancer treatments. It specializes in transforming widely-used chemotherapy agents into more effective and safer medicines using its proprietary ProTide technology. NuCana's pipeline includes Acelarin, in Phase Ib and III trials for biliary tract and pancreatic cancers, and NUC-3373 and NUC-7738, in Phase I trials for various advanced solid tumors. The company collaborates with academic institutions for research and licensing agreements. NuCana's management team has a proven track record in building successful biopharmaceutical companies and developing oncology medicines.

NuCana plc is a UK-based, clinical-stage biopharmaceutical company dedicated to developing innovative cancer treatments. It specializes in transforming widely-used chemotherapy agents into more effective and safer medicines using its proprietary ProTide technology. NuCana's pipeline includes Acelarin, in Phase Ib and III trials for biliary tract and pancreatic cancers, and NUC-3373 and NUC-7738, in Phase I trials for various advanced solid tumors. The company collaborates with academic institutions for research and licensing agreements. NuCana's management team has a proven track record in building successful biopharmaceutical companies and developing oncology medicines.

ZS Pharma, Inc. is a biopharmaceutical company based in Coppell, Texas, focused on developing non-absorbed drugs for renal, cardiovascular, liver, and metabolic diseases. Established in 2008, the company specializes in highly selective ion-trap therapies, particularly its lead candidate, ZS-9. This innovative treatment employs proprietary zirconium silicate technology to create ion traps that can effectively reduce excess potassium levels in patients suffering from hyperkalemia, a serious condition often associated with chronic kidney disease, hypertension, diabetes, and heart failure. ZS-9 is currently undergoing late-stage clinical trials to evaluate its efficacy and safety. As of December 2015, ZS Pharma operates as a subsidiary of Zeneca, Inc.

Versartis is a biotechnology company based in Redwood City, California, focused on developing therapeutic proteins for endocrine disorders. The company specializes in recombinant human growth hormones and employs a novel half-life extension technology known as XTEN to enhance treatment efficacy. Versartis is advancing its lead program, VRS-317, which has recently completed enrollment in a multi-center Phase 1 clinical study targeting adult patients with growth hormone deficiency. The company aims to improve patient compliance and treatment outcomes by reducing the frequency of daily injections associated with traditional therapies. Founded in December 2008 through a joint venture with Amunix Inc. and Index Ventures, Versartis is supported by a team of drug development experts and leverages external contract services to efficiently execute its product development strategies.

Innocoll is a privately held biopharmaceutical company specializing in collagen-based pharmaceutical products and medical devices. The company develops biodegradable surgical implants and topically applied healthcare solutions aimed at enhancing patient care in various medical contexts. Its product lineup includes CollaGUARD, a bioresorbable collagen film to prevent post-operative adhesions, and Collatamp Gentamicin, which helps treat or prevent surgical infections. Septocoll serves as a dual-action sponge for infection management, while RegenePro is designed for dental applications. Innocoll is also advancing product candidates like XaraColl, currently in phase III trials for post-operative pain relief, and Cogenzia, aimed at diabetic foot infections. With its headquarters in Athlone, Ireland, Innocoll utilizes a proprietary technology platform to create products that are fully bioresorbable, offering non-opioid alternatives for pain management in surgical settings.

Ziarco, Inc. is a biotechnology company founded in 2012 and based in Palo Alto, California. The company specializes in the development of therapeutic agents aimed at treating inflammatory and allergic diseases. Its primary focus is on inflammatory skin disorders, and its product pipeline includes a histamine H4 receptor antagonist program targeting various conditions such as asthma, allergic rhinitis, pruritus, skin diseases, and pain. Ziarco's innovations aim to provide effective treatment options for patients suffering from atopic dermatitis and psoriasis, enhancing their quality of life through improved therapeutic solutions.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Versartis is a biotechnology company based in Redwood City, California, focused on developing therapeutic proteins for endocrine disorders. The company specializes in recombinant human growth hormones and employs a novel half-life extension technology known as XTEN to enhance treatment efficacy. Versartis is advancing its lead program, VRS-317, which has recently completed enrollment in a multi-center Phase 1 clinical study targeting adult patients with growth hormone deficiency. The company aims to improve patient compliance and treatment outcomes by reducing the frequency of daily injections associated with traditional therapies. Founded in December 2008 through a joint venture with Amunix Inc. and Index Ventures, Versartis is supported by a team of drug development experts and leverages external contract services to efficiently execute its product development strategies.

Marinus Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapeutics for rare seizure disorders and neuropsychiatric conditions. The company's primary product candidate, ganaxolone, is an allosteric modulator of GABAA receptors, designed for both intravenous and oral administration for various patient populations. It aims to address multiple indications, including status epilepticus, cyclin dependent kinase-like 5 deficiency disorder, PCDH19-related epilepsy, tuberous sclerosis complex, postpartum depression, and treatment-resistant depression. Marinus Pharmaceuticals collaborates with NovaMedica and has license agreements with Purdue Neuroscience Company and CyDex Pharmaceuticals to advance its research and therapeutic offerings. Founded in 2003, the company is headquartered in Radnor, Pennsylvania.

Labrys Biologics Inc. is a biotechnology company dedicated to developing treatments for chronic migraine. The company has secured worldwide rights to RN-307, an antibody initially developed by Pfizer Inc., specifically designed for the treatment of chronic migraine. Additionally, Labrys Biologics is advancing its lead candidate, LBR-101, which is an anti-CGRP monoclonal antibody aimed at preventing chronic and high-frequency migraines. Through these innovative therapies, Labrys Biologics aims to address the significant unmet medical needs of individuals suffering from chronic migraine conditions.

Prothena Corporation plc, established in 2012 and headquartered in Dublin, Ireland, is a clinical-stage neuroscience company dedicated to developing novel therapies for life-threatening diseases. Its primary focus is on protein dysregulation, with a pipeline of investigational therapeutics targeting various neurodegenerative and peripheral amyloid diseases. Prothena's clinical pipeline includes PRX002 for Parkinson's disease and other synucleinopathies (Phase II), PRX004 for ATTR amyloidosis (Phase I), and discovery-stage programs for Alzheimer's disease, tauopathies, and other neurodegenerative conditions. The company collaborates with industry leaders like Roche and Bristol-Myers Squibb to advance its pipeline.

Labrys Biologics Inc. is a biotechnology company dedicated to developing treatments for chronic migraine. The company has secured worldwide rights to RN-307, an antibody initially developed by Pfizer Inc., specifically designed for the treatment of chronic migraine. Additionally, Labrys Biologics is advancing its lead candidate, LBR-101, which is an anti-CGRP monoclonal antibody aimed at preventing chronic and high-frequency migraines. Through these innovative therapies, Labrys Biologics aims to address the significant unmet medical needs of individuals suffering from chronic migraine conditions.

Catalyst Biosciences, Inc. is a clinical-stage biopharmaceutical company headquartered in South San Francisco, California. The company specializes in developing innovative treatments for hemophilia and other rare bleeding disorders, utilizing its engineered subcutaneous coagulation factors to promote blood clotting. This approach aims to address the inherent limitations of traditional intravenous treatments and improve patient outcomes. Catalyst's product portfolio includes Marzeptacog alfa (activated), an advanced coagulation Factor VIIa that has completed Phase II development for hemophilia A or B with inhibitors, and Dalcinonacog alfa, a next-generation Factor IX therapy that has finished Phase IIb clinical trials for hemophilia B. Additionally, the company is working on CB 2679d-GT, an early-stage gene therapy for hemophilia B, as well as other treatments targeting dry age-related macular degeneration and systemic complement inhibitors. Catalyst Biosciences has also formed strategic collaborations with Mosaic Biosciences and Biogen to enhance its research and development efforts in these therapeutic areas. The company was founded in 2002 and has a commitment to addressing unmet medical needs in coagulation and complement disorders.

Tesaro, Inc. is an oncology-focused biopharmaceutical company established in 2010 and headquartered in Waltham, Massachusetts. The company is dedicated to developing and commercializing cancer therapeutics and supportive care products. Its primary offerings include ZEJULA, a potent oral poly polymerase inhibitor for the maintenance treatment of recurrent ovarian, fallopian tube, or primary peritoneal cancer, and VARUBI, a neurokinin-1 receptor antagonist designed to prevent chemotherapy-induced nausea and vomiting. Additionally, Tesaro is engaged in the development of several immunotherapy antibody candidates, including TSR-042, TSR-022, and TSR-033, all currently in phase I clinical trials. The company collaborates with various organizations, such as AnaptysBio, Janssen Biotech, and Merck Sharp & Dohme, to enhance its research and development efforts. Tesaro aims to leverage its management team's expertise to identify and commercialize innovative treatments that improve the lives of cancer patients. As of January 2019, Tesaro operates as a subsidiary of GlaxoSmithKline.

Vicept Therapeutics, Inc., located in Malvern, Pennsylvania, is a privately held specialty biopharmaceutical company founded in August 2009. The company focuses on developing innovative topically applied therapies aimed at treating erythema, or skin redness, associated with rosacea and other dermatological conditions characterized by redness and flushing. In addition to its research and development efforts, Vicept also manufactures and distributes dermatological therapeutic products, positioning itself as a key player in addressing unmet medical needs in skin health.

Alvine Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing therapeutics for autoimmune and gastrointestinal diseases, with a particular emphasis on celiac disease. The company's lead product candidate, ALV003, is an orally administered mixture of two gluten-specific proteases designed to degrade gluten, the primary immunologic trigger for celiac disease. In addition to ALV003, Alvine is working on proteases that target other aspects of celiac disease, including Transglutaminase 2 (TG2) and the HLA-DQ2 antigen. Founded in 2005 and based in San Carlos, California, Alvine Pharmaceuticals operates as a subsidiary of ImmunogenX, LLC as of February 2016.

Intellikine is engaged in the discovery and development of small molecule therapies that specifically target the PI3K/Akt/mTOR signaling pathway. The company focuses on creating innovative drug candidates aimed at treating various conditions, including cancer, inflammation, and autoimmune disorders. Its product lineup features INK128, an orally available TORC1/2 inhibitor designed for solid tumor malignancies, and INK1197, a dual PI3K selective inhibitor intended for patients suffering from immune-mediated inflammatory diseases like rheumatoid arthritis and asthma. Intellikine is dedicated to assembling a talented team and an effective discovery platform to accelerate the development of next-generation medical breakthroughs in kinase inhibitor therapies.

Hyperion Therapeutics, Inc. is a biopharmaceutical company based in Brisbane, California, focused on developing and commercializing therapies for orphan diseases. The company specializes in treatments for urea cycle disorders (UCD), offering products such as RAVICTI, a nitrogen-binding agent used for the chronic management of UCD in both adults and pediatric patients. In addition to RAVICTI, Hyperion provides BUPHENYL and AMMONAPS for the treatment of specific UCD subtypes. The company is also exploring the application of glycerol phenylbutyrate, the active ingredient in RAVICTI, for the treatment of hepatic encephalopathy. Founded in 2006, Hyperion Therapeutics aims to address the needs of underserved patient populations through its innovative therapeutic developments.

Marinus Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapeutics for rare seizure disorders and neuropsychiatric conditions. The company's primary product candidate, ganaxolone, is an allosteric modulator of GABAA receptors, designed for both intravenous and oral administration for various patient populations. It aims to address multiple indications, including status epilepticus, cyclin dependent kinase-like 5 deficiency disorder, PCDH19-related epilepsy, tuberous sclerosis complex, postpartum depression, and treatment-resistant depression. Marinus Pharmaceuticals collaborates with NovaMedica and has license agreements with Purdue Neuroscience Company and CyDex Pharmaceuticals to advance its research and therapeutic offerings. Founded in 2003, the company is headquartered in Radnor, Pennsylvania.

Vicept Therapeutics, Inc., located in Malvern, Pennsylvania, is a privately held specialty biopharmaceutical company founded in August 2009. The company focuses on developing innovative topically applied therapies aimed at treating erythema, or skin redness, associated with rosacea and other dermatological conditions characterized by redness and flushing. In addition to its research and development efforts, Vicept also manufactures and distributes dermatological therapeutic products, positioning itself as a key player in addressing unmet medical needs in skin health.

Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Malvern, Pennsylvania, dedicated to developing innovative therapeutics aimed at enhancing the effectiveness of radiotherapy for cancer patients. The company's lead product candidate, avasopasem manganese (GC4419), is a small molecule dismutase mimetic currently undergoing Phase III clinical trials for the treatment of radiation-induced severe oral mucositis in patients with head and neck cancer. Additionally, GC4419 is being evaluated in Phase IIa trials for the treatment of radiotherapy-induced esophagitis in lung cancer patients. Galera is also advancing another candidate, GC4711, which is in Phase I/II trials for pancreatic and lung cancers. The company's research is focused on targeting oxygen metabolic pathways to improve patient outcomes in radiation therapy, and GC4419 has received Fast Track and Breakthrough Therapy designations from the U.S. Food and Drug Administration.

Catalyst Biosciences, Inc. is a clinical-stage biopharmaceutical company headquartered in South San Francisco, California. The company specializes in developing innovative treatments for hemophilia and other rare bleeding disorders, utilizing its engineered subcutaneous coagulation factors to promote blood clotting. This approach aims to address the inherent limitations of traditional intravenous treatments and improve patient outcomes. Catalyst's product portfolio includes Marzeptacog alfa (activated), an advanced coagulation Factor VIIa that has completed Phase II development for hemophilia A or B with inhibitors, and Dalcinonacog alfa, a next-generation Factor IX therapy that has finished Phase IIb clinical trials for hemophilia B. Additionally, the company is working on CB 2679d-GT, an early-stage gene therapy for hemophilia B, as well as other treatments targeting dry age-related macular degeneration and systemic complement inhibitors. Catalyst Biosciences has also formed strategic collaborations with Mosaic Biosciences and Biogen to enhance its research and development efforts in these therapeutic areas. The company was founded in 2002 and has a commitment to addressing unmet medical needs in coagulation and complement disorders.

Anthera Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing therapeutics for serious diseases related to inflammation, such as cardiovascular and autoimmune disorders. The company is advancing several product candidates, including Sollpura, a non-porcine pancreatic enzyme replacement therapy currently in Phase III trials for exocrine pancreatic insufficiency, and Blisibimod, which is in Phase II trials targeting B-cell mediated autoimmune diseases, including IgA nephropathy. Additionally, Anthera has other candidates like varespladib methyl and varespladib sodium, which are in various stages of clinical trials for acute coronary syndrome and sickle cell disease, respectively. Established in 2004, Anthera Pharmaceuticals is headquartered in Hayward, California, and has formed licensing agreements with prominent companies such as Amgen and Eli Lilly to facilitate the development of its therapies.

InteKrin Therapeutics is a clinical-stage, privately held biopharmaceutical company dedicated to developing innovative therapeutics for neuroendocrine, metabolic, and immune disorders. The company addresses the growing need for effective treatments for conditions such as diabetes, metabolic syndrome, and cancer, which significantly impact healthcare costs. InteKrin is actively in-licensing both early- and late-stage compounds aimed at exploring the complex hormonal interactions related to metabolism, insulin resistance, and obesity. One of its key developments includes INT131, a non-thiazolidinedione peroxisome proliferator-activated receptor gamma modulator, which targets Relapsing Remitting Multiple Sclerosis. This treatment seeks to meet the dual objectives of neuroprotection and halting the progression of this challenging disease.

Amarin Corporation plc, a pharmaceutical company based in Dublin, Ireland, develops and markets therapeutics for cardiovascular disease treatment. Its primary product, Vascepa, is a prescription omega-3 fatty acid capsule used to lower triglyceride levels in adults with severe hypertriglyceridemia. Amarin also has a pipeline for REDUCE-IT, targeting patients with high triglyceride levels on statin therapy. The company sells its products through a direct sales force to wholesalers and specialty pharmacies, and collaborates with Mochida Pharmaceutical Co., Ltd. for further drug development. Amarin's expertise lies in lipid science and the therapeutic benefits of essential fatty acids in cardiovascular disease management.

Hyperion Therapeutics, Inc. is a biopharmaceutical company based in Brisbane, California, focused on developing and commercializing therapies for orphan diseases. The company specializes in treatments for urea cycle disorders (UCD), offering products such as RAVICTI, a nitrogen-binding agent used for the chronic management of UCD in both adults and pediatric patients. In addition to RAVICTI, Hyperion provides BUPHENYL and AMMONAPS for the treatment of specific UCD subtypes. The company is also exploring the application of glycerol phenylbutyrate, the active ingredient in RAVICTI, for the treatment of hepatic encephalopathy. Founded in 2006, Hyperion Therapeutics aims to address the needs of underserved patient populations through its innovative therapeutic developments.

Prestwick Pharmaceuticals develops and markets therapeutic pharmaceutical drugs for chronic central nervous systems diseases. The company offers products for restless legs syndrome, schizophrenia, autism, Alzheimer’s, and sleep apnea ailments. Prestwick Pharmaceuticals was founded in 2002 and is based in Washington, District of Columbia.

Intellikine is engaged in the discovery and development of small molecule therapies that specifically target the PI3K/Akt/mTOR signaling pathway. The company focuses on creating innovative drug candidates aimed at treating various conditions, including cancer, inflammation, and autoimmune disorders. Its product lineup features INK128, an orally available TORC1/2 inhibitor designed for solid tumor malignancies, and INK1197, a dual PI3K selective inhibitor intended for patients suffering from immune-mediated inflammatory diseases like rheumatoid arthritis and asthma. Intellikine is dedicated to assembling a talented team and an effective discovery platform to accelerate the development of next-generation medical breakthroughs in kinase inhibitor therapies.

DiObex, Inc. operates as a biotechnology company in the United States. The company develops novel products for the treatment of metabolic disorders, including type-one and type-two diabetes, dyslipidemia, obesity, and diabetic nephropathy. Its products include DIO-901, a very low dose glucagon product that is developed for the reduction of nocturnal hypoglycemia in patients with type-one diabetes; and DIO-902, a cortisol synthesis inhibitor that is developed as a therapy for glucose and cholesterol control in patients with type-two diabetes. DiObex, Inc. was founded in 2003 and is based in San Francisco, California.

Ascenta Therapeutics is a privately-held, clinical stage biopharmaceutical company dedicated to the discovery and development of new medicines to treat cancer. Ascenta's current focus is a portfolio of novel, orally-active, small molecule drugs that restore the natural potential for cancer cells to undergo cell death (apoptosis).

As of October 2010, Phenomix closed down as it was unable to find a partner to pay for is clinical trial bills after Forest Laboratories walked away. Phenomix is a drug discovery and development company building a portfolio of novel therapeutics for the treatment of major human diseases. Phenomix' lead clinical candidate, PHX1149, is a DPP4 inhibitor in phase 2 testing as a once daily oral treatment for type 2 diabetes. Phenomix' second program is directed at the treatment of hepatitis C through inhibition of the NS3/4A viral protease. Phenomix is based in San Diego, California.

Trius Therapeutics is a biopharmaceutical company based in San Diego, California, dedicated to the discovery, development, and commercialization of innovative antibiotics aimed at treating life-threatening infections. The company has successfully completed two Phase 3 clinical trials for its lead product, Tedizolid Phosphate, a second-generation oxazolidinone designed for the treatment of serious gram-positive bacterial infections, including those caused by methicillin-resistant Staphylococcus aureus (MRSA). As bacterial resistance to existing antibiotics continues to rise, the need for new therapeutics to combat multi-drug resistant infections becomes increasingly urgent. Trius Therapeutics is led by a skilled management team with extensive experience in drug development and regulation, contributing to the company's efforts to address critical healthcare challenges posed by antibiotic resistance.