Sofinnova Investments

Edgewise Therapeutics, founded in 2017 and based in Boulder, Colorado, is a clinical-stage biopharmaceutical company dedicated to developing precision medicine therapies for severe, rare muscle disorders. The company's core expertise lies in muscle biology and small molecule engineering, which it employs through its proprietary drug discovery platform. This platform uses custom-built systems to measure integrated muscle function, enabling the identification of small molecule therapies targeting key proteins in muscle tissue. Edgewise's pipeline focuses on addressing genetically defined muscle disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophies.

Atsena Therapeutics is a clinical-stage gene therapy company dedicated to developing innovative treatments for inherited retinal diseases. The company focuses on therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Utilizing an advanced adeno-associated virus (AAV) technology platform, including a novel spreading capsid, Atsena aims to target specific retinal conditions to prevent blindness resulting from genetic mutations. Their research is centered on providing effective ocular gene therapies that address significant challenges associated with inherited retinal diseases, with the goal of reversing or halting vision loss.

Avenzo Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative therapies for oncology, specifically targeting underserved and rare cancers. The company operates a specialized platform that focuses on creating novel treatments aimed at genomically defined cancer types. By prioritizing targeted therapies, Avenzo Therapeutics seeks to advance cancer treatment options for patients who may not have access to effective solutions, thus addressing significant gaps in the current therapeutic landscape.

Seaport Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative neuropsychiatric medicines for conditions with significant unmet needs. The company's approach emphasizes the creation of therapies that offer oral bioavailability, which allows for effective drug delivery while minimizing first-pass metabolism and reducing the risk of hepatotoxicity and other side effects. By addressing these critical challenges, Seaport Therapeutics aims to improve the quality of life for patients living with neuropsychiatric disorders.

Seaport Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative neuropsychiatric medicines for conditions with significant unmet needs. The company's approach emphasizes the creation of therapies that offer oral bioavailability, which allows for effective drug delivery while minimizing first-pass metabolism and reducing the risk of hepatotoxicity and other side effects. By addressing these critical challenges, Seaport Therapeutics aims to improve the quality of life for patients living with neuropsychiatric disorders.

Avenzo Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative therapies for oncology, specifically targeting underserved and rare cancers. The company operates a specialized platform that focuses on creating novel treatments aimed at genomically defined cancer types. By prioritizing targeted therapies, Avenzo Therapeutics seeks to advance cancer treatment options for patients who may not have access to effective solutions, thus addressing significant gaps in the current therapeutic landscape.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to treat aging and age-related diseases. The company utilizes a proprietary systems biology and artificial intelligence platform to identify significant drug targets that influence aging, leveraging extensive datasets to uncover the molecular drivers of age-related pathologies. Among its key offerings is BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor that activates HIF-1 target genes involved in various biological processes, including tissue regeneration and vascular remodeling. Another notable product is BGE-175, an orally administered inhibitor targeting the prostaglandin D2 DP1 signaling pathway, which is linked to an increased risk of mortality and infections, including those associated with immune aging. Founded in 2015 and headquartered in Richmond, California, BioAge Labs is dedicated to harnessing the biology of human aging to create innovative therapies for metabolic and age-related diseases.

OnCusp Therapeutics is a biopharmaceutical company dedicated to transforming advanced research into innovative cancer treatments. Founded by Bing Yuan, Eric Slosberg, and Andy Fu, the company focuses on accelerating the development of oncology medications to provide help and hope to cancer patients globally. OnCusp Therapeutics specializes in translating preclinical drug candidates into clinical proof-of-concept stages, aiming to swiftly advance these assets into early global clinical development.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing precision medicines for neurological disorders. The company focuses on creating transformational small-molecule therapies aimed at patients with central nervous system conditions. Leveraging foundational research in neuronal receptor biology, Rapport Therapeutics maps and targets specific neuronal receptor complexes, which are complex assemblies of proteins that include principal receptor subunits and receptor-associated proteins. These receptor-associated proteins are essential for regulating receptor expression and functionality, allowing the company to innovate in the treatment of neurological diseases.

Alkeus Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston that specializes in developing treatments for serious ophthalmic conditions. The company's primary focus is on addressing untreatable diseases of the eye, with its lead drug candidate being an orally delivered compound aimed at treating Stargardt disease and age-related macular degeneration. Through its innovative approach, Alkeus aims to empower medical professionals to manage symptoms of irreversible vision loss at an earlier stage in affected patients.

Antiva Biosciences, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral therapies for human papillomavirus (HPV) infections. Established in 2012, the company aims to prevent cancer by treating precancerous lesions associated with HPV. Antiva's innovative approach includes localized therapeutics and small molecule antivirals that target HPV and other viral infections. The company is currently conducting clinical trials for high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias. Through its medicinal chemistry platform, Antiva strives to enhance health outcomes for patients affected by HPV-related diseases and other viral infections.

ArriVent Biopharma is a clinical-stage biopharmaceutical company focused on developing innovative treatments for currently untreatable cancers. The company aims to address unmet medical needs by identifying, developing, and commercializing differentiated medicines. ArriVent is advancing its lead development candidate, furmonertinib, alongside a pipeline of novel therapeutics, including next-generation antibody drug conjugates. The company's efforts are primarily centered on solid tumors, leveraging the team's extensive experience in drug development to enhance the potential of its therapeutic candidates from initial development through to approval and commercialization.

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. The company specializes in developing tumor-targeted small molecule medicines that leverage radioisotopes to improve cancer treatment outcomes. RayzeBio focuses on creating innovative radiopharmaceuticals, particularly utilizing alpha-emitting radioisotopes like Actinium-225, to target solid tumors effectively. With a robust pipeline of drug candidates, RayzeBio is committed to addressing significant market opportunities in oncology through late-stage clinical programs, development initiatives, and discovery efforts. The company's mission is to provide effective therapeutic solutions aimed at defeating cancer.

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

Antiva Biosciences, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral therapies for human papillomavirus (HPV) infections. Established in 2012, the company aims to prevent cancer by treating precancerous lesions associated with HPV. Antiva's innovative approach includes localized therapeutics and small molecule antivirals that target HPV and other viral infections. The company is currently conducting clinical trials for high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias. Through its medicinal chemistry platform, Antiva strives to enhance health outcomes for patients affected by HPV-related diseases and other viral infections.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of innovative treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its clinical candidates toward marketing approval, CinCor seeks to provide effective solutions for patients suffering from these 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, Inc. is a biopharmaceutical company specializing in the development of gene therapies for cardiovascular diseases, particularly advanced coronary artery disease. Founded in 2013 and based in Newtown Square, Pennsylvania, the company focuses on innovative treatments aimed at addressing significant unmet medical needs. Its lead product candidate, XC001, is currently in clinical development for patients suffering from refractory angina, targeting individuals who have exhausted other treatment options. XyloCor's approach centers on stimulating the formation of new coronary blood vessels to enhance blood supply to areas of the heart that are inadequately perfused, thereby improving patient outcomes in those affected by heart failure and ischemic conditions. The company collaborates with Weill Cornell Medical College to advance its gene therapy technologies.

Vera Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2016 and formerly known as Trucode Gene Repair, Inc. The company focuses on developing innovative biologic therapeutics with the potential to transform treatment for patients suffering from serious immunological diseases and genetic disorders. Its lead product candidate, atacicept, is a fusion protein administered as a subcutaneous injection once weekly, designed to inhibit factors that stimulate the production of autoantibodies associated with autoimmune diseases. Additionally, Vera Therapeutics is utilizing gene-editing technology aimed at addressing conditions such as sickle cell disease and cystic fibrosis, striving to enhance gene detection and regulation for improved patient outcomes.

Atsena Therapeutics is a clinical-stage gene therapy company dedicated to developing innovative treatments for inherited retinal diseases. The company focuses on therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Utilizing an advanced adeno-associated virus (AAV) technology platform, including a novel spreading capsid, Atsena aims to target specific retinal conditions to prevent blindness resulting from genetic mutations. Their research is centered on providing effective ocular gene therapies that address significant challenges associated with inherited retinal diseases, with the goal of reversing or halting vision loss.

Aerovate Therapeutics, founded in 2018 and headquartered in Boston, Massachusetts, is a biotechnology company dedicated to improving the lives of patients with rare cardiopulmonary diseases. Its primary focus is AV-101, an inhaled dry powder formulation of imatinib, designed to deliver medication directly to affected lung tissues while minimizing systemic side effects. This innovative approach aims to treat pulmonary arterial hypertension (PAH), a severe and progressive disease.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various hematological cancers and solid tumors. Established in 2017, the company specializes in therapies that target receptor tyrosine kinase-like orphan receptor 1 (ROR1), a protein present on many cancerous cells but absent in normal tissues, making it a promising target for cancer treatment. VelosBio's lead product, VLS-101, is an ROR1-directed ADC designed for patients with both hematologic and solid tumor malignancies. The company's approach aims to provide novel targeted therapies that can be utilized as monotherapy or in combination with other treatments across a wide array of cancer types.

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

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and halting neurodegeneration. The company's lead product candidate, ATH-1017, is a small molecule designed to penetrate the blood-brain barrier and acts as a hepatocyte growth factor/MET activator, currently undergoing various clinical trials for treating Alzheimer’s and Parkinson’s diseases. In addition to ATH-1017, Athira is developing several other candidates, including ATH-1019 for depression and ATH-1018 for peripheral neuropathy, which are in the preclinical stage. Founded in 2011 and initially known as M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its mission of advancing innovative therapies to improve the lives of individuals affected by brain disorders.

Amplyx Pharmaceuticals, Inc. is a small molecule drug development company focused on creating innovative therapies for debilitating and life-threatening diseases, particularly those affecting individuals with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in the development of oral and injectable anti-infective drugs, including antifungal agents designed to treat invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by incorporating a second small molecule. This strategy aims to provide effective treatment options for patients undergoing chemotherapy and other immunosuppressive therapies, ensuring they receive appropriate care both during hospitalization and after discharge.

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

Vera Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2016 and formerly known as Trucode Gene Repair, Inc. The company focuses on developing innovative biologic therapeutics with the potential to transform treatment for patients suffering from serious immunological diseases and genetic disorders. Its lead product candidate, atacicept, is a fusion protein administered as a subcutaneous injection once weekly, designed to inhibit factors that stimulate the production of autoantibodies associated with autoimmune diseases. Additionally, Vera Therapeutics is utilizing gene-editing technology aimed at addressing conditions such as sickle cell disease and cystic fibrosis, striving to enhance gene detection and regulation for improved patient outcomes.

Intrepida Bio, Inc. is a biotechnology company focused on developing innovative cancer therapies that enhance the body’s innate immune response. Founded in 2016 and headquartered in San Diego, California, the company specializes in creating medicines that modulate the innate immune system to combat cancer and other diseases. Intrepida Bio is particularly engaged in the development of monoclonal antibodies aimed at novel targets such as BAG3 and its receptor IFITM-2, which are involved in establishing a tumor-friendly environment. Through its research efforts, Intrepida Bio aims to provide patients with effective treatment options to fight cancer.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of innovative treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its clinical candidates toward marketing approval, CinCor seeks to provide effective solutions for patients suffering from these conditions.

Karuna Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Boston, Massachusetts, that specializes in developing innovative therapies for neuropsychiatric disorders and pain management. The company is advancing its lead product candidate, KarXT, an oral modulator that has completed a Phase II clinical trial for acute psychosis in schizophrenia and is undergoing Phase Ib trials for various central nervous system disorders, including the cognitive and negative symptoms associated with schizophrenia, Alzheimer's disease, and dementia-related psychosis. Karuna Therapeutics is also actively developing a pipeline of muscarinic-targeted drug candidates to address unmet medical needs in these challenging areas. Founded in 2009, the company has established partnerships and licensing agreements to support its drug discovery efforts and to enhance its research capabilities.

Promedior, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing innovative therapeutics for rare fibrotic diseases and retinal fibrovascular conditions. The company targets conditions such as myelofibrosis, idiopathic pulmonary fibrosis, age-related macular degeneration, and diabetic retinopathy. Promedior's portfolio includes PRM-151, a recombinant form of human pentaxin-2 protein designed for intravenous injection, and PRM-167, a variant formulated for intravitreal delivery. Promedior employs a novel drug discovery platform that aims to regulate monocyte-derived cell populations, which are critical in inflammatory, fibrotic, and autoimmune diseases. By specifically addressing these cells at the injury site, Promedior seeks to treat the underlying causes of immune system dysregulation, promote tissue healing, and minimize systemic side effects associated with traditional therapies. The company's commitment to addressing severe and challenging conditions positions it as a significant player in the biopharmaceutical landscape.

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

XyloCor Therapeutics, Inc. is a biopharmaceutical company specializing in the development of gene therapies for cardiovascular diseases, particularly advanced coronary artery disease. Founded in 2013 and based in Newtown Square, Pennsylvania, the company focuses on innovative treatments aimed at addressing significant unmet medical needs. Its lead product candidate, XC001, is currently in clinical development for patients suffering from refractory angina, targeting individuals who have exhausted other treatment options. XyloCor's approach centers on stimulating the formation of new coronary blood vessels to enhance blood supply to areas of the heart that are inadequately perfused, thereby improving patient outcomes in those affected by heart failure and ischemic conditions. The company collaborates with Weill Cornell Medical College to advance its gene therapy technologies.

NextCure, Inc. is a clinical-stage biopharmaceutical company based in Beltsville, Maryland, that focuses on discovering and developing novel immunomedicines aimed at treating cancer and other immune-related diseases by restoring normal immune function. Its lead product candidate, NC318, is currently undergoing Phase 2 clinical trials for advanced or metastatic solid tumors. Additionally, NextCure is developing NC410, designed to inhibit immune suppression through the modulation of a specific immune receptor. The company is also advancing other product candidates, including LNCB74, an antibody-drug conjugate targeting B7-H4, and is exploring additional immunomodulatory molecules in preclinical stages. NextCure has established collaborations with Yale University for licensing and with Eli Lilly and Company for research and development. Founded in 2015, the company leverages proprietary platforms to identify biologically relevant targets in its pursuit of 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 hematological cancers and solid tumors. Established in 2017, the company specializes in therapies that target receptor tyrosine kinase-like orphan receptor 1 (ROR1), a protein present on many cancerous cells but absent in normal tissues, making it a promising target for cancer treatment. VelosBio's lead product, VLS-101, is an ROR1-directed ADC designed for patients with both hematologic and solid tumor malignancies. The company's approach aims to provide novel targeted therapies that can be utilized as monotherapy or in combination with other treatments across a wide array 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, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral therapies for human papillomavirus (HPV) infections. Established in 2012, the company aims to prevent cancer by treating precancerous lesions associated with HPV. Antiva's innovative approach includes localized therapeutics and small molecule antivirals that target HPV and other viral infections. The company is currently conducting clinical trials for high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias. Through its medicinal chemistry platform, Antiva strives to enhance health outcomes for patients affected by HPV-related diseases and other viral infections.

ENYO Pharma SA is a clinical-stage biopharmaceutical company based in Lyon, France, founded in 2014 by scientists from the Infectiology Research Center. The company focuses on drug discovery and development for the treatment of acute and chronic viral infections, utilizing a unique platform to identify intracellular therapeutic targets and molecules. ENYO Pharma is developing a pipeline of drug candidates addressing various indications, including hepatitis B virus, nonalcoholic steatohepatitis, and oncology. Its lead compound, EYP001, is designed to modulate specific nuclear receptors, aiming to reduce viral reservoirs and inhibit harmful viral protein expression. The company is also advancing EYP002 through preclinical studies. ENYO Pharma collaborates closely with established research institutions and aims to conduct its molecules into Phase II clinical trials, striving to become a global leader in antiviral therapeutics.

Rainier Therapeutics, Inc. is a biotechnology company focused on developing targeted therapies for bladder cancer. The company specializes in Vofatamab, a human monoclonal antibody designed to specifically block the activity of fibroblast growth factor receptor 3 (FGFR3). This innovative treatment aims to improve outcomes for patients with both early-stage and metastatic bladder cancer. Founded in 2010 and based in San Leandro, California, Rainier Therapeutics was previously known as BioClin Therapeutics, Inc. before rebranding in November 2018. Through its advanced therapeutic technology, Rainier Therapeutics seeks to enhance treatment options for those affected by this challenging disease.

Merus B.V. is a clinical-stage biomedical company focused on the discovery and development of innovative antibody-based biopharmaceuticals. The company specializes in bispecific antibody therapeutics, utilizing its proprietary Oligoclonics technology to produce a unique class of human antibodies. This technology enables the generation of a mixture of therapeutic antibodies targeting a common antigen from a single cell clone. Merus is also advancing its full-length human multispecific antibody therapeutics, known as Biclonics. The company has a diverse pipeline of product candidates, including MCLA-128, MCLA-117, and MCLA-158, among others, all designed to address various challenges in immuno-oncology.

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 focused on developing and commercializing innovative antibody-based therapies for cancer treatment in the United States. The company's lead product, DANYELZA, is an FDA-approved monoclonal antibody targeting ganglioside GD2, which is present in various neuroectoderm-derived tumors and sarcomas. Y-mAbs is advancing several other candidates, including naxitamab, currently in Phase 2 trials for pediatric patients with relapsed osteosarcoma and high-risk neuroblastoma, and omburtamab, also in Phase 2 trials for desmoplastic small round cell tumors. Additionally, the company is developing a GD2-GD3 vaccine and exploring the huB7-H3 candidate for adult solid tumors. Y-mAbs collaborates with leading institutions, including Memorial Sloan Kettering Cancer Center and the Massachusetts Institute of Technology, to enhance its antibody constructs using advanced therapeutic platforms. Founded in 2015 and headquartered in New York, Y-mAbs aims to provide novel treatments that may reduce the long-term toxicities associated with traditional chemotherapies while potentially offering curative options for patients with widespread disease.

ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions affecting women. Its key products in development include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and heavy menstrual bleeding associated with uterine fibroids. ObsEva is also working on OBE022, a once-daily oral prostaglandin F2α receptor antagonist designed for managing preterm labor between 24 and 34 weeks of gestation, and Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily within the Swiss market while targeting global solutions for women's reproductive health needs.

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.

Apellis Pharmaceuticals Inc., established in 2009, is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts. The company specializes in developing therapeutic compounds that inhibit the complement system, focusing on autoimmune and inflammatory diseases with high unmet needs. Its lead product candidate, pegcetacoplan, is currently in Phase III trials for treating geographic atrophy in age-related macular degeneration and paroxysmal nocturnal hemoglobinuria, and in Phase II trials for various kidney and blood disorders. Additionally, Apellis is developing APL-9 for preventing immune system activation during adeno-associated virus administration. The company collaborates with Swedish Orphan Biovitrum AB (publ) to advance pegcetacoplan for treating multiple rare diseases.

Amplyx Pharmaceuticals, Inc. is a small molecule drug development company focused on creating innovative therapies for debilitating and life-threatening diseases, particularly those affecting individuals with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in the development of oral and injectable anti-infective drugs, including antifungal agents designed to treat invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by incorporating a second small molecule. This strategy aims to provide effective treatment options for patients undergoing chemotherapy and other immunosuppressive therapies, ensuring they receive appropriate care both during hospitalization and after discharge.

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

Iterum Therapeutics plc is a clinical-stage pharmaceutical company based in Dublin, Ireland, focused on developing innovative anti-infectives to address the pressing issue of multi-drug resistant pathogens. Founded in 2015, the company is advancing sulopenem, a novel penem anti-infective compound that is currently undergoing Phase III clinical trials. Sulopenem is designed for both oral and intravenous use and aims to treat various serious infections, including uncomplicated and complicated urinary tract infections as well as complicated intra-abdominal infections. The company’s mission is to significantly improve the lives of patients affected by serious and life-threatening diseases by providing effective solutions to antibiotic resistance.

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

Antiva Biosciences, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral therapies for human papillomavirus (HPV) infections. Established in 2012, the company aims to prevent cancer by treating precancerous lesions associated with HPV. Antiva's innovative approach includes localized therapeutics and small molecule antivirals that target HPV and other viral infections. The company is currently conducting clinical trials for high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias. Through its medicinal chemistry platform, Antiva strives to enhance health outcomes for patients affected by HPV-related diseases and other viral infections.

Rainier Therapeutics, Inc. is a biotechnology company focused on developing targeted therapies for bladder cancer. The company specializes in Vofatamab, a human monoclonal antibody designed to specifically block the activity of fibroblast growth factor receptor 3 (FGFR3). This innovative treatment aims to improve outcomes for patients with both early-stage and metastatic bladder cancer. Founded in 2010 and based in San Leandro, California, Rainier Therapeutics was previously known as BioClin Therapeutics, Inc. before rebranding in November 2018. Through its advanced therapeutic technology, Rainier Therapeutics seeks to enhance treatment options for those affected by this challenging disease.

InCarda Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for cardiovascular diseases. Founded in 2009 and based in Newark, California, the company specializes in a novel inhaled treatment approach to manage acute cardiovascular conditions. Its lead product, InRhythm, is an inhaled formulation of an antiarrhythmic drug designed to treat paroxysmal atrial fibrillation quickly, allowing patients to administer the therapy in various settings, such as at home or work. By delivering medication directly to cardiac tissue, InCarda's therapy aims to provide effective treatment for atrial arrhythmias with a simplified approach. The company has completed Phase 1 clinical trials and is currently advancing its lead product through Phase 2 trials.

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.

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 based in Dublin, Ireland, focused on developing innovative anti-infectives to address the pressing issue of multi-drug resistant pathogens. Founded in 2015, the company is advancing sulopenem, a novel penem anti-infective compound that is currently undergoing Phase III clinical trials. Sulopenem is designed for both oral and intravenous use and aims to treat various serious infections, including uncomplicated and complicated urinary tract infections as well as complicated intra-abdominal infections. The company’s mission is to significantly improve the lives of patients affected by serious and life-threatening diseases by providing effective solutions to antibiotic resistance.

NextCure, Inc. is a clinical-stage biopharmaceutical company based in Beltsville, Maryland, that focuses on discovering and developing novel immunomedicines aimed at treating cancer and other immune-related diseases by restoring normal immune function. Its lead product candidate, NC318, is currently undergoing Phase 2 clinical trials for advanced or metastatic solid tumors. Additionally, NextCure is developing NC410, designed to inhibit immune suppression through the modulation of a specific immune receptor. The company is also advancing other product candidates, including LNCB74, an antibody-drug conjugate targeting B7-H4, and is exploring additional immunomodulatory molecules in preclinical stages. NextCure has established collaborations with Yale University for licensing and with Eli Lilly and Company for research and development. Founded in 2015, the company leverages proprietary platforms to identify biologically relevant targets in its pursuit of first-in-class immunotherapy solutions.

First Aid Shot Therapy, founded in 2010 and headquartered in Burlingame, California, specializes in clinical beverages aimed at addressing acute and chronic health needs. The company offers a variety of easy-to-swallow, single-dose products that provide rapid relief for common everyday symptoms such as hangover, pain, sleep issues, cold, allergy, and heartburn. By focusing on fast-acting solutions, First Aid Shot Therapy aims to help consumers manage their symptoms quickly and conveniently.

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.

Aclaris Therapeutics is a clinical-stage biopharmaceutical company based in the United States, specializing in the development of innovative therapies for dermatological and immuno-inflammatory conditions. Founded by the creators of Vicept Therapeutics, the company is dedicated to addressing significant unmet needs in these fields, focusing on areas where no FDA-approved treatments exist or where treatment options are limited. Aclaris operates primarily in two segments: therapeutics and contract research. The therapeutics segment is engaged in the identification and development of novel drug candidates, while the contract research segment generates revenue through laboratory services. Among its developmental candidates is Zunsemetinib, an oral MK2 inhibitor aimed at treating immuno-inflammatory diseases.

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

Principia Biopharma Inc. is a late-stage biopharmaceutical company headquartered in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead product, rilzabrutinib, is an inhibitor undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 trials for immune thrombocytopenia and Phase 2 trials for IgG4-related disease. Additionally, Principia is developing PRN2246/SAR442168, which is in Phase II for multiple sclerosis and other central nervous system conditions, alongside PRN473, a drug candidate in Phase I trials for immune-mediated diseases. Principia Biopharma maintains a collaboration agreement with Genzyme Corporation for the development of treatments for MS and other CNS diseases. Originally incorporated in 2008, the company is dedicated to addressing significant unmet medical needs in immunology and oncology through its innovative drug development platform.

Antiva Biosciences, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on developing antiviral therapies for human papillomavirus (HPV) infections. Established in 2012, the company aims to prevent cancer by treating precancerous lesions associated with HPV. Antiva's innovative approach includes localized therapeutics and small molecule antivirals that target HPV and other viral infections. The company is currently conducting clinical trials for high-grade anal intraepithelial neoplasias and high-grade cervical intraepithelial neoplasias. Through its medicinal chemistry platform, Antiva strives to enhance health outcomes for patients affected by HPV-related diseases and other viral infections.

Mirna Therapeutics, Inc. is a discovery-stage biopharmaceutical company that specializes in developing miRNA-directed therapies for human oncology. With a strong focus on research and a deep understanding of microRNA and cancer biology, Mirna is positioned to advance the emerging field of miRNA-based therapeutics. The company boasts a robust intellectual property portfolio that supports its innovative approach to cancer treatment.

Merus B.V. is a clinical-stage biomedical company focused on the discovery and development of innovative antibody-based biopharmaceuticals. The company specializes in bispecific antibody therapeutics, utilizing its proprietary Oligoclonics technology to produce a unique class of human antibodies. This technology enables the generation of a mixture of therapeutic antibodies targeting a common antigen from a single cell clone. Merus is also advancing its full-length human multispecific antibody therapeutics, known as Biclonics. The company has a diverse pipeline of product candidates, including MCLA-128, MCLA-117, and MCLA-158, among others, all designed to address various challenges in immuno-oncology.

Edge Therapeutics is a clinical-stage biotechnology company based in Berkeley Heights, New Jersey, focused on developing innovative therapies for acute, life-threatening neurological conditions. Established in 2009, the company aims to transform treatment paradigms in hospital settings through its proprietary platform, Precisa, which facilitates the creation of polymer-based therapeutics designed for targeted delivery to injury sites. The lead product candidate, EG-1962, is currently undergoing evaluation in the Phase 3 NEWTON 2 study, aimed at improving outcomes for patients suffering from aneurysmal subarachnoid hemorrhage (aSAH). Additionally, Edge is assessing EG-1964 as a potential prophylactic treatment for chronic subdural hematoma, aimed at preventing recurrent bleeding on the brain's surface. Through these efforts, Edge Therapeutics seeks to address serious unmet medical needs in the field of neurology.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

aTyr Pharma, Inc., established in 2005 and headquartered in San Diego, California, is a biotherapeutics company dedicated to discovering and developing innovative medicines based on novel immunological pathways. The company's primary focus is efzofitimod (formerly ATYR1923), a fusion protein currently in clinical trials for treating interstitial lung diseases by modulating immune engagement through the neuropilin-2 receptor. aTyr Pharma has collaborations with academic institutions and industry partners, including the University of Nebraska Medical Center, CSL Behring, Boston Children's Hospital, Kyorin Pharmaceutical Co., Ltd., and the Medical University of South Carolina, to advance its pipeline of immunomodulatory therapeutics derived from tRNA synthetases. The company is backed by prominent life sciences investors such as Alta Partners, Cardinal Partners, Domain Associates, and Polaris Ventures.

Chiasma, Inc. is a late-stage biopharmaceutical company based in Needham, Massachusetts, established in 2001. The company specializes in developing oral medications using its proprietary Transient Permeability Enhancer technology, which allows for the conversion of injectable drugs to oral formulations. This platform not only enhances drug absorption but also enables the exploration of new indications for existing therapies. Chiasma's primary product is MYCAPSSA, an oral formulation of octreotide capsules designed for the treatment of acromegaly, a condition characterized by excessive production of growth hormone. Currently, MYCAPSSA is undergoing Phase III clinical trials in the United States and is also being developed for regulatory approval in the European Union, targeting adult patients with this rare and serious condition.

NextCure, Inc. is a clinical-stage biopharmaceutical company based in Beltsville, Maryland, that focuses on discovering and developing novel immunomedicines aimed at treating cancer and other immune-related diseases by restoring normal immune function. Its lead product candidate, NC318, is currently undergoing Phase 2 clinical trials for advanced or metastatic solid tumors. Additionally, NextCure is developing NC410, designed to inhibit immune suppression through the modulation of a specific immune receptor. The company is also advancing other product candidates, including LNCB74, an antibody-drug conjugate targeting B7-H4, and is exploring additional immunomodulatory molecules in preclinical stages. NextCure has established collaborations with Yale University for licensing and with Eli Lilly and Company for research and development. Founded in 2015, the company leverages proprietary platforms to identify biologically relevant targets in its pursuit of first-in-class immunotherapy solutions.

Iterum Therapeutics plc is a clinical-stage pharmaceutical company based in Dublin, Ireland, focused on developing innovative anti-infectives to address the pressing issue of multi-drug resistant pathogens. Founded in 2015, the company is advancing sulopenem, a novel penem anti-infective compound that is currently undergoing Phase III clinical trials. Sulopenem is designed for both oral and intravenous use and aims to treat various serious infections, including uncomplicated and complicated urinary tract infections as well as complicated intra-abdominal infections. The company’s mission is to significantly improve the lives of patients affected by serious and life-threatening diseases by providing effective solutions to antibiotic resistance.

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.

Aclaris Therapeutics is a clinical-stage biopharmaceutical company based in the United States, specializing in the development of innovative therapies for dermatological and immuno-inflammatory conditions. Founded by the creators of Vicept Therapeutics, the company is dedicated to addressing significant unmet needs in these fields, focusing on areas where no FDA-approved treatments exist or where treatment options are limited. Aclaris operates primarily in two segments: therapeutics and contract research. The therapeutics segment is engaged in the identification and development of novel drug candidates, while the contract research segment generates revenue through laboratory services. Among its developmental candidates is Zunsemetinib, an oral MK2 inhibitor aimed at treating immuno-inflammatory diseases.

Civitas Therapeutics is a biopharmaceutical company dedicated to the development and commercialization of innovative pulmonary delivery therapies aimed at treating central nervous system and respiratory disorders. Utilizing its proprietary ARCUS technology, Civitas aims to transform the therapeutic landscape for patients across the United States. The company's focus is on creating effective treatments that enhance patient outcomes and address unmet medical needs in these critical areas.

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.

Principia Biopharma Inc. is a late-stage biopharmaceutical company headquartered in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead product, rilzabrutinib, is an inhibitor undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 trials for immune thrombocytopenia and Phase 2 trials for IgG4-related disease. Additionally, Principia is developing PRN2246/SAR442168, which is in Phase II for multiple sclerosis and other central nervous system conditions, alongside PRN473, a drug candidate in Phase I trials for immune-mediated diseases. Principia Biopharma maintains a collaboration agreement with Genzyme Corporation for the development of treatments for MS and other CNS diseases. Originally incorporated in 2008, the company is dedicated to addressing significant unmet medical needs in immunology and oncology through its innovative drug development platform.

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 III trials for biliary tract and metastatic pancreatic cancers, and NUC-3373 & NUC-7738, in Phase I trials for advanced solid tumors. The company collaborates with academic institutions for research and licensing agreements. NuCana was established in 1997 and is headquartered in Edinburgh, with a focus on the U.S. market.

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 III trials for biliary tract and metastatic pancreatic cancers, and NUC-3373 & NUC-7738, in Phase I trials for advanced solid tumors. The company collaborates with academic institutions for research and licensing agreements. NuCana was established in 1997 and is headquartered in Edinburgh, with a focus on the U.S. market.

ZS Pharma, Inc. is a biopharmaceutical company based in Coppell, Texas, specializing in the development and commercialization of non-absorbed drugs aimed at treating renal, cardiovascular, liver, and metabolic diseases. Founded in 2008, the company focuses on creating highly selective ion-trap therapies using its proprietary zirconium silicate technology, which allows for the reduction of toxic electrolyte levels while maintaining overall electrolyte balance. Its lead product, ZS-9, is an insoluble, non-absorbed zirconium silicate currently in Phase III clinical trials for the treatment of hyperkalemia, a serious condition characterized by elevated potassium levels in the blood, often affecting patients with chronic kidney disease, hypertension, diabetes, or heart failure. As of December 2015, ZS Pharma operates as a subsidiary of Zeneca, Inc.

Versartis is a biotechnology company focused on developing therapeutic proteins for the treatment of endocrine disorders. Headquartered in Redwood City, California, the company utilizes its proprietary half-life extension technology, XTEN, to create innovative drug candidates aimed at improving patient outcomes. Versartis is currently advancing its lead program, VRS-317, which has recently completed enrollment in a Phase 1 study for adult patients with growth hormone deficiency. Founded in December 2008 as a joint venture between Amunix Inc. and Index Ventures, the company is staffed by experts in drug development and collaborates with external contract services to efficiently implement its product development strategies. Versartis aims to enhance treatment compliance and reduce the burden of daily injections for patients with endocrine disorders, while also targeting critical survival pathways in various malignancies.

First Aid Shot Therapy, founded in 2010 and headquartered in Burlingame, California, specializes in clinical beverages aimed at addressing acute and chronic health needs. The company offers a variety of easy-to-swallow, single-dose products that provide rapid relief for common everyday symptoms such as hangover, pain, sleep issues, cold, allergy, and heartburn. By focusing on fast-acting solutions, First Aid Shot Therapy aims to help consumers manage their symptoms quickly and conveniently.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Innocoll Holdings plc is a privately held specialty pharmaceutical and medical device company headquartered in Athlone, Ireland. It specializes in the development and manufacture of collagen-based products aimed at addressing surgical needs, particularly in post-operative care. Innocoll's product offerings include CollaGUARD, a bioresorbable collagen film designed to prevent adhesions in various types of surgeries; Collatamp Gentamicin, a surgical implant for infection prevention; Septocoll, a dual-action collagen sponge for infection treatment; and RegenePro, a collagen sponge for dental applications. The company is also advancing innovative product candidates such as XaraColl, an implantable collagen matrix in phase III trials for post-operative pain relief, and Cogenzia, a topical collagen sponge for diabetic foot infections. Innocoll leverages a proprietary technology platform to develop biodegradable products that provide non-opioid alternatives for pain management, enhancing patient care in surgical settings.

Ziarco, Inc. is a biotechnology company founded in 2012 and headquartered 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 it has established a product pipeline that includes a histamine H4 receptor antagonist program. This program targets various therapeutic areas, such as asthma, allergic rhinitis, pruritus, and skin diseases, providing innovative treatment options for conditions like atopic dermatitis and psoriasis. Through its research and development efforts, Ziarco aims to offer effective solutions for patients suffering from these challenging health issues.

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 focused on developing therapeutic proteins for the treatment of endocrine disorders. Headquartered in Redwood City, California, the company utilizes its proprietary half-life extension technology, XTEN, to create innovative drug candidates aimed at improving patient outcomes. Versartis is currently advancing its lead program, VRS-317, which has recently completed enrollment in a Phase 1 study for adult patients with growth hormone deficiency. Founded in December 2008 as a joint venture between Amunix Inc. and Index Ventures, the company is staffed by experts in drug development and collaborates with external contract services to efficiently implement its product development strategies. Versartis aims to enhance treatment compliance and reduce the burden of daily injections for patients with endocrine disorders, while also targeting critical survival pathways in various malignancies.

ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions affecting women. Its key products in development include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and heavy menstrual bleeding associated with uterine fibroids. ObsEva is also working on OBE022, a once-daily oral prostaglandin F2α receptor antagonist designed for managing preterm labor between 24 and 34 weeks of gestation, and Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily within the Swiss market while targeting global solutions for women's reproductive health needs.

First Aid Shot Therapy, founded in 2010 and headquartered in Burlingame, California, specializes in clinical beverages aimed at addressing acute and chronic health needs. The company offers a variety of easy-to-swallow, single-dose products that provide rapid relief for common everyday symptoms such as hangover, pain, sleep issues, cold, allergy, and heartburn. By focusing on fast-acting solutions, First Aid Shot Therapy aims to help consumers manage their symptoms quickly and conveniently.

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 lead product candidate, ganaxolone, functions as an allosteric modulator of GABAA receptors and is being developed in both intravenous and oral formulations for use in various patient populations, including adults and children. Ganaxolone is targeted for multiple indications, such as status epilepticus, cyclin dependent kinase like 5 deficiency disorder, PCDH19-related epilepsy, tuberous sclerosis complex, postpartum depression, and treatment-resistant depression. Marinus Pharmaceuticals emphasizes collaboration with healthcare professionals and partners to enhance patient care and improve the lives of those affected by these conditions. Founded in 2003 and headquartered in Radnor, Pennsylvania, the company is committed to advancing its therapeutic offerings through rigorous scientific research and development.

Labrys Biologics Inc. is a biotechnology company dedicated to developing innovative treatments for chronic migraines. The company has acquired worldwide rights to RN-307 from Pfizer Inc., an antibody specifically designed to address chronic migraine conditions. In addition to RN-307, Labrys Biologics is advancing its lead candidate, LBR-101, which is an anti-CGRP monoclonal antibody aimed at the prevention of chronic and high-frequency migraines. Through these targeted therapies, Labrys Biologics seeks to provide effective solutions for individuals suffering from debilitating 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 neurodegenerative diseases. Its pipeline includes PRX002, in Phase II trials for Parkinson's disease and related synucleinopathies, and PRX004, in Phase I trials for ATTR amyloidosis. Prothena also has discovery-stage programs targeting tauopathies, Alzheimer's disease, 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 innovative treatments for chronic migraines. The company has acquired worldwide rights to RN-307 from Pfizer Inc., an antibody specifically designed to address chronic migraine conditions. In addition to RN-307, Labrys Biologics is advancing its lead candidate, LBR-101, which is an anti-CGRP monoclonal antibody aimed at the prevention of chronic and high-frequency migraines. Through these targeted therapies, Labrys Biologics seeks to provide effective solutions for individuals suffering from debilitating migraine conditions.

Mirna Therapeutics, Inc. is a discovery-stage biopharmaceutical company that specializes in developing miRNA-directed therapies for human oncology. With a strong focus on research and a deep understanding of microRNA and cancer biology, Mirna is positioned to advance the emerging field of miRNA-based therapeutics. The company boasts a robust intellectual property portfolio that supports its innovative approach to cancer treatment.

Aclaris Therapeutics is a clinical-stage biopharmaceutical company based in the United States, specializing in the development of innovative therapies for dermatological and immuno-inflammatory conditions. Founded by the creators of Vicept Therapeutics, the company is dedicated to addressing significant unmet needs in these fields, focusing on areas where no FDA-approved treatments exist or where treatment options are limited. Aclaris operates primarily in two segments: therapeutics and contract research. The therapeutics segment is engaged in the identification and development of novel drug candidates, while the contract research segment generates revenue through laboratory services. Among its developmental candidates is Zunsemetinib, an oral MK2 inhibitor aimed at treating immuno-inflammatory diseases.

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 established 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 manufactures and distributes dermatological therapeutic products, positioning itself as a key player in addressing the unmet needs of patients with these skin disorders.