Sofinnova Investments

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.

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.

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.

Axonis Therapeutics is a biotechnology company focused on developing therapies for neurological disorders by targeting KCC2, a key mediator of inhibition in the brain. The company utilizes a proprietary discovery engine centered on the neuron-specific K-Cl cotransporter (KCC2) to address various conditions, including epilepsy and pain, as well as other central nervous system pathologies. By harnessing breakthrough research from institutions such as Boston Children’s Hospital, Harvard, and Laval University, Axonis aims to enhance the lives of individuals affected by these disorders through improved modulation of neuronal inhibition.

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.

Aeovian Pharmaceuticals is a biopharmaceutical company focused on developing innovative therapeutics for age-related and rare diseases. Founded in 2012 and based in the San Francisco Bay Area, the company specializes in compounds that inhibit the mTORC1 and mTORC2 pathways. These therapeutics aim to address the underlying causes of disease initiation and progression, including oxidative, cellular, and environmental stress. By targeting these fundamental factors, Aeovian Pharmaceuticals seeks to provide effective treatment options for patients and healthcare professionals.

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.

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

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.

OnCusp Therapeutics is a biopharmaceutical company focused on developing innovative oncology therapies aimed at improving treatments for cancer patients worldwide. Founded by Bing Yuan, Eric Slosberg, and Andy Fu, the company is dedicated to translating cutting-edge research into clinical applications. It specializes in advancing preclinical drug candidates to the clinical proof-of-concept stage, enabling a swift transition into global early clinical development. By prioritizing this process, OnCusp Therapeutics aims to accelerate the innovation of oncology medications, ultimately providing critical support and hope to those affected by cancer.

Long Description: Aiolos Bio is a biotech company focused on developing therapies for respiratory diseases like asthma as well as other immune conditions. The company is initially focused on advancing AIO-001, a monoclonal antibody therapy for moderate-to-severe asthma that only requires dosing every 6 months. Aiolos Bio was founded by an accomplished team with decades of drug development experience and leadership in the biotech industry.

Star Therapeutics is a biotechnology company focused on developing innovative therapies for a wide range of rare diseases. The company aims to identify diseases that share common biological characteristics and create novel therapeutics that can effectively treat multiple conditions with a single treatment. This approach not only addresses the needs of patients suffering from rare diseases but also presents a unique opportunity for pharmaceutical companies to broaden their therapeutic offerings within a streamlined pipeline. By concentrating on life-changing therapies, Star Therapeutics aspires to make a significant impact on the lives of millions of patients.

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.

Quanta Therapeutics is a biotechnology company focused on developing innovative cancer therapies targeting driver oncogenes, specifically RAS-driven cancers. The company employs advanced optical technology to discover small molecules that can modulate the conformation of RAS proteins. This allosteric modulation aims to restore conformational control of active RAS signaling at the cell membrane, thereby inhibiting a wide range of cancers associated with RAS mutations. By addressing the challenges of previously untreatable cancers, Quanta Therapeutics is committed to advancing new treatment options for patients and enhancing the understanding of RAS biology in cancer therapy.

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.

Quanta Therapeutics is a biotechnology company focused on developing innovative cancer therapies targeting driver oncogenes, specifically RAS-driven cancers. The company employs advanced optical technology to discover small molecules that can modulate the conformation of RAS proteins. This allosteric modulation aims to restore conformational control of active RAS signaling at the cell membrane, thereby inhibiting a wide range of cancers associated with RAS mutations. By addressing the challenges of previously untreatable cancers, Quanta Therapeutics is committed to advancing new treatment options for patients and enhancing the understanding of RAS biology in cancer therapy.

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.

Visen Pharmaceuticals is a biopharmaceutical company that specializes in the development and commercialization of innovative therapies for endocrine diseases. The company is dedicated to addressing significant unmet medical needs in this field, particularly within the Chinese market, and aims to provide both first-in-class and best-in-class treatment solutions for patients of all ages, including those with rare endocrine conditions. With a commitment to patient-centric care, Visen strives to improve treatment outcomes and quality of life for individuals affected by these diseases. The company is backed by a team of experienced professionals with extensive backgrounds in the pharmaceutical industry and operates offices in major Chinese cities such as Shanghai, Beijing, Hong Kong, and Taipei. To further enhance its research, development, and manufacturing capabilities, Visen has established a Greater China research and development site in Suzhou, aiming to deliver advanced treatment options to Chinese patients more effectively.

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.

Bolt Biotherapeutics, Inc. is a biotechnology company focused on developing innovative immunotherapy solutions for cancer treatment. The company employs its proprietary Boltbody platform, which features immune-stimulating antibody conjugates (ISAC) that link tumor-targeting antibodies with powerful immune stimulants. This approach aims to activate the immune system against tumors, effectively transforming cold tumors into immunologically active ones. Bolt Biotherapeutics is advancing several candidates in its pipeline, including BDC-1001, a monotherapy targeting HER2-expressing solid tumors, and BDC-3042, among others. Founded in 2015 and headquartered in Redwood City, California, the company leverages its expertise in myeloid biology and cancer drug development to create targeted therapies that harness both innate and adaptive immune responses.

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.

Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating 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 combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

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.

Aeovian Pharmaceuticals is a biopharmaceutical company focused on developing innovative therapeutics for age-related and rare diseases. Founded in 2012 and based in the San Francisco Bay Area, the company specializes in compounds that inhibit the mTORC1 and mTORC2 pathways. These therapeutics aim to address the underlying causes of disease initiation and progression, including oxidative, cellular, and environmental stress. By targeting these fundamental factors, Aeovian Pharmaceuticals seeks to provide effective treatment options for patients and healthcare professionals.

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.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

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.

Visen Pharmaceuticals is a biopharmaceutical company that specializes in the development and commercialization of innovative therapies for endocrine diseases. The company is dedicated to addressing significant unmet medical needs in this field, particularly within the Chinese market, and aims to provide both first-in-class and best-in-class treatment solutions for patients of all ages, including those with rare endocrine conditions. With a commitment to patient-centric care, Visen strives to improve treatment outcomes and quality of life for individuals affected by these diseases. The company is backed by a team of experienced professionals with extensive backgrounds in the pharmaceutical industry and operates offices in major Chinese cities such as Shanghai, Beijing, Hong Kong, and Taipei. To further enhance its research, development, and manufacturing capabilities, Visen has established a Greater China research and development site in Suzhou, aiming to deliver advanced treatment options to Chinese patients more effectively.

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.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

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.

Rainier Therapeutics is a biotechnology company focused on developing targeted therapies for bladder cancer. The company specializes in Vofatamab, a human monoclonal antibody designed to inhibit the activity of FGFR3 (fibroblast growth factor receptor 3), which is crucial in the treatment of both early-stage and metastatic bladder cancer. Founded in 2010 and based in San Leandro, California, Rainier Therapeutics was previously known as BioClin Therapeutics before rebranding in November 2018. Through its innovative approach, Rainier Therapeutics aims to enhance treatment options for patients suffering from this challenging disease.

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.

Quanta Therapeutics is a biotechnology company focused on developing innovative cancer therapies targeting driver oncogenes, specifically RAS-driven cancers. The company employs advanced optical technology to discover small molecules that can modulate the conformation of RAS proteins. This allosteric modulation aims to restore conformational control of active RAS signaling at the cell membrane, thereby inhibiting a wide range of cancers associated with RAS mutations. By addressing the challenges of previously untreatable cancers, Quanta Therapeutics is committed to advancing new treatment options for patients and enhancing the understanding of RAS biology in cancer therapy.

Pionyr Immunotherapeutics Inc. is a biotechnology company based in San Francisco, California, that focuses on developing innovative cancer immunotherapies. Founded in 2015, the company aims to enhance the body's antitumor immunity by targeting the tumor microenvironment. Utilizing advanced target discovery and antibody generation technologies, Pionyr has created a unique approach called Myeloid Tuning™, which modifies the cellular infiltrate within tumors to improve the immune system's response against cancer. This strategy is intended to be particularly effective when used in conjunction with checkpoint inhibitors, offering new avenues for cancer treatment.

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.

Medeor Therapeutics, Inc. is a biotechnology company based in San Mateo, California, focused on researching, developing, and commercializing personalized cellular immunotherapies. Founded in 2012, the company specializes in creating innovative treatments aimed at improving outcomes for kidney transplant recipients. Under the MDR-10X brand, Medeor Therapeutics develops cellular immunotherapy products that enhance organ transplant immune tolerance and advance immuno-oncology. These therapies are designed to better preserve or improve kidney function and reduce the risk of graft rejection compared to traditional immunosuppressive drugs.

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 such as endometriosis, heavy menstrual bleeding, and preterm labor. Its key products include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and uterine fibroids, and OBE022, a selective prostaglandin F2α receptor antagonist designed for once-daily treatment of preterm labor. Additionally, ObsEva is advancing 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 in Switzerland and is committed to improving outcomes for women facing reproductive health challenges.

Entasis Therapeutics is a late-stage clinical biopharmaceutical company dedicated to developing innovative treatments for serious infections caused by multidrug-resistant Gram-negative bacteria, a growing global health concern. The company’s anti-infective discovery platform has led to a diverse pipeline of differentiated drug candidates, including ETX2514SUL and SUL-DUR, which target Acinetobacter baumannii, zoliflodacin for Neisseria gonorrhoeae, and ETX0282CPDP aimed at Enterobacterales. Additionally, ETX0462 is being developed to address infections caused by Pseudomonas. Through its focused efforts, Entasis Therapeutics aims to provide effective solutions for patients suffering from these challenging bacterial infections.

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 is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating 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 combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.

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.

AFYX Therapeutics A/S is a pharmaceutical company based in Copenhagen, Denmark, specializing in the development and manufacturing of treatments for mucosal diseases. Founded in 2014 and formerly known as Dermtreat ApS, the company focuses on addressing conditions that currently lack approved therapies. Its flagship product, the Rivelin®-CLO patch, 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 therapeutic agents directly to affected areas. The design of the patch allows for effective treatment without disrupting patients’ daily activities, thereby facilitating recovery from mucosal lesions.

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.

Rainier Therapeutics is a biotechnology company focused on developing targeted therapies for bladder cancer. The company specializes in Vofatamab, a human monoclonal antibody designed to inhibit the activity of FGFR3 (fibroblast growth factor receptor 3), which is crucial in the treatment of both early-stage and metastatic bladder cancer. Founded in 2010 and based in San Leandro, California, Rainier Therapeutics was previously known as BioClin Therapeutics before rebranding in November 2018. Through its innovative approach, Rainier Therapeutics aims to enhance treatment options for patients suffering from this challenging disease.

Dauntless Pharmaceuticals, Inc. is a biopharmaceutical company based in San Diego, California, focused on developing specialty therapeutic drugs for endocrine cancers. The company offers Dauntless 1, a treatment specifically designed for these cancers, and is also developing DP1038, another therapeutic agent in the same area. Established in 2015, Dauntless Pharmaceuticals aims to provide innovative alternatives to existing treatments, including a non-injectable somatostatin analog, which offers a less invasive option compared to traditional injectable therapies used for conditions like acromegaly and neuroendocrine tumors. The company holds a diverse portfolio of assets at various stages of development, reflecting its commitment to advancing patient care through its therapeutic innovations.

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.

Dauntless Pharmaceuticals, Inc. is a biopharmaceutical company based in San Diego, California, focused on developing specialty therapeutic drugs for endocrine cancers. The company offers Dauntless 1, a treatment specifically designed for these cancers, and is also developing DP1038, another therapeutic agent in the same area. Established in 2015, Dauntless Pharmaceuticals aims to provide innovative alternatives to existing treatments, including a non-injectable somatostatin analog, which offers a less invasive option compared to traditional injectable therapies used for conditions like acromegaly and neuroendocrine tumors. The company holds a diverse portfolio of assets at various stages of development, reflecting its commitment to advancing patient care through its therapeutic innovations.

First Aid Shot Therapy is a company specializing in clinical beverages aimed at addressing acute and chronic health needs. Founded in 2010 and headquartered in Burlingame, California, it offers a variety of easy-to-swallow, single-dose products that deliver fast-acting relief for common everyday symptoms such as hangovers, pain, sleep issues, colds, allergies, and heartburn. The company's focus is on providing effective solutions that 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, 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.

Dauntless Pharmaceuticals, Inc. is a biopharmaceutical company based in San Diego, California, focused on developing specialty therapeutic drugs for endocrine cancers. The company offers Dauntless 1, a treatment specifically designed for these cancers, and is also developing DP1038, another therapeutic agent in the same area. Established in 2015, Dauntless Pharmaceuticals aims to provide innovative alternatives to existing treatments, including a non-injectable somatostatin analog, which offers a less invasive option compared to traditional injectable therapies used for conditions like acromegaly and neuroendocrine tumors. The company holds a diverse portfolio of assets at various stages of development, reflecting its commitment to advancing patient care through its therapeutic innovations.

Crocus Technology SA specializes in magnetic random access memory products and technologies tailored for the semiconductor and electronic systems industries. The company develops magnetic sensors and embedded memory technology that facilitate efficient sensor data gathering and processing. Its products are designed for diverse applications, including telecommunications, networking, storage, computing, and handheld devices. Crocus Technology's innovative solutions enable fast read and write operations while maintaining a compact footprint, which enhances chip performance and security for devices such as smart cards, mobile phones, and data servers. By focusing on high sensitivity, stable temperature performance, and low power consumption, the company aims to improve productivity for its clients across various sectors.

Mirna Therapeutics, Inc. (Mirna) is a discovery-stage biopharmaceutical research and development company focused on miRNA-directed human oncology therapies. Featuring world-class research capabilities, a strong understanding of miRNA and cancer biology, and an expansive IP portfolio, Mirna Therapeutics is well-positioned to capitalize on the emerging field of miRNA-based therapeutics.

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.

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 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.

Vestagen Technical Textiles, founded in 2009 and based in Orlando, Florida, specializes in developing, manufacturing, and marketing innovative textile products and technologies. The company holds exclusive North American rights to patented Swiss technologies for medical textiles, allowing for versatile applications ranging from medical uniforms to athletic apparel. Vestagen's primary focus is on addressing critical needs within the healthcare industry, particularly through its proprietary VESTEX Active Barrier fabric. This advanced material is designed to protect healthcare workers and patients by minimizing the risks associated with fluid exposure and reducing the retention of harmful microorganisms on clothing.

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.

Histogenics Corporation, founded in 2000 and headquartered in Waltham, Massachusetts, is a regenerative medicine company specializing in the development and commercialization of therapeutic medical products for musculoskeletal conditions. The company's innovative platform integrates cell processing, scaffolding, tissue engineering, bioadhesives, and growth factors to address cartilage damage and improve joint function. Its primary investigational product, NeoCart, is a tissue implant designed to treat cartilage injuries in the knee, aiming to regenerate healthy cartilage and enhance overall joint performance. In 2019, Histogenics was acquired by Ocugen, Inc., marking a significant transition in its corporate structure.

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 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.