Pharvaris N.V., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for rare diseases. The company develops PHA121, a small molecule bradykinin B2-receptor antagonist for the treatment of hereditary angioedema (HAE). It also develops PHVS416, an on-demand, rapid exposure soft capsule for patients suffering from acute HAE attacks which is under Phase 2 clinical trial; and PHVS719, a prophylactic extended-release tablet for HAE patients which is under Phase 1 clinical trial. The company operates in the Netherlands, Switzerland, and the United States. Pharvaris N.V. was incorporated in 2015 and is based in Leiden, the Netherlands.

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

Pharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company offers RUCONEST, a recombinant C1 esterase inhibitor for the treatment of acute attacks in adult and adolescent patients with acute hereditary angioedema (HAE); and Joenja (leniolisib), an oral small molecule PI3K? inhibitor for the treatment of activated phosphoinositide 3-kinase delta syndrome. It also develops OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. was incorporated in 1988 and is headquartered in Leiden, the Netherlands.

Esperion Therapeutics, Inc., a pharmaceutical company, develops and commercializes medicines for the treatment of patients with elevated low density lipoprotein cholesterol (LDL-C). Its marketed products include NEXLETOL (bempedoic acid) and NEXLIZET (bempedoic acid and ezetimibe) tablets that are oral, once-daily, non-statin medicines for the treatment of primary hyperlipidemia in adults with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular disease who require additional lowering of LDL-C. The company's products also include NILEMDO, an ATP Citrate Lyase (ACL) inhibitor that lowers LDL-C and cardiovascular risk by reducing cholesterol biosynthesis and up-regulating the LDL receptors; and NUSTENDI, a bempedoic acid and ezetimibe tablet to treat elevated LDL-C. The company has license and collaboration agreements with Daiichi Sankyo Co. Ltd to; Otsuka Pharmaceutical Co., Ltd; and Daiichi Sankyo Europe GmbH. Esperion Therapeutics, Inc. was incorporated in 2008 and is headquartered in Ann Arbor, Michigan.

Genfit S.A., a late-stage biopharmaceutical company, discovers and develops drug candidates and diagnostic solutions for metabolic and liver-related diseases. The company develops Elafibranor, which is in Phase III clinical trial to treat patients with primary biliary cholangitis. It also engages in the development of NIS4 technology for the diagnosis of nonalcoholic steatohepatitis (NASH) and fibrosis; VS-01 for the treatment of Urea Cycle Disorder (UCD) and Organic Acidemia Disorder (OAD); GNS561, which is in Phase 1b/2a trial to treat patients with cholangiocarcinoma (CCA); VS-01-ACLF and Nitazoxanide (NTZ), which is in Phase 1 trial to treat acute-on-chronic liver failure, as well as VS-02-HE, which is in preclinical trial for the treatment of Reduction of Hyperammonemia and the Stabilization of Blood Ammonia; CML-022; SRT-015, an ASK1 inhibitor targets the inhibition of cellular apoptosis, inflammation, and fibrosis. The company has a licensing agreement with Labcorp for the commercialization of NASHnext, a blood-based molecular diagnostic test; and Genoscience Pharma to develop and commercialize the investigational treatment GNS561 for CCA. The company was incorporated in 1999 and is headquartered in Loos, France.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

MetaVia Inc., a clinical-stage biotechnology company focuses on developing and commercializing novel pharmaceuticals to treat cardiometabolic diseases. It develops DA-1241, a novel G-Protein-Coupled Receptor 119 agonist with development optionality as a standalone and/or combination therapy that is in Phase 2a clinical trial for metabolic dysfunction-associated steatohepatitis (MASH), and has completed Phase 1 clinical trial for the treatment of type 2 diabetes mellitus (T2DM); and DA-1726, a novel oxyntomodulin analogue functioning as a GLP-1 receptor and glucagon receptor dual agonist, which is in preclinical development for the treatment of obesity. The company's therapeutic programs include ANA001, a proprietary oral niclosamide formulation for the treatment of patients with moderate COVID-19; NB-01 for the treatment of painful diabetic neuropathy; NB-02 for the treatment of cognitive impairment; and Gemcabene for the treatment of dyslipidemia. It has a license agreement with Pfizer Inc. for the research, development, manufacture, and commercialization of Gemcabene; and joint research agreement with Dong-A ST and ImmunoForge for the development of DA-1726. The company was formerly known as NeuroBo Pharmaceuticals, Inc. and changed its name to MetaVia Inc. in November 2024. MetaVia Inc. is headquartered in Cambridge, Massachusetts.

MetaVia Inc., a clinical-stage biotechnology company focuses on developing and commercializing novel pharmaceuticals to treat cardiometabolic diseases. It develops DA-1241, a novel G-Protein-Coupled Receptor 119 agonist with development optionality as a standalone and/or combination therapy that is in Phase 2a clinical trial for metabolic dysfunction-associated steatohepatitis (MASH), and has completed Phase 1 clinical trial for the treatment of type 2 diabetes mellitus (T2DM); and DA-1726, a novel oxyntomodulin analogue functioning as a GLP-1 receptor and glucagon receptor dual agonist, which is in preclinical development for the treatment of obesity. The company's therapeutic programs include ANA001, a proprietary oral niclosamide formulation for the treatment of patients with moderate COVID-19; NB-01 for the treatment of painful diabetic neuropathy; NB-02 for the treatment of cognitive impairment; and Gemcabene for the treatment of dyslipidemia. It has a license agreement with Pfizer Inc. for the research, development, manufacture, and commercialization of Gemcabene; and joint research agreement with Dong-A ST and ImmunoForge for the development of DA-1726. The company was formerly known as NeuroBo Pharmaceuticals, Inc. and changed its name to MetaVia Inc. in November 2024. MetaVia Inc. is headquartered in Cambridge, Massachusetts.