Novartis AG engages in the research, development, manufacture, and marketing of healthcare products in Switzerland and internationally. The company offers prescription medicines for patients and physicians. The company also provides Pluvicto, lutetium Lu 177 vipivotide tetraxetan, indicated for the treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer; and Lutathera, lutetium Lu 177 dotatate, a prescription medicine used to treat adults and children aged 12 years and older with gastroenteropancreatic neuroendocrine tumors. It focuses on therapeutic areas, such as cardiovascular, renal and metabolic, immunology, neuroscience, and oncology, as well as ophthalmology and hematology. The company has a license and collaboration agreement with Alnylam Pharmaceuticals to develop, manufacture, and commercialize inclisiran, a therapy to reduce LDL cholesterol; and Dawn Health for the development and commercialization of Ekiva, a digital solution designed for people living with Paroxysmal Nocturnal Hemoglobinuria. It also has a research collaboration and license agreement with Schrödinger, Inc. to identify and develop various therapeutic candidates; and a strategic collaboration with Vyriad, Inc. for the discovery and development for vivo chimeric antigen receptor T-cell therapies. The company was incorporated in 1996 and is headquartered in Basel, Switzerland.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

NewAmsterdam Pharma Company N.V., a late-stage biopharmaceutical company, develops therapies to enhance patient care in populations with metabolic disease. It is developing obicetrapib, an oral low-dose cholesteryl ester transfer protein (CETP) inhibitor, that is in various clinical trials as a monotherapy and a combination therapy with ezetimibe for lowering LDL-C for cardiovascular diseases. The company also develops Obicetrapib which is in Phase 2a clinical trial for Alzheimer's disease. NewAmsterdam Pharma Company N.V. is headquartered in Naarden, the Netherlands.

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.

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.

Voyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.

4D Molecular Therapeutics, Inc., a clinical-stage biotherapeutics company, develops genetic medicines using its therapeutic vector evolution platform in the Netherland and the United States. The company develops a portfolio of genetic medicine product candidates focuses in three therapeutic areas for ophthalmology, cardiology, and pulmonology. Its product pipeline includes 4D-150, which is in phase 1/2 clinical trial for the treatment of wet age-related macular degeneration; and is in phase 1/2 clinical trial for the treatment of diabetic macular edema. The company develops 4D-125, which is in phase 1/2 clinical trial for the treatment of x-linked retinitis pigmentosa; 4D-110, which is in phase 1/2 clinical trial for the treatment of choroideremia; 4D-710, which is in phase 1/2 clinical trial for the treatment cystic fibrosis lung disease; and 4D-310 to treat fabry disease cardiomyopathy and is in phase 1/2 clinical trial. In addition, its product candidates comprises 4D-175, which is in pre-clinical development for the treatment for geographic trophy; and 4D-725 to treat alpha-1 antitrypsin deficiency lung disease and is in pre-clinical development. The company has collaboration and licensing agreements with Arbor Biotechnologies, Inc.; Astellas Gene Therapies, Inc; uniQure biopharma B.V.; and Cystic Fibrosis Foundation. 4D Molecular Therapeutics, Inc. was founded in 2013 and is headquartered in Emeryville, California.

NGM Biopharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of novel therapeutics to treat liver and metabolic diseases, retinal diseases, and cancer. The company develops NGM707, an immunoglobulin-like transcript 2/ immunoglobulin-like transcript 4 dual antagonist monoclonal antibody that is in Phase I/II clinical trials for the treatment of patients with advanced metastatic solid tumors; and Aldafermin, an engineered analog of human hormone fibroblast growth factor 19, which is in Phase IIb clinical trials for the treatment of primary sclerosing cholangitis and non-alcoholic steatohepatitis. It also develops NGM120, an GFRAL antagonist antibody, which is in Phase II clinical trial to treat hyperemesis gravidarum; NGM831 and NGM438 which is in phase I/II clinical trial for the treatment of advanced solid tumors; and NGM621, an monoclonal antibody in Phase II clinical trials for the treatment of geographic atrophy. The company has research collaboration, product development, and license agreements with Merck Sharp & Dohme Corp.; and a collaboration agreement with Merck to focus primarily on the identification, research and development of collaboration compounds directed to targets in the fields of ophthalmology and cardiovascular or metabolic, or CVM, disease, including heart failure. NGM Biopharmaceuticals, Inc. was incorporated in 2007 and is headquartered in South San Francisco, California.