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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.

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the delivery of RNA therapeutics. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat diseases previously untreatable with RNA therapeutics. The company's lead product candidate AOC 1001 for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease that is in phase 1/2 clinical trial. Its other products in pipeline include AOC 1044 for the treatment of duchenne muscular dystrophy, which is under phase 1/2 clinical development trial; and AOC 1020 to treat facioscapulohumeral muscular dystrophy that is in phase 1/2 clinical trial. The company is developing products for rare skeletal muscle and rare cardiac diseases. Avidity Biosciences, Inc. was incorporated in 2012 and is headquartered in San Diego, California.

Trillium Therapeutics, Inc. is a clinical stage immuno-oncology company, which engages in the research and development of therapies for the treatment of cancer. It offers clinical programs, TTI-621 and TTI-622, target CD47, a signal that cancer cells frequently use to evade the immune system. The company was founded on March 31, 2004 and is headquartered in Mississauga, Canada.