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COMPASS Pathways plc operates as a mental health care company in the United Kingdom and the United States. It develops COMP360, a psilocybin therapy that is in Phase III clinical trials for the treatment of treatment-resistant depression; and is in Phase II clinical trials for the treatment of post-traumatic stress disorder and anorexia nervosa. The company was formerly known as COMPASS Rx Limited and changed its name to COMPASS Pathways plc in August 2020. COMPASS Pathways plc was incorporated in 2020 and is headquartered in London, the United Kingdom.

Design Therapeutics, Inc. a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.

Eagle Pharmaceuticals, Inc., a pharmaceutical company, focuses on developing and commercializing product candidates to treat diseases of the central nervous system or metabolic critical care, and oncology in the United States. The company offers Ryanodex for malignant hyperthermia; and Belrapzo and Bendeka for chronic lymphocytic leukemia and indolent B-cell non-Hodgkin's lymphoma. Its product candidates also include EP-4104, a dantrolene sodium to treat organophosphate exposure; PEMFEXY, a ready-to-use/dilute liquid form of pemetrexed for non-small cell lung cancer and mesothelioma; EA-114 (fulvestrant) for HR+/HER- breast cancer; and Vasopressin injection, which is indicated to enhance blood pressure in adults with vasodilatory shock. The company has license and collaboration agreements with Combioxin, SA for the development and commercialization rights to CAL02, an antitoxin agent for the treatment of severe pneumonia in combination with traditional antibacterial drugs; and AOP Orphan Pharmaceuticals GmbH for the commercial rights of Landiolol, a novel therapeutic product candidate for the short-term reduction of ventricular rate in patients with supraventricular tachycardia, including atrial fibrillation and atrial flutter. It has a strategic collaboration with Tyme Technologies, Inc. for the development of SM-88 to treat breast cancer (HR+/HER2-) and high-risk metastatic sarcomas. Eagle Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Woodcliff Lake, New Jersey.

Wave Life Sciences Ltd., a clinical-stage biotechnology company, designs, develops, and commercializes ribonucleic acid (RNA) medicines through PRISM, a discovery and drug development platform. The company's RNA medicines platform, PRISM, combines multiple modalities, chemistry innovation, and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. It is developing WVE-006, a RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; siRNA clinical candidate for the treatment of obesity and other metabolic disorders; WVE-N531, a exon skipping oligonucleotide for the treatment of duchenne muscular dystrophy; and WVE-003, an antisense silencing oligonucleotide for the treatment of Huntington's disease (HD). The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was incorporated in 2012 and is based in Singapore.