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Albireo Pharma, Inc. is a clinical stage biopharmaceutical company, which engages in the development and commercialization of novel bile acid modulators for the treatment of orphan pediatric liver diseases and gastrointestinal disorders. Its pipeline products include Odevixibat, A3384, and Elobixibat. The company was founded by Jan Peter Mattsson and Per-Göran Gillberg on December 8, 2003 and is headquartered in Boston, MA.

Aerovate Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops drugs that enhance the lives of patients with rare cardiopulmonary diseases in the United States. It focuses on advancing AV-101, a dry powder inhaled formulation of imatinib for the treatment of pulmonary arterial hypertension, which is in Phase 2b/Phase 3 trial. The company was incorporated in 2018 and is headquartered in Waltham, Massachusetts.

BioXcel Therapeutics, Inc., a commercial-stage biopharmaceutical company, engages in utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology. The company's drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indices. Its commercial product, IGALMI, a sublingual film formulation of dexmedetomidine for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. The company also continues to conduct clinical trials evaluating BXCL501 for the acute treatment of agitation in Alzheimer's disease patients, and for adjunctive treatment of patients with major depressive disorder, as well as in the community for agitation associated with bipolar disorders and schizophrenia. In addition, it is developing BXCL502 as a potential therapy for chronic agitation in dementia; and BXCL701, an investigational, orally innate immunity activator for the treatment of aggressive forms of prostate cancer, pancreatic cancer, and other solid and liquid tumors; BXCL503, a drug candidate to target apathy in dementia; and BXCL504, a drug candidate to target aggression in dementia. BioXcel Therapeutics, Inc. was incorporated in 2017 and is headquartered in New Haven, Connecticut.

Dyne Therapeutics, Inc., a clinical-stage muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It is developing a portfolio of muscle disease therapeutics, including programs in myotonic dystrophy type 1; duchenne muscular dystrophy; and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapeutics. Dyne Therapeutics, Inc. was incorporated in 2017 and is headquartered in Waltham, Massachusetts.

Generation Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.