Cabaletta Bio, Inc. Common Stock
Cabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with autoimmune diseases. Its lead product candidate is resecabtagene autoleucel, a 4-1BB co-stimulatory domain-containing fully human CD19-CAR T, which is in Phase 1/2 clinical trials for the treatment of dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, juvenile myositis, lupus nephritis, non-renal systemic lupus erythematosus, relapsing and progressive multiple sclerosis, mucocutaneous and mucosal pemphigus vulgaris (PV), systemic sclerosis, and generalized myasthenia gravis. The company also develops DSG3-CAART, a CAAR T cell therapy that is in Phase 1 clinical trial for the treatment of mucosal PV; and MuSK-CAART, an investigational cell therapy in Phase 1 trial for treating patients with anti-muscle-specific kinase (MuSK) antibody positive myasthenia gravis. It has collaborations with the University of Pennsylvania and the Children's Hospital of Philadelphia; Nanjing IASO Biotherapeutics Co., Ltd; Oxford Biomedica; Autolus Holdings (UK) Limited; and WuXi Advanced Therapies, Inc. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.
Open CABA dashboard ↗Harmony Biosciences Holdings, Inc. Common Stock
Harmony Biosciences Holdings, Inc., a commercial-stage pharmaceutical company, focuses on developing and commercializing therapies for patients with rare and other neurological diseases in the United States. The company offers WAKIX (pitolisant), a molecule with a novel mechanism of action for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. It also develops Pitolisant that is in Phase 3 clinical trial for the treatment of Prader-Willi Syndrome (PWS); in Phase 2 clinical trial to treat Myotonic Dystrophy (DM1); and is in Phase 1 clinical trials for the treatment of Pitolisant Gastro-Resistant (GR) and High-Dose (HD). In addition, the company's products under development include BP1.15205, an orexin 2 receptor agonist for the treatment of narcolepsy and other potential indications; and HBS-102, a melanin-concentrating hormone receptor type 1 (MCHR1) antagonist. Further, it develops ZYN-002 that is in a Phase 3 registrational trial for the treatment of Fragile X Syndrome (FXS), rare genetic disorder that causes inherited intellectual disability and autism spectrum disorder; and 22q Deletion Syndrome, a disorder caused by a small missing piece on the long arm of the 22nd chromosome. Additionally, the company is developing EPX-100 (clemizole hydrochloride), a serotonin (5HT-2) receptor agonist to treat dravet syndrome and lenox-gastaut syndrome; and EPX-200 (liquid formulation of lorcaserin), a selective 5HT-2C agonist to treat developmental and epileptic encephalopathies (DEE). The company was formerly known as Harmony Biosciences II, Inc. and changed its name to Harmony Biosciences Holdings, Inc. in February 2020. Harmony Biosciences Holdings, Inc. was incorporated in 2017 and is headquartered in Plymouth Meeting, Pennsylvania.
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