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 ↗Zevra Therapeutics, Inc. Common Stock
Zevra Therapeutics, Inc., a commercial-stage company, focuses on addressing unmet needs for the treatment of rare diseases in the United States. The company develops its products through Ligand Activated Technology platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase 1/2 clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 3 clinical trial for the treatment of vascular Ehlers Danlos syndrome. In addition, it offers AZSTARYS, a once-daily treatment for attention deficit and hyperactivity disorder in patients aged six years and older; OLPRUVA to treat urea cycle disorders; and MIPLYFFA for the treatment of niemann pick disease type C, an ultra-rare neurodegenerative lysosomal storage disorder. The company has collaboration and license agreement with Commave Therapeutics SA to develop, manufacture and commercialize the company's product candidates containing SDX and d-methylphenidate; and license agreement with Acer and Relief Therapeutics, Inc. to develop and commercialize rights for OLPRUVA. The company was formerly known as KemPharm, Inc. and changed its name to Zevra Therapeutics, Inc. in February 2023. Zevra Therapeutics, Inc. was incorporated in 2006 and is headquartered in Celebration, Florida.
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