Passage Bio, Inc. Common Stock
Passage Bio, Inc., a genetic medicines company, develops gene therapies for central nervous system diseases. It develops PBGM01, a functional GLB1 gene encoding ß-galactosidase for infantile GM1; PBFT02, a functional granulin (GRN) and gene encoding progranulin (PGRN) for the treatment of FTD caused by progranulin deficiency; and PBKR03, a functional GALC gene encoding the hydrolytic enzyme galactosylceramidase for infantile Krabbe disease. The company develops PBML04 for the treatment of metachromatic leukodystrophy; PBAL05 for the treatment of amyotrophic lateral sclerosis; and other program for huntington's disease. It has a strategic research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program; and collaboration agreement, and a development services and clinical supply agreement with Catalent Maryland, Inc. The company was incorporated in 2017 and is based in Philadelphia, Pennsylvania.
Open PASG dashboard ↗X4 Pharmaceuticals, Inc. Common Stock
X4 Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases of the immune system. It offers XOLREMDI, an orally bioavailable selective antagonist of chemokine receptor C-X-C chemokine receptor type 4 (CXCR4) for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome. The company also develops mavorixafor that is in Phase 3 4WARD clinical trial to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor with or without stable doses of G-CSF in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications; and with Norgine to develop, manufacture, and commercialize mavorixafor in Europe, Australia, and New Zealand. The company is headquartered in Boston, Massachusetts.
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