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STOK vs XFOR

Stoke Therapeutics, Inc. Common Stock and X4 Pharmaceuticals, Inc. Common Stock

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Stoke Therapeutics, Inc. Common Stock

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, engages in the development of treatments for severe genetic diseases by upregulating protein expression. The company utilizes its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach in developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Its lead product candidates include STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy; and Zorevunersen (STK-001), an investigational new medicine for the treatment of Dravet syndrome is being evaluated in phase I/II clinical trials. The company also develops programs focused on various targets, including haploinsufficiency diseases of the central nervous system and eye. It has a license and collaboration with Biogen Inc. for the development and commercialization of zorevunersen medicine for the treatment of Dravet syndrome; and Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of genetic neurodevelopmental diseases. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.

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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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