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QURE vs STOK

uniQure N.V. and Stoke Therapeutics, Inc. Common Stock

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uniQure N.V.

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in Phase I/II clinical study for the treatment of Huntington's disease. The company also develops AMT-260, which is in Phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in Phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

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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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