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QURE vs SLDB

uniQure N.V. and Solid Biosciences 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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Solid Biosciences Inc. Common Stock

Solid Biosciences Inc. develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, which is in phase 1/2 for the treatment of Duchenne muscular dystrophy. It also develops SGT-212 for the treatment of Friedreich's ataxia that is in phase 1b clinical trial; SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia that is in preclinical phase; SGT-601 the treatment of TNNT2-mediated dilated cardiomyopathy, which is in preclinical phase; SGT-401 for the treatment of BAG3-mediated dilated cardiomyopathy that is in preclinical phase; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, which is in discovery phase; and other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries; genetic regulators, such as promoters, UTRs, and introns; immunomodulation technologies; manufacturing purity; and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc.; and a license agreement with the University of Washington, University of Missouri, University of Florida, and ICS Maugeri S.p.A. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.

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