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QURE vs RAPP

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

Rapport Therapeutics, Inc., operates as a clinical-stage biopharmaceutical company that focuses on the discovery and development of transformational small molecule medicines for patients suffering from central nervous system (CNS) disorders. Its lead product candidate is receptor associated protein (RAP)-219, an investigational small molecule that is designed to inhibit TARPy8-containing AMPARs with picomolar affinity for the treatment of focal epilepsy and other CNS disorders, including peripheral neuropathic pain and bipolar disorder. The company also develops RAP-199, a TARPy8 targeted molecule with differentiated chemical and pharmacokinetic properties; and nicotinic acetylcholine receptor (nAChR) programs, such as a6 nAChR to treat chronic pain and a9a10 nAChR for the treatment of hearing disorders. The company was formerly known as Precision Neuroscience NewCo, Inc. and changed its name to Rapport Therapeutics, Inc. in October 2022. Rapport Therapeutics, Inc. was incorporated in 2022 and is based in Boston, Massachusetts.

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