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PRAX vs QURE

Praxis Precision Medicines, Inc. Common Stock and uniQure N.V.

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Praxis Precision Medicines, Inc. Common Stock

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance. The company's platforms include cerebrum, a small molecule platform for CNS small molecule therapies to develop orally available precision therapies; and solidus to discover and develop antisense oligonucleotide. It also develops Ulixacaltamide, a small molecule inhibitor of T-type calcium channels that is in Phase 3 clinical trial for the treatment of essential tremor; Vormatrigine, a small molecule targeting the hyperexcitable state of sodium-channels in the brain to treat focal onset epilepsy; Relutrigine for the treatment of developmental and epileptic encephalopathies; PRAX-020 to treat KCNT1; and PRAX-050. In addition, the company develops Elsunersen, a clinical-stage ASO designed to regulate NaV1.2 expression for the treatment of SCN2A-DEE; PRAX-080 that targets PCDH19-related developmental epilepsy; PRAX-090 for targeting SYNGAP1 loss-of-function; and PRAX-100 that targets SCN2A LoF mutations. It has a license agreement with RogCon Inc.; and research collaboration and license agreement with Ionis Pharmaceuticals, Inc. Praxis Precision Medicines, Inc. was incorporated in 2015 and is based in Boston, Massachusetts.

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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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