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PRAX vs RGNX

Praxis Precision Medicines, Inc. Common Stock and REGENXBIO Inc.

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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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REGENXBIO Inc.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase III clinical trial for the treatment of Duchenne muscular dystrophy, as well as RGX-121 for the treatment of mucopolysaccharidosis type II. It also develops RGX-111 for the treatment of mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for the treatment of the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.

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