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MGTX vs RGNX

MeiraGTx Holdings plc Ordinary Shares and REGENXBIO Inc.

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MeiraGTx Holdings plc Ordinary Shares

MeiraGTx Holdings plc, a clinical stage genetics medicines company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases; neurodegenerative diseases; and xerostomia. It is also developing AAV-AQP1 in Phase 2 trial for radiation-induced grade 2/3 radiation-induced xerostomia, as well as in Phase ½ trial for Sjogren's syndrome; AAV-GAD in Phase 2 trial for Parkinson's disease; AAV-RPE65 in Phase 2 trial for RPE65-associated retinal dystrophy; and AAV-CNGB3 and AAV-CNGA3, which are both in Phase 2 trial for achromatopsia, as well as offers AAV-AIPL1 for Leber congenital amaurosis (LCA) 4. In addition, the company's products under preclinical development include AAV-RDH12 for retinol dehydrogenase 12 mutation-associated retinal dystrophy; AAV-BBS10 for Bardet-Biedl syndrome (BBS) due to BBS10 mutations; AAV-RetGC for LCA type 1; and AAV-UPF1 for amyotrophic lateral sclerosis, as well as programs for Alzheimer's disease, and wet and dry neovascular age related macular degeneration. Further, it provides riboswitch platform for the delivery of metabolic peptides; and cell therapies for oncology, autoimmune disease, and long term intractable pain. The company has collaborations with Johnson & Johnson Innovative Medicine for the research, development, and commercialization of gene therapies for the treatment of inherited retinal disease; and Hologen Limited for the research, development, manufacture, and commercialization of AAV-GAD investigational gene therapy for the treatment of Parkinson's disease, AAV-BDNF investigational gene therapy for genetic obesity disorders, and other genetic medicines to the central nervous system. MeiraGTx Holdings plc was founded in 2015 and is based in New York, New York.

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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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