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MGTX vs RAPP

MeiraGTx Holdings plc Ordinary Shares and Rapport Therapeutics, Inc. Common Stock

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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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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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