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MGTX vs VOR

MeiraGTx Holdings plc Ordinary Shares and Vor Biopharma 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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Vor Biopharma Inc. Common Stock

Vor Biopharma Inc. operates as a clinical-stage cell and genome engineering company. The company offers the Vor Bio platform, a technology platform for the treatment of acute myeloid leukemia (AML) and other blood cancers. It develops tremtelectogene empogeditemcel (trem-cel), a genome-edited hematopoietic stem and progenitor allogeneic donor product candidate to treat acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and other blood cancers; trem-cel in combination with Mylotarg to treat patients in VBP101, which is in phase 1 / 2; and VCAR33, a CAR-T therapy designed to target CD33, which is under preclinical studies to treat AML post-transplant. The company also develops trem-cel + VCAR33 treatment system, CD33-directed CAR-T program, which is under pre-clinical studies for the treatment of AML; VADC45 for the treatment of hematologic malignancies, conditioning, and immune reset, which is under preclinical studies; and CD33-CLL1 treatment system made with CD33-CLL1 multiplex-edited HSC therapy and a CD33-CLL1 multi-specific CAR-T therapy, which is under preclinical studies for the treatment of AML. It has a license agreement with Columbia University and the National Institutes of Health for patents; and with Editas Medicine, Inc. for ex-vivo Cas9 gene-edited HSC therapies for the treatment and/or prevention of hematological malignancies. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

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