Design Therapeutics, Inc. Common Stock
Design Therapeutics, Inc. a clinical-stage biopharmaceutical company, research, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.
Open DSGN dashboard ↗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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