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 ↗Theriva Biologics, Inc.
Theriva Biologics, Inc., a clinical-stage company, develops therapeutics to treat cancer and related diseases in areas of high unmet need in the United States. The company's lead product candidate is VCN-01, a clinical stage oncolytic human adenovirus that is in a Phase 2 clinical study for the treatment of pancreatic cancer; a Phase 1 clinical study for the treatment of retinalblastoma; a Phase 1 clinical study for the treatment of head and neck squamous cell carcinoma, ovarian cancer, colorectal cancer; and a Phase 1 clinical study for the treatment of solid tumors. It also develops VCN-11 for treating solid tumors; SYN-004 designed to degrade various commonly used intravenous beta-lactam antibiotics in gastrointestinal (GI) tract for the prevention of microbiome damage to prevent overgrowth and infection by pathogenic organisms, such Clostridioides difficile infection and vancomycin resistant Enterococci, and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant recipients; and SYN-020, a recombinant oral formulation for the enzyme intestinal alkaline phosphatase to treat both local GI and systemic diseases. In addition, the company develops clinical stage products, such as SYN-006 to prevent aGVHD and infection by carbapenem resistant enterococci; and SYN-007 for preventing antibiotic associated diarrhea with oral ß-lactam antibiotics. It has collaboration agreements with The University of Texas at Austin, Catalan Institute of Oncology, Fundació Privada Institut d'Investigacio Biomedica de Bellvitge, Saint Joan De Déu, and Massachusetts General Hospital, as well as a clinical trial agreement with Washington University School of Medicine in St. Louis to conduct a Phase 1b/2a clinical trial of SYN-004. Theriva Biologics, Inc. is headquartered in Rockville, Maryland.
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