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 ↗Genprex, Inc. Common Stock
Genprex, Inc., a clinical-stage gene therapy company, focuses on developing therapies for patients with cancer and diabetes. The company's technologies are designed to administer disease-fighting genes to provide new therapies for patient populations with cancer and diabetes. Its oncology program utilizes its systemic, non-viral Oncoprex Delivery System, which encapsulates the gene-expressing plasmids using lipid-based nanoparticles in a lipoplex form. The company's lead product candidate, Reqorsa Immunogene Therapy (quaratusugene ozeplasmid), is being evaluated in three clinical trials as a treatment for NSCLC and SCLC. Its three lung cancer clinical programs have received a Fast Track Designation from the FDA for the treatment of that patient population, and SCLC program has received an FDA Orphan Drug Designation. The company's diabetes gene therapy comprises of a novel infusion process that uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, its GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which produce insulin but distinct enough from beta cells to evade the body's immune system; and GPX-002 for Type 2 diabetes, where autoimmunity is not at play, is believed to rejuvenate and replenish exhausted beta cells. It has research agreement with The University of Michigan Rogel Cancer Center and Lung Cancer Study Collaboration with ALK Positive. Genprex, Inc. was incorporated in 2009 and is headquartered in Austin, Texas.
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