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 ↗Rafael Holdings, Inc. Class B Common Stock
Rafael Holdings, Inc. primarily engages in holding interests in clinical and early-stage pharmaceutical companies, and medical devices in the United States and Israel. It operates in three segments, Healthcare, Infusion Technology, and Real Estate. The company engages in the development and commercialization of therapies that exploit the metabolic differences between normal cells and cancer cells. Its lead drug candidate is Trappsol Cyclo, which is in phase 3 clinical trial being evaluated for the potential treatment of Niemann-Pick Disease Type C1 (NPC1), a rare, fatal and progressive genetic disorder; and CPI-613 (devimistat), a stable analog of normally transient, acylated catalytic intermediates of lipoate, which is in phase II of clinical trial. The company is also involved in developing Promitil, a molecule designed for the targeted delivery of mitomycin-C in a proprietary prodrug form, completed Phase 1A and 1B clinical studies targeting patients with advanced cancers. In addition, it engages in the development of surgical and procedural devices, including orthopedic arthroscopy instrument for Carpal Tunnel syndrome. Rafael Holdings, Inc. was incorporated in 2017 and is headquartered in Newark, New Jersey.
Open RFL dashboard ↗