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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Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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