Abeona Therapeutics
NASDAQ:ABEOAbeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.
Genprex
NASDAQ:GNPXGenprex, Inc., a clinical-stage gene therapy company, focuses on developing therapies for patients with cancer and diabetes. Its lead product candidate is REQORSA (GPX-001) that is in Phase 1/2 and 2 clinical trials to treat non-small cell lung cancer and small cell lung cancer. The company is also developing GPX-002 for the treatment of type 1 diabetes, and GPX-003 for the treatment of type 2 diabetes. Genprex, Inc. was incorporated in 2009 and is headquartered in Austin, Texas.
IO Biotech
NASDAQ:IOBTIO Biotech, Inc., a clinical-stage biopharmaceutical company, develops immune-modulating cancer therapies based on the T-win technology platform. The company's lead product candidate, IO102-IO103, which is designed to target immunosuppressive proteins, such as Indoleamine 2,3-dehydrogenase (IDO), and programmed death-ligand (PD-L1) that is in phase 3 clinical trial to treat melanoma, as well as in phase 1 clinical trial to treat lung, head and neck, bladder, and melanoma cancer. It also develops IO112, a product candidate that contains a single Arginase 1-derived peptide designed to target T cells that recognize epitopes derived from Arginase 1 for the treatment of cancers. The company was incorporated in 2014 and is based in Copenhagen, Denmark.
Oncternal Therapeutics
NASDAQ:ONCTOncternal Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of oncology therapies for cancers with critical unmet medical needs. The company's clinical pipeline includes zilovertamab, a humanized monoclonal antibody that binds to receptor-tyrosine kinase-like Orphan Receptor 1 (ROR1); and ONCT-216, a small molecule inhibiting the biological activity of ETS-family transcription factor oncoproteins, which is in Phase 1/2 clinical trial. It is also developing ONCT-808, a chimeric antigen receptor T-cells (CAR-T), which is in Phase 1/2 clinical trial for the treatment of hematologic malignancies and solid tumors, as well as targets ROR1; and ONCT-534, a dual-action androgen receptor inhibitor product candidate in preclinical development for the treatment of castration-resistant prostate and other androgen receptor-driven cancers. Oncternal Therapeutics, Inc. has license agreements with the Regents of the University of California; Georgetown University; The University of Texas MD Anderson Cancer Center; Shanghai Pharmaceutical (USA) Inc.; and University of Tennessee Research Foundation. Oncternal Therapeutics, Inc. has a research collaboration with Celularity Inc. to evaluate placental derived-cellular therapies targeting ROR1. The company is headquartered in San Diego, California.