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Mateon Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing drugs for the treatment of orphan oncology indications. The company's lead product candidate is OT-101, an antisense against TGF-beta, which is in phase 3 clinical trials for pancreatic cancer and glioblastoma, as well as develops OT-101 for the treatment of various viruses, including severe acute respiratory syndrome and coronavirus. It also develops OXi4503 for the treatment of acute myeloid leukemia and myelodysplastic syndromes; and CA4P for the treatment of advanced metastatic melanoma. The company is headquartered in Agoura Hills, California.

Novelion Therapeutics Inc., a biopharmaceutical company, develops a portfolio of therapies for individuals living with rare diseases in the United States, Brazil, and internationally. The company's commercial products include lomitapide hard capsule for the treatment of homozygous familial hypercholesterolemia in adults under the JUXTAPID and LOJUXTA brands; and metreleptin, a recombinant analogue of human leptin, which is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy under the MYALEPT brand. Its orphan drug-designated product candidate is zuretinol acetate, an oral synthetic retinoid, which is in development for the treatment of inherited retinal disease caused by underlying mutations in retinal pigment epithelium protein 65 and lecithin: retinol acyltransferase genes. The company was formerly known as QLT Inc. and changed its name to Novelion Therapeutics Inc. in November 2016. Novelion Therapeutics Inc. was founded in 1981 and is headquartered in Vancouver, Canada.

RegeneRx Biopharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of therapeutic peptide, Thymosin beta 4, for tissue and organ protection, repair, and regeneration. The company is developing RGN-259, a preservative-free topical eye drop for regeneration of corneal tissues damaged by injury, disease, or other pathology; RGN-352, an injectable formulation for the treatment of cardiovascular diseases, central and peripheral nervous system diseases, and other medical indications; and RGN-137, a topical gel for the treatment of dermal wounds and reduction of scar tissue. It has strategic partnerships with Lee's Pharmaceutical Group and HLB Therapeutics. The company was formerly known as Alpha 1 Biomedicals, Inc. and changed its name to RegeneRx Biopharmaceuticals, Inc. in 2000. RegeneRx Biopharmaceuticals, Inc. was incorporated in 1982 and is based in Rockville, Maryland.

Soligenix, Inc., a late-stage biopharmaceutical company, focuses on developing and commercializing products to treat rare diseases in the United States. The company operates through two segments, Specialized BioTherapeutics and Public Health Solutions. The Specialized BioTherapeutics segment develops SGX301 (HyBryte), a novel photodynamic therapy, which has completed Phase III clinical trial for the treatment of cutaneous T-cell lymphoma; SGX942, an innate defense regulator technology that is in Phase III clinical trial for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer; SGX302, an IDR technology which is in Phase IIa study to treat mil-to-moderate Psoriasis; and SGX945 and IDR technology that is in Phase IIa protocol for the treatment of Aphthous Ulcers in Behçet's Disease. The Public Health Solutions segment is involved in the development of RiVax, a ricin toxin vaccine candidate, which has completed Phase Ia, Ib, and Ic clinical trials; SGX943, a therapeutic candidate that is in pre-clinical stage for the treatment of antibiotic-resistant and emerging infectious diseases; ThermoVax, a technology in pre-clinical development for thermostabilizing vaccines; and CiVax, a vaccine candidate for the prevention of COVID-19. The company was formerly known as DOR BioPharma, Inc. and changed its name to Soligenix, Inc. in 2009. Soligenix, Inc. was incorporated in 1987 and is headquartered in Princeton, New Jersey.

Tocagen Inc., a clinical-stage cancer-selective gene therapy company, focuses on developing and commercializing product candidates designed to activate a patient's immune system against their cancer. Its cancer-selective gene therapy platform is built on retroviral replicating vectors (RRVs), which are designed to deliver therapeutic genes into the DNA of cancer cells. The company's lead product candidate is Toca 511 & Toca FC that is under Phase III clinical trial for recurrent high-grade glioma. It is also developing Toca 511 & Toca FC in a Phase Ib clinical trial for intravenous treatment of advanced cancers. In addition, the company is developing other RRVs to deliver genes to cancer cells against validated immunotherapy targets. The company has a license agreement with ApolloBio to develop and commercialize Toca 511 & Toca FC; and a collaboration agreement with NRG Oncology to develop a clinical trial utilizing Toca 511 & Toca FC for the treatment of patients with newly diagnosed glioblastoma. Tocagen Inc. was founded in 2007 and is based in San Diego, California.