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Abeona 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.

Humanigen, Inc., a clinical-stage biopharmaceutical company, focuses on preventing and treating an immune hyper-response. The company is developing lenzilumab, an antibody that binds to and neutralizes granulocyte-macrophage colony-stimulating factor (GM-CSF), to treat cytokine storm associated with COVID-19 for which it has completed a Phase 3 study and is the focus of a Phase 2/3 study sponsored by the NIH. It is also developing lenzilumab, which is associated with CD19-targeted CAR-T cell therapies, as well as exploring the effectiveness of lenzilumab in other inflammatory conditions, such as acute Graft versus Host Disease in patients undergoing allogeneic hematopoietic stem cell transplantation and in eosinophilic asthma, and rheumatoid arthritis. In addition, the company focuses on studying lenzilumab for patients with chronic myelomonocytic leukemia exhibiting RAS pathway mutations. Its pipeline also includes two other Humaneered monoclonal antibodies, ifabotuzumab, which binds to EphA3, and HGEN005, which targets EMR1, as well as treats a range of eosinophilic diseases, including eosinophilic leukemia as an optimized naked antibody and as the backbone for a novel CAR-T construct. Humanigen, Inc. was incorporated in 2000 and is headquartered in Short Hills, New Jersey. On January 3, 2024, Humanigen, Inc. filed a voluntary petition for reorganization under Chapter 11 in the U.S. Bankruptcy Court for the District of Delaware.

NexImmune, Inc., a clinical-stage biotechnology company, engages in developing therapies with curative potential for patients with cancer and other life-threatening immune-mediated diseases. It develops approaches to T cell immunotherapies based on its proprietary Artificial Immune Modulation, a nanoparticle technology platform. The company's product candidates include NEXI-001, an allogeneic cell therapy, which is in Phase I/II clinical trials for the treatment of patients with acute myeloid leukemia; NEXI-002, an autologous cell therapy that is in Phase I/II clinical trials for the treatment of patients with relapsed and/or refractory in multiple myeloma; and NEXI-003, a candidate targeted against HPV-associated malignancies, which is in preclinical stage. The company was incorporated in 2011 and is headquartered in Gaithersburg, Maryland.

Panbela Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of disruptive therapeutics for the treatment of patients with urgent unmet medical needs. The company's lead product candidates are Ivospemin (SBP-101), a proprietary polyamine analogue, which has completed Phase Ia/Ib clinical trial for the treatment of patients with metastatic pancreatic ductal adenocarcinoma; Flynpovi, a combination of eflornithine (CPP-1X) and sulindac which is in Phase III clinical trials; and Eflornithine, an enzyme-activated irreversible inhibitor of the enzyme ornithine decarboxylase, currently under Phase I/II trial. It has a research agreement with the Johns Hopkins University School of Medicine for the development of ivospemin. The company was formerly known as Sun BioPharma, Inc. and changed its name to Panbela Therapeutics, Inc. in December 2020. Panbela Therapeutics, Inc. was incorporated in 2011 and is based in Waconia, Minnesota.