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ESSA Pharma Inc., a clinical stage pharmaceutical company, focuses the development of small molecule drugs for the treatment of prostate cancer. The company's lead candidate EPI-7386, an androgen receptor based resistance mechanisms that develop in patients with castration-resistant prostate cancer and metastatic castration-resistant prostate cancer. It has collaboration agreements with Bayer Consumer Care AG; Janssen Research & Development, LLC; and Astellas Pharma Inc. ESSA Pharma Inc. was incorporated in 2009 and is headquartered in Vancouver, Canada.

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.

KemPharm, Inc. is a clinical-stage specialty pharmaceutical company, which engages in the discovery and development of proprietary prodrugs. It focuses on the treatment of serious medical conditions such as attention deficit hyperactivity disorder, pain, and other central nervous system disorders through its platform technology known as Ligand Activated Therapy. The company was founded by Christal M. M. Mickle and Travis C. Mickle on October 30, 2006 and is headquartered in Celebration, FL.

Magenta Therapeutics, Inc., a biotechnology company focus on improving stem cell transplantation. Its product candidates are designed to bring the curative power of stem cell transplant to patients with blood cancers, genetic diseases, and autoimmune diseases. The company's product portfolio includes MGTA-117, an anti-CD117 antibody conjugated to an amanitin payload that targets hematopoietic stem cells (HSCs) and leukemia cells; and MGTA-45, an anti-human CD45 antibody conjugated to a DNA-interacting payload for HSCs, leukemia cells, and immune cells. In addition, it focus on development of ADC-based conditioning program that targets a receptor of T cells; and novel conditioning and translation stem cell sciences, as well as develops cell therapy program, E478, which is a small molecule aryl hydrocarbon receptor antagonist for stem cell-based gene therapy and genome editing. In addition, the company has a research and clinical collaboration agreement with AVROBIO, Inc. for the treatment of lysosomal storage disorders; and Beam Therapeutics, Inc. to evaluate the potential utility of MGTA-117 for conditioning of patients with sickle cell disease and beta-thalassemia. Magenta Therapeutics, Inc. was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.