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Eliem Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing therapies for neuronal excitability disorders to address unmet needs in chronic pain, psychiatry, epilepsy, and other disorders of the peripheral and central nervous systems. Its lead drug candidate is ETX-123, a Kv7.2/3 potassium channel opener, which is in preclinical for the treatment of neuronal excitability disorders, such as epilepsy, pain, depression, and others; and ETX-155 for the treatment of major depressive disorder and focal onset seizures that is in Phase 2a clinical trial. The company was incorporated in 2018 and is headquartered in Redmond, Washington.

Epizyme, Inc., a commercial-stage biopharmaceutical company, discovers, develops, and commercializes novel epigenetic medicines for patients with cancer and other diseases in the United States. The company offers Tazemetostat for the treatment of metastatic or locally advanced epithelioid sarcoma for adults and pediatric patients. It also develops R-CHOP in front-line patients with high risk diffuse large B-cell lymphoma; and PARP inhibitor in patients with PARPi-resistant solid tumors, such as castration-resistant prostate cancer, small cell lung cancer, and others. In addition, the company develops Tazemetostat in patients with INI1-negative tumors in adults and pediatrics; PRMT5 inhibitor for patients with solid tumors; and PRMT1 inhibitor. It has collaboration agreements with Roche Molecular Systems, Inc.; Lymphoma Academic Research Organization; Eisai Co. Ltd.; HUTCHMED (China) Limited; and Roche Sequencing Solutions, Inc. The company was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

KalVista Pharmaceuticals, Inc., a clinical stage pharmaceutical company, engages in the discovery, development, and commercialization of small molecule protease inhibitors for diseases with unmet needs in the United States. The company's product portfolio comprises small molecule plasma kallikrein inhibitors targeting hereditary angioedema (HAE) and diabetic macular edema (DME); and oral plasma kallikrein inhibitors. Its products include sebetralstat, which is initiation of the Phase 3 KONFIDENT trial as a potential oral, on-demand therapy for HAE attacks; and Factor XIIa, an oral inhibitor program which is in preclinical stage targets an enzyme in HAE. The company is headquartered in Cambridge, Massachusetts.

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.