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Generation Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Janux Therapeutics, Inc., a clinical stage biopharmaceutical company, develops immunotherapies based on Tumor Activated T Cell Engagers (TRACTr) and Tumor Activated Immunomodulators (TRACIr) platforms technology to treat patients suffering from cancer. The company's clinical candidates comprise JANX007, a prostate-specific membrane antigen or PSMA-TRACTr, which is in Phase 1 clinical trial in adults for the treatment of metastatic castration-resistant prostate cancer (mCRPC) and the vasculature of other tumors; and JANX008, an epidermal growth factor receptor or EGFR-TRACTr that is in Phase 1 clinical trial for the treatment of multiple solid cancers, including colorectal cancer, squamous cell carcinoma of the head and neck, non-small cell lung cancer, and renal cell carcinoma. Its development pipeline also consists of discovery stage products. The company has strategic research collaboration agreement with Merck Sharp & Dohme Corp. to develop TRACTr product candidates. Janux Therapeutics, Inc. was incorporated in 2017 and is headquartered in San Diego, California.

NRx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops novel therapeutics based on N-methyl-D-aspartate platform for the treatment of central nervous system disorders, including suicidal depression, post-traumatic stress disorder, and chronic pain. Its products include NRX-101 (D-cycloserine/Lurasidone), an oral, fixed dosed combination of D-cycloserine and lurasidone that earned FDA-designated investigational breakthrough therapy for suicidal treatment-resistant bipolar depression and chronic pain; and NRX-100 (ketamine), which has been awarded FDA fast track designation for the treatment of acute suicidality in depression. The company has a partnership with Alvogen and Lotus to develop and commercialize NRX-101 for the suicidal bipolar depression treatment, as well as an agreement with Nephron Pharmaceuticals, Inc. to develop ketamine. The company was founded in 2015 and is based in Wilmington, Delaware.

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

Vectura Group Plc engages in the provision of pharmaceutical development. Its activities include research, development, and commercialization of novel therapeutic products. The company operates through the following segments: Switzerland, United Kingdom, Germany, United States of America, and France. Vectura Group was founded by David Anthony Gough in 1997 and is headquartered in Chippenham, the United Kingdom.