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AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

CEL-SCI Corporation, a clinical-stage biotechnology company, engages in the research and development of immune system therapy for the treatment of cancer and other diseases in the United States. The company's lead immunotherapy is Multikine, which has completed Phase 3 clinical trials for the potential treatment of certain head and neck cancers. It is also developing products based on its proprietary technology Ligand Epitope Antigen Presentation System (LEAPS) technology, a patented T-cell modulation process that stimulates the human immune system to fight bacterial, viral, parasitic infections, autoimmune conditions, allergies, transplantation rejections, and cancer. In addition, the company's product pipeline includes CEL-2000; CEL-4000; and CEL-5000, which are LEAPS-based product candidates in preclinical trials for the treatment of rheumatoid arthritis disease. CEL-SCI Corporation was incorporated in 1983 and is headquartered in Vienna, Virginia.

Fusion Pharmaceuticals Inc., a clinical-stage oncology company, focuses on developing radiopharmaceuticals as precision medicines in Canada and the United States. The company's lead product candidates include FPI-2265 that is in Phase 2 clinical trial to treat prostate-specific membrane antigens; and FPI-1434 that is in Phase 1 clinical trial as a monotherapy in patients with solid tumors expressing that target insulin-like growth factor 1 receptor. It also conducting additional preclinical studies of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors to further assess the anti-tumor activity, and dosing schedule and pharmacodynamics of the combinations. In addition, the company develops FPI-1966 for the treatment of multiple cancers, including colorectal, ovarian, bladder, and head and neck cancers.; FPI-2068, a bispecific antibody, which is in Phase 1 clinical trial targeting epidermal growth factor receptor, and mesenchymal epithelial transition factor. It has a strategic collaboration agreement with AstraZeneca UK Limited to discover, develop, and commercialize alpha-emitting radiopharmaceuticals and combination therapies for the treatment of cancer. Fusion Pharmaceuticals Inc. was incorporated in 2014 and is headquartered in Hamilton, Canada.

MeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases, and xerostomia. Its programs in clinical development include Phase I/II clinical stage programs in achromatopsia, X-linked retinitis pigmentosa, and RPE65-deficiency; Phase I clinical trials for radiation-induced xerostomia; and Parkinson's program that has completed a Phase II trial. It has a research collaboration agreement with Janssen Pharmaceuticals, Inc. for the research, development, and commercialization of gene therapies for the treatment of inherited retinal disease. The company was incorporated in 2015 and is based in New York, New York.

Proteon Therapeutics, Inc., a late-stage biopharmaceutical company, focuses on the development of pharmaceuticals to address the needs of patients with renal and vascular disease. Its lead product candidate is vonapanitase, an investigational drug intended to enhance hemodialysis vascular access outcomes. The company develops vonapanitase, a recombinant human elastase, which has completed Phase II and Phase III clinical trials for patients with chronic kidney disease. It is also evaluating vonapanitase in a Phase I clinical trial in patients with peripheral artery disease. The company was founded in 2001 and is based in Waltham, Massachusetts.