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AVROBIO, Inc., a clinical-stage gene therapy company, develops ex vivo lentiviral-based gene therapies to treat rare diseases following a single dose worldwide. 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, as well as to treat Gaucher disease type 3; AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease; AVR-RD-04, which is in phase 1/2 clinical trial for the treatment of cystinosis; and AVR-RD-05 that is in preclinical stage for the treatment of Hunter syndrome. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

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.

Oyster Point Pharma, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of pharmaceutical therapies to treat ophthalmic diseases in the United States. The company's product candidate is TYRVAYA, a nicotinic acetylcholine receptor agonist for the treatment of signs and symptoms of dry eye disease. It is also developing TYRVAYA that is in Phase II clinical trial for the treatment of for neurotrophic keratopathy. The company was incorporated in 2015 and is headquartered in Princeton, New Jersey.

Rubius Therapeutics, Inc., a biopharmaceutical company, focuses on the development of red cell therapeutics for the treatment of cancer and autoimmune diseases. The company was formerly known as VL26, Inc and as changed its name to Rubius Therapeutics, Inc. in 2015. Rubius Therapeutics, Inc. was incorporated in 2013 and is based in Foxborough, Massachusetts.