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Aerie Pharmaceuticals, Inc., a pharmaceutical company, focuses on the discovery, development, and commercialization of ophthalmic therapies for open-angle glaucoma, dry eye, diabetic macular edema, and wet age-related macular degeneration in the United States. Its products include Rhopressa, a once-daily eye drop to reduce elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension; and Rocklatan, a once-daily fixed-dose combination of Rhopressa and latanopros to reduce IOP to treat patients with open-angle glaucoma or ocular hypertension. The company also develops AR-15512 to treat signs and symptoms of dry eye; and AR-1105 and AR-14034 SR sustained-release implants focused on retinal diseases. It has a collaborative research, development, and licensing agreement with DSM Biomedical. Aerie Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Durham, North Carolina.

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.

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.

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.

Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company's lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson's disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.