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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.

Freeline Therapeutics Holdings plc, a clinical-stage biotechnology company, develops transformative adeno-associated virus (AAV) vector-mediated gene therapies. It develops FLT180a for the treatment of hemophilia B.; FLT201 for the treatment of Type 1 Gaucher disease; and FLT190 for the treatment of Fabry disease. in addition, it has research programs for systemic gene therapy. The company was founded in 2015 and is headquartered in Stevenage, the United Kingdom.

Gritstone bio, Inc., a clinical-stage biotechnology company, engages in developing vaccine-based immunotherapy candidates against cancer and infectious diseases. Its primary product candidate is GRANITE, an individualized immunotherapy candidate, which is in Phase 2/3 clinical trials for the treatment of microsatellite stable colorectal cancers; and has completed Phase 1/2 clinical trials for treating solid tumors. The company is also developing SLATE, an off-the-shelf immunotherapy candidate, which is in Phase 2 clinical trials for the treatment of metastatic solid tumors. In addition, it develops CORAL, a COVID-19 vaccine program; and a therapeutic vaccine candidate that is in Phase 1 clinical trials designed to treat and cure human immunodeficiency virus (HIV) infection. Gritstone bio, Inc. has a strategic collaboration with bluebird bio, Inc.; collaboration agreement with Gilead Sciences, Inc.; and license agreement with Genevant Sciences GmbH. The company was formerly known as Gritstone Oncology, Inc. and changed its name to Gritstone bio, Inc. in May 2021. Gritstone bio, Inc. was incorporated in 2015 and is headquartered in Emeryville, California.

Jounce Therapeutics, Inc. is a clinical stage immunotherapy company, which engages in developing treatments for cancer. It also provides novel cancer immunotherapies to attack tumors. The company was founded by Louis M. Weiner, Drew M. Pardoll, Thomas F. Gajewski, James P. Allison, Robert Schreiber, and Padmanee Sharma in 2013 and is headquartered in Cambridge, MA.

Opthea Limited, a clinical stage biopharmaceutical company, engages in the development and commercialization of therapies primarily for eye disease in Australia. The company's development activities are based on the intellectual property portfolio covering Vascular Endothelial Growth Factors (VEGF) VEGF-C, VEGF-D, and VEGF Receptor-3 for the treatment of diseases associated with blood and lymphatic vessel growth, as well as vascular leakage. Its lead product candidate is Sozinibercept (OPT 302), a soluble form of vascular endothelial growth factor receptor-3 VEGFR-3, currently under Phase 3 clinical development as a novel therapy for wet age-related macular degeneration and diabetic macular edema. The company was formerly known as Circadian Technologies Limited and changed its name to Opthea Limited in December 2015. Opthea Limited was incorporated in 1984 and is based in South Yarra, Australia.