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Adverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.

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.

Codiak BioSciences, Inc., a clinical-stage biopharmaceutical company, focuses on developing exosome-based therapeutics for the treatment of oncology, neuro-oncology, neurology, neuromuscular diseases, infectious diseases, and other diseases. Its lead product candidates are exoSTING and exoIL-12 exosome therapeutic candidates for the treatment of various solid tumors. The company is also developing exoASO-STAT6, an antisense oligonucleotide targeting the STAT6 transcription factor; and exoVACC, a modular platform for constructing precision vaccines. It has a collaboration agreement with the Jazz Pharmaceuticals Ireland Limited, Sarepta Therapeutics, Inc., Kayla Therapeutics S.A.S., Washington University, Ragon Institute of MGH, MIT, and Harvard. 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.

Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology company, develops novel cell therapies for unmet medical needs in the United States and internationally. The company develops OpRegen, an allogeneic retinal pigment epithelium cell replacement therapy, which is in Phase 2a clinical trial for the treatment of the dry age-related macular degeneration; OPC1, an allogeneic oligodendrocyte progenitor cell therapy that is in Phase 1/2a multicenter clinical trial for the treatment of cervical spinal cord injuries; and VAC, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells, which is in Phase I clinical trial to treat non-small cell lung cancer. It also offers ANP1, an allogeneic auditory neuron progenitor cell transplant, which is in preclinical development for the treatment of debilitating hearing loss; and PNC1, an allogeneic photoreceptor cell transplant, which is in preclinical development for the treatment of vision loss due to photoreceptor dysfunction or damage. In addition, the company engages in the research and development of therapeutic products for retinal diseases, neurological diseases, and disorders and oncology. Lineage Cell Therapeutics, Inc. has a collaboration with Immunomic Therapeutics, Inc., for the treatment of glioblastoma multiforme. The company was formerly known as BioTime, Inc. and changed its name to Lineage Cell Therapeutics, Inc. in August 2019. Lineage Cell Therapeutics, Inc. was incorporated in 1990 and is headquartered in Carlsbad, California.