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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.

Cortexyme, Inc., a clinical stage biopharmaceutical company, focuses on developing therapeutics for Alzheimer's and other degenerative diseases. Its lead drug candidate is atuzaginstat (COR388), an orally administered brain-penetrating small molecule gingipain inhibitor, which is in Phase II/III clinical trial for use in patients with mild to moderate Alzheimer's disease, as well as for the treatment of oral squamous cell carcinoma, periodontitis, and coronavirus infection. The company was incorporated in 2012 and is headquartered in South San Francisco, California.

Mesoblast Limited engages in the development of regenerative medicine products in Australia, the United States, Singapore, and Switzerland. The company offers products in the areas of cardiovascular, spine orthopedic disorder, oncology, hematology, and immune-mediated and inflammatory diseases. Its proprietary regenerative medicine technology platform is based on specialized cells known as mesenchymal lineage cells. The company offers Remestemcel-L that is in Phase III clinical trials for the treatment of systemic inflammatory diseases, including steroid refractory acute graft versus host disease, acute respiratory distress syndrome, and biologic refractory inflammatory bowel disease; and Remestemcel-L, which is in Phase III clinical trials to treat chronic heart failure and chronic low back pain due to degenerative disc disease. It is also developing MPC-300-IV to treat biologic refractory rheumatoid arthritis diabetic nephropathy; and MPC-25-IC for the treatment or prevention of acute myocardial infarction. It has strategic partnerships with Tasly Pharmaceutical Group to offer MPC-150-IM for heart failure and MPC-25-IC for heart attacks in China; JCR Pharmaceuticals Co. Ltd. to treat wound healing in patients with epidermolysis bullosa; and Grünenthal to develops and commercializes cell therapy for the treatment of chronic low back pain. The company was incorporated in 2004 and is headquartered in Melbourne, Australia.

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.

Taysha Gene Therapies, Inc., a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-120 for the treatment of giant axonal neuropathy; TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN7 disease; TSHA-118 for the treatment of CLN1 disease; TSHA-105 for the treatment of for SLC13A5 deficiency; TSHA-113 for the treatment of tauopathies; TSHA-106 for the treatment of angelman syndrome; TSHA-114 for the treatment of fragile X syndrome; and TSHA-101 for the treatment of GM2 gangliosidosis. Taysha Gene Therapies, Inc. has a strategic partnership with The University of Texas Southwestern Medical Center. Taysha Gene Therapies, Inc. was incorporated in 2019 and is headquartered in Dallas, Texas.