Adverum Biotechnologies
NASDAQ:ADVMAdverum 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.
AVITA MED LTD/S
OTCMKTS:AVMXYAvita Medical Limited operates as a regenerative medicine company in the Asia Pacific, Europe, the Middle East, Africa, and the Americas. It offers regenerative products for the treatment of burns, chronic wounds, and aesthetics indications. The company's patented and proprietary collection and application technology provides treatment solutions derived from a patient's own skin. Its lead product, RECELL System, is used in the treatment of various skin defects, such as burns and plastic reconstructive procedures. The company also offers ReGenerCell for chronic wounds and ReNovaCell for the restoration of pigmentation. Avita Medical Limited is based in Valencia, California.
Precision BioSciences
NASDAQ:DTILPrecision BioSciences, Inc., a clinical stage gene editing company, develops ex vivo allogeneic chimeric antigen receptor (CAR) T immunotherapies and in vivo therapies for genetic and infectious diseases in the United States. The company offers ARCUS, a genome editing platform to cure genetic disorders. It also provides Ex vivo Allogeneic CAR T Immunotherapy, a form of immunotherapy in which T cell, a specific type of immune cell is genetically engineered to recognize and kill cancer cells; PBCAR0191, an azercabtagene zapreleucel, for the treatment of acute lymphoblastic leukemia and B-cell precursor acute lymphoblastic leukemia; PBCAR19B, an anti-CD19 CAR T candidate built on the stealth cell platform utilizing a single-step gene edit to minimize the risk of chromosome abnormalities; and PBCAR269A, an investigational allogeneic CAR T cell candidate targeting B-cell maturation antigen for relapsed or refractory (R/R) multiple myeloma in combination with nirogacestat, a gamma secretase inhibitor. The company has development and commercial license agreements with Les Laboratoires Servier to develop allogeneic CAR T cell therapies for antigen targets, hematological cancer targets beyond CD19, and solid tumor targets; Tiziana Life Sciences to evaluate foralumab, a fully human anti-CD3 monoclonal antibody as a lymphodepleting agent for the potential treatment of cancers; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.
Mesoblast
NASDAQ:MESOMesoblast 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
NASDAQ:RUBYRubius 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.