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Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs for serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration.
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness`s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases.
BioMarin is a global pharmaceutical company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We remain steadfast to our original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options and are usually ignored. Time is critical to patients with rare diseases, and we strive to quickly develop important therapies for them. The efficiency and speed of our research, development, manufacturing, and commercial efforts is at the heart of our ability to urgently deliver therapies. Our track record of developing and commercializing new treatments has been significantly faster than the industry average and is a remarkable accomplishment that is ingrained in our culture. The company`s product portfolio comprises five marketed products and multiple clinical and pre-clinical product candidates.
Nova Biologics. Inc. is a Carlsbad, CA-based company in the Healthcare, Pharmaceuticals, & Biotech sector.
Connect Biopharma (Nasdaq: CNTB) is a global clinical-stage biopharmaceutical company dedicated to improving the lives of patients with chronic inflammatory diseases through the development of therapies derived from T cell-driven research. With operations and expertise in China, the United States and Australia and clinical development activities in those geographies as well as Europe, Connect Biopharma is building a rich pipeline of internally designed, wholly owned small molecules and antibodies targeting several aspects of T cell biology. Our core expertise is in the use of functional cellular assays with T cells to screen and discover potent drug candidates against immune targets. Our two most advanced clinical-stage programs include highly differentiated product candidates against validated targets.