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Amyloid diseases are a diverse group of disorders, characterized by the deposition of amyloid protein fibrils in vital organs and tissues. There are approximately 30 different types of amyloidosis, each resulting from the misfolding and aggregation of a specific protein, and they are all severely debilitating, progressive, and often fatal.
MeMed is a host response, med-tech & bioconvergence company, offering innovative diagnostic host response tests that address complex clinical dilemmas & improve patient outcomes.
Miraculins is a pre-revenue biotechnology company that acquires, develops and commercializes new, non-invasive diagnostic and risk assessment tests to aid physicians in the earlier diagnosis of disease. The Company specializes in the development and manufacturing of medical devices and has been successfully accessing capital on the public markets as required to finance its operations to date. It is currently preparing for transition to a commercialization phase. Miraculins began as a research and development company in 2002, with a focus on biomarker discovery. In June of 2008, the company shifted its focus to diagnostic asset development where it began to in-license and acquire diagnostic and risk assessment technologies that had completed early stage research and addressed significant unmet medical needs in the U.S., Canada, and global markets.
Avidity Biosciences, Inc.`s mission is to profoundly improve people`s lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity`s proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity`s advancing and expanding pipeline has three programs in clinical development. AOC 1001 is designed to treat people with myotonic dystrophy type 1 (DM1) and is currently in Phase 1/2 development with the ongoing MARINA™ and MARINA-OLE™ trials. AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy (FSHD) and is currently in Phase 1/2 development with the FORTITUDE™ trial. AOC 1044 is designed for people with Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping and is currently in Phase 1/2 development with the EXPLORE44™ trial. AOC 1044 is the first of multiple AOCs the company is developing for DMD. Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in San Diego, CA.
AveXis, now a Novartis Company, is a clinical-stage gene therapy company relentlessly focused on bringing gene therapy out of the lab and into the clinical setting for patients and families devastated by rare and life-threatening neurological genetic d...