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We are a rare disease therapeutics company leading with science to make life-changing therapeutics available to patients with significant unmet needs. We involve key thought leaders, physicians, patients, care partners, and advocacy groups in all of our clinical and regulatory development strategies. With a keen understanding that drug development often requires creative solutions, we have the insight and expertise to forge new pathways to success that others have missed. By following the data without bias, our transparent narratives and common-sense perspective have successfully overcome complex development challenges to make much-needed therapies available to patients. Nimble and dauntless, we push boundaries beyond what is thought to be possible and advance new therapies that have the potential to bring meaningful improvement to patients lives.
AES OPTICS is a Senatobia, MS-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Regional Clinical Research is a Endicott, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Sarepta Therapeutics is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.