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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.
Hecker and Associates is a Arlington, VA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Founded in 2011, PANTHERx Rare is a specialty pharmacy solely focused on rare and orphan diseases, distributing several orphan products and providing access and support services to those needing them most. Known as The Rare Disease Pharmacy®, PANTHERx transforms lives by delivering therapeutic breakthroughs, clinical excellence, personalized support, and a range of access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans. Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is the seven-time winner of the prestigious MMIT Patient Choice Award, including the 2024 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories. #ReinventingSpecialty #RevolutionizingPharmacy #RedefiningCare #DefiningRarePharmacy #TheRareDiseasePharmacy
Nemus Bioscience Inc. is a biopharmaceutical company focused on discovering, developing and commercializing new chemical entities from a class of chemically diverse compounds called cannabinoids, designed to alleviate a variety of diseases and symptoms by selectively targeting CB1 and CB2 receptors. NEMUS Bioscience Inc. is developing a novel and proprietary class of product candidates that are designed to target the body`s nervous and immune systems without inducing many of the undesirable side effects typically associated with currently available therapeutics. NEMUS Bioscience Inc., a Nevada corporation is listed on the OTC Bulletin Board and trading under the symbol NMUS. To become a leading developer of cannabis-based therapeutics to address unmet medical needs on a global basis.
SciFluor Life Sciences is a drug discovery company applying expertise in fluorine chemistry to create a portfolio of differentiated best-in-class therapeutics. The company is creating a portfolio of new chemical entities (NCEs) directed towards precedented biological targets. SciFluor strategically incorporates fluorine or fluorine-containing groups to design drugs with improved pharmacological profiles that provide important benefits over existing therapies such as improved safety, efficacy, more convenient dosing and better patient compliance. This capital-efficient and de-risked drug discovery approach has resulted in the generation of a proprietary pipeline of novel and differentiated small molecule drugs across a range of therapeutic categories and disease areas including retinal disease, CNS disorders, fungal and bacterial infections and inflammatory disease.