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Avalanche is a clinical-stage biotechnology company focused on discovering and developing novel gene therapies to transform the lives of patients with sight-threatening ophthalmic diseases. Using a next generation gene therapy platform, the Ocular BioFactory™, the Company is developing products designed to provide long-term benefit or a functional cure by inducing a sustained expression of a therapeutic protein with a one-time administration in the eye.
Prothena Corporation plc (Nasdaq: PRTA) Prothena is a global biotechnology company seeking to fundamentally alter the course of progressive diseases. We are a late-stage clinical company focused on the discovery, development, and commercialization of novel protein immunotherapies for the treatment of diseases that involve protein misfolding and inflammatory cell adhesion disorders. Our team has a recognized track record both for profound scientific discoveries and for the development of therapeutics that have become leading commercial products in their respective therapeutic categories. Prothena`s pipeline focuses on therapeutic monoclonal antibodies directed specifically to disease-causing proteins and our antibody-based product candidates target a number of potential indications, including AL amyloidosis (NEOD001), Parkinson’s disease and other related synucleinopathies (PRX002), ATTR amyloidosis (PRX004), and novel cell adhesion targets involved in psoriasis, psoriatic arthritis and other inflammatory diseases (PRX003). Prior to December 2012, Prothena’s business operated as part of Elan Corporation, plc. Prothena’s business consists of a substantial portion of Elan’s former drug discovery business platform. After the separation from Elan and the related distribution of our ordinary shares to Elan’s stockholders, our ordinary shares began trading on The Nasdaq Global Market under the symbol “PRTA” on December 21, 2012.
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.
BioAgilytix is a leading bioanalytical contract research organization (CRO) that supports the development of novel therapeutic biologics and biosimilars. Located in Research Triangle Park, North Carolina, BioAgilytix specializes in large-molecule bioanalysis providing critical insight into the safety, efficacy, and mechanism of action of drug candidates throughout the clinical trial process. Specifically, BioAgilytix offers assay development, validation, and sample analysis services for immunoassays and cell-based assays under non-GLP, spirit-of-GLP, and GLP, as well as product release testing under GMP. BioAgilytix is an engaged and energetic partner committed to delivering exceptional value and veteran expertise through a collaborative process that consistently results in successful projects.
Gyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness. The company was acquired by Novartis, a leading global medicines company, in February 2022.