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Find Drugs for Genetic Disease in Preclinical Development
ReCode is developing an inhaled ARCT-032 (mRNA therapy), designed to provide a correct copy of the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA to lung cells to make a functional CFTR protein.
SEL-018 (xork) is being studied as a potential next generation immunoglobulin G (IgG) protease that will be developed by Astellas for use with AT845, an investigational, adeno-associated virus (AAV)-based treatment for Late-Onset Pompe disease (LOPD) in adults.
ENTR-601-45 is a Endosomal Escape Vehicle-conjugated phosphorodiamidate morpholino oligomer, has the potential to restore the mRNA reading frame and allow for the translation of dystrophin protein, it Duchenne muscular dystrophy patients.
Company plans to nominate additional therapeutic targets to its proprietary genetic medicines pipeline, fueled by its automated discovery engine and versatile gene editing toolbox, to expand into additional preclinical proof-of-concept studies.
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