Avidity Biosciences. has been granted a patent for a method to treat facioscapulohumeral muscular dystrophy (FSHD) using a polynucleic acid conjugate. This conjugate targets the DUX4 gene, reducing its mRNA levels through RNA interference, thereby addressing muscular dystrophy in affected individuals. GlobalData’s report on Avidity Biosciences gives a 360-degree view of the company including its patenting strategy. Buy the report here.
According to GlobalData’s company profile on Avidity Biosciences, Nanoparticle drug conjugates was a key innovation area identified from patents. Avidity Biosciences's grant share as of July 2024 was 23%. Grant share is based on the ratio of number of grants to total number of patents.
Treatment of facioscapulohumeral muscular dystrophy using polynucleic acids
The patent US12071485B2 outlines a novel method for treating muscular dystrophy, specifically targeting Facioscapulohumeral muscular dystrophy (FSHD). The method involves administering a polynucleic acid conjugate that consists of an anti-transferrin receptor antibody or its antigen-binding fragment linked to a polynucleic acid molecule. This polynucleic acid is designed to hybridize with a specific target sequence of the DUX4 gene, which is implicated in the pathology of muscular dystrophy. The therapeutic approach aims to reduce the mRNA transcript levels of DUX4 through RNA interference, thereby mitigating the effects of muscular dystrophy in affected individuals.
The claims further detail the parameters of the treatment, including the degree of mRNA reduction, which can be at least 50% to 70% or more compared to untreated cells. Additionally, the method encompasses the potential reduction of expression of various marker genes associated with muscular dystrophy. The polynucleic acid molecule is specified to be between 21 to 30 nucleotides in length and may include modifications such as a 5'-terminal vinylphosphonate nucleotide. The conjugate can utilize various types of antibodies, including humanized or monoclonal forms, and may employ different linkers to connect the antibody to the polynucleic acid. The claims also specify the ratios of the polynucleic acid to the antibody, providing a comprehensive framework for the therapeutic application of this innovative treatment strategy.
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