4D Molecular Therapeutics has been granted a patent for variant adeno-associated virus (AAV) capsid proteins that increase infectivity of muscle cells. The recombinant AAV virions can deliver nucleic acid sequences for treating muscle disorders. GlobalData’s report on 4D Molecular Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.
According to GlobalData’s company profile on 4D Molecular Therapeutics, Adeno-associated virus vectors was a key innovation area identified from patents. 4D Molecular Therapeutics's grant share as of January 2024 was 34%. Grant share is based on the ratio of number of grants to total number of patents.
Recombinant aav for muscle cell infectivity with heterologous peptide
A recently granted patent (Publication Number: US11840704B2) discloses a recombinant adeno-associated virus (rAAV) designed for gene therapy applications. The rAAV comprises a variant AAV capsid protein with a heterologous peptide insertion of 7 to 20 amino acids in the GH-loop of the capsid protein, along with a heterologous nucleic acid encoding a gene product. The patent specifies various embodiments of the rAAV, including specific amino acid sequences for the peptide insertion and the capsid protein, as well as the location of the insertion site within different AAV serotypes.
Furthermore, the patent details the use of the rAAV for delivering various gene products, such as proteins like alpha galactosidase A (GLA), frataxin (FXN), dystrophin (DMD), acid alpha glucosidase (GAA), and glycogen phosphorylase, muscle (PYGM). The gene product can also include small interfering RNA, antisense RNA, microRNA, or short hairpin RNA. The patent also covers pharmaceutical compositions comprising the rAAV and a pharmaceutically acceptable carrier, with recommended ranges of rAAV virions for effective therapy. Overall, the patent provides a comprehensive framework for utilizing the rAAV in gene therapy applications, offering a promising avenue for the treatment of various genetic disorders.
To know more about GlobalData’s detailed insights on 4D Molecular Therapeutics, buy the report here.
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