4D Molecular Therapeutics. has filed a patent for variant adeno-associated virus (AAV) capsid proteins that increase infectivity of muscle cells compared to unmodified AAV capsid proteins. The patent also covers recombinant AAV virions and pharmaceutical compositions containing the variant AAV capsid proteins, as well as methods for making and using them in the treatment of muscle disorders and diseases. The claim specifically describes a recombinant AAV with a variant capsid protein containing a heterologous peptide insertion and a heterologous nucleic acid encoding a gene product. GlobalData’s report on 4D Molecular Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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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 September 2023 was 34%. Grant share is based on the ratio of number of grants to total number of patents.
The patent is filed for a modified aav virus for muscle cell infectivity
A recently filed patent (Publication Number: US20230279435A1) describes a recombinant adeno-associated virus (rAAV) that includes a variant AAV capsid protein with a heterologous peptide insertion and a heterologous nucleic acid encoding a gene product. The peptide insertion has a length of 7 to 20 amino acids and includes the amino acid sequence NKIQRTD (SEQ ID NO: 13). The patent also mentions that the insertion peptide can have 1 to 3 spacer amino acids (Y1-Y3) at the amino and/or carboxyl terminus of the NKIQRTD sequence.
The patent further specifies that the variant capsid protein of the rAAV includes a V708I amino acid substitution relative to VP1 of AAV2 (SEQ ID NO:2). The variant capsid protein is at least 90% identical to the entire length of the amino acid sequence set forth as SEQ ID NO:43, and in some cases, it is at least 95% identical.
The patent provides information about the specific insertion sites for the peptide in different AAV serotypes. These insertion sites are located within specific amino acid ranges in the capsid proteins of various AAV serotypes, including AAV1, AAV3A, AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, and AAV10.
The rAAV described in the patent can be used to deliver various gene products, including proteins, small interfering RNA, antisense RNA, microRNA, and short hairpin RNA. Specific examples of gene products mentioned in the patent include alpha galactosidase A (GLA), frataxin (FXN), dystrophin (DMD) or a functional fragment thereof, acid alpha glucosidase (GAA), and glycogen phosphorylase, muscle (PYGM).
The patent also covers pharmaceutical compositions comprising the rAAV and a pharmaceutically acceptable carrier. The compositions can contain a range of rAAV virions, from 1×1011 to 1×1015.
In summary, the patent describes a recombinant adeno-associated virus (rAAV) with a variant capsid protein and a heterologous nucleic acid encoding a gene product. The rAAV includes a specific peptide insertion and can be used to deliver various gene products. The patent also covers pharmaceutical compositions containing the rAAV.
To know more about GlobalData’s detailed insights on 4D Molecular Therapeutics, buy the report here.
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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies
