4D Molecular Therapeutics. has filed a patent for a method involving codon optimized sequences encoding human alpha-galactosidase A to treat disorders like Fabry disease. The claim includes delivering nucleic acid to kidney, brain, or lung cells using a recombinant adeno-associated virus with a variant capsid protein. 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 January 2024 was 34%. Grant share is based on the ratio of number of grants to total number of patents.

Gene therapy for fabry disease using optimized alpha-galactosidase a sequence

Source: United States Patent and Trademark Office (USPTO). Credit: 4D Molecular Therapeutics Inc

A patent application (Publication Number: US20240018498A1) discloses a method for delivering a heterologous nucleic acid to kidney, brain, and lung cells using a recombinant adeno-associated virus (rAAV). The method involves a variant AAV capsid protein with a heterologous peptide insertion of 7 to 20 amino acids in the GH-loop, along with a nucleotide sequence encoding a gene product linked to a promoter. The insertion peptide may have spacer amino acids at its terminus, and the variant capsid protein includes specific amino acid substitutions and sequence identities. The insertion site is defined within the capsid protein, and the promoter used can be constitutive or tissue-specific.

Furthermore, the method extends to delivering the nucleic acid to the kidney, brain, spinal cord, or lung of a primate, including humans, by administering the rAAV or a pharmaceutical composition containing it. The process involves similar components as mentioned earlier, with specific details on the insertion peptide, capsid protein characteristics, insertion site, and promoter type. The administration can be done through intravenous injection, targeting specific tissues in the primate. Overall, the patent application outlines a detailed method for efficient delivery of nucleic acids to specific cells and tissues using rAAV technology, with potential applications in gene therapy and related fields.

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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