Amicus Therapeutics. has filed a patent for improved gene therapy vectors and methods of use. The vectors aim to enhance the expression and cellular targeting of therapeutic proteins. The patent claim describes a gene therapy vector that includes a nucleic acid construct encoding a polypeptide with a therapeutic protein, a peptide that binds to the cation-independent mannose 6-phosphate receptor (CI-MPR) with high affinity, and a linker between the therapeutic protein and the CI-MPR binding peptide. GlobalData’s report on Amicus Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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Improved gene therapy vector for targeted protein expression
A recently filed patent (Publication Number: US20230233711A1) describes a gene therapy vector that can be used for treating genetic disorders. The gene therapy vector consists of a nucleic acid construct that encodes a polypeptide. The polypeptide includes a therapeutic protein, a peptide that binds to the cation-independent mannose 6-phosphate (M6P) receptor with high affinity, and a linker between the therapeutic protein and the peptide that binds to the M6P receptor.
The patent claims specify various aspects of the gene therapy vector. Claim 2 states that the peptide is a variant IGF2 (vIGF2) peptide. Claim 3 further describes the vIGF2 peptide as having an amino acid sequence that is at least 90% identical to a specific sequence (SEQ ID NO: 1) and having substitutions at certain positions. Claim 4 provides examples of substitutions that can be made in the vIGF2 peptide.
Claims 5 and 6 describe the vIGF2 peptide having at least two substitutions at certain positions, with Claim 6 providing examples of these substitutions. Claim 7 mentions an N-terminal deletion in the vIGF2 peptide. Claim 8 states that the vIGF2 peptide has decreased affinity for insulin receptor and IGF1R compared to the native IGF2 peptide.
The patent claims also cover the functionality of the gene therapy vector. Claims 9 and 10 state that the vIGF2 peptide is capable of facilitating the uptake of the therapeutic protein into a cell or a lysosome, respectively. Claim 11 specifies that the therapeutic protein can replace a defective or deficient protein associated with a genetic disorder. Claims 12 to 15 provide examples of genetic disorders that can be treated using the gene therapy vector, including lysosomal storage disorders, Pompe disease, and CLN1 disease.
Claims 16 to 19 describe the therapeutic protein as a soluble lysosomal enzyme or an enzymatically active fragment thereof, with examples of specific enzymes provided. Claims 20 and 21 mention additional elements in the nucleic acid construct, such as a translation initiation sequence and a signal peptide that can increase the secretion of the therapeutic protein.
Finally, claims 22 to 30 specify that the vIGF2 peptide can be located at the N-terminus or C-terminus of the polypeptide, the linker peptide can have specific sequences, and the gene therapy vector can be a virus vector selected from various types.
Overall, the patent claims describe a gene therapy vector that incorporates a therapeutic protein, a peptide that binds to the M6P receptor, and a linker peptide. The vector has potential applications in treating various genetic disorders, particularly lysosomal storage disorders, by facilitating the uptake of the therapeutic protein into cells or lysosomes.
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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
