View source for 20250213726. Therapeutic (ASKLEPIOS BIOPHARMACEUTICAL, .)

= THERAPEUTIC ADENO-ASSOCIATED VIRUS USING CODON OPTIMIZED NUCLEIC ACID ENCODING ALPHA-GLUCOSIDASE (GAA) FOR TREATING POMPE DISEASE, WITH SIGNAL PEPTIDE MODIFICATIONS =

'''Abstract''':
disclosed herein is a method for the treatment of pompe disease comprising administering a recombinant aav (raav) vector comprising a ravv genome comprising a heterologous nucleic acid encoding a gaa signal peptide or portion thereof, a heterologous signal peptide, and an acid alpha-glucosidase (gaa) polypeptide, or n-terminal truncation thereof, where the heterologous nucleic acid is operatively linked to a liver-specific promoter, where the nucleic acid encoding gaa polypeptide can be wild type nucleic acid sequence, or modified nucleic acid sequence, or a codon optimized nucleic acid sequence, and can optionally be modified to reduce or completely eliminate cg and cpg dinucleotides and, optionally eliminated alternative reading frames (arf) content.

'''Inventor(s)''': [[:Category:Anna TRETIAKOVA of Research Triangle Park NC US|Anna TRETIAKOVA]], [[:Category:Xavier AUGUELA MARTINEZ of Research Triangle Park NC US|Xavier AUGUELA MARTINEZ]], [[:Category:Lester SUAREZ of Research Triangle Park NC US|Lester SUAREZ]], [[:Category:Scott HAMMOND of Research Triangle Park NC US|Scott HAMMOND]]

'''CPC Classification''': [[:Category:CPC_A61K48/0058|A61K48/0058]] (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)


[[Category:Patent Applications]]
[[Category:ASKLEPIOS BIOPHARMACEUTICAL, INC.]]
[[Category:CPC_A61K48/0058]]
[[Category:Anna TRETIAKOVA of Research Triangle Park NC US]]
[[Category:Xavier AUGUELA MARTINEZ of Research Triangle Park NC US]]
[[Category:Lester SUAREZ of Research Triangle Park NC US]]
[[Category:Scott HAMMOND of Research Triangle Park NC US]]

[https://wikipatents.org/index.php?search=20250213726+rejection&title=Special%3ASearch&wprov=acrw1_-1 Search for rejections for patent application number 20250213726]


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