US2022325302A1PendingUtilityA1

Adeno-associated virus compositions for restoring pah gene function and methods of use thereof

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Assignee: HOMOLOGY MEDICINES INCPriority: Feb 1, 2018Filed: Mar 25, 2022Published: Oct 13, 2022
Est. expiryFeb 1, 2038(~11.6 yrs left)· nominal 20-yr term from priority
Inventors:Albert Barnes SeymourSeemin Seher AhmedJason Boke WrightSerena Nicole DolliveJames McswiggenJaime Michelle ProutDanielle Lauren Sookiasian
C12N 2750/14121C12N 2750/14143A61K 35/761A01K 2217/072C12N 7/00C12N 15/907A01K 2217/075A01K 2227/105A61K 48/005C12Y 114/16001C12N 2710/10033A01K 2267/0306C12N 2750/14152C12N 2710/10052A61P 13/00C12N 9/0071
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Claims

Abstract

Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

Claims

exact text as granted — not AI-modified
We claim: 
     
         1 . A polynucleotide comprising: a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130; or a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127. 
     
     
         2 . The polynucleotide of  claim 1 , comprising a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130. 
     
     
         3 . The polynucleotide of  claim 1 , comprising the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130. 
     
     
         4 . The polynucleotide of  claim 1 , comprising the nucleotide sequence of SEQ ID NO: 130. 
     
     
         5 . The polynucleotide of  claim 1 , comprising a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127. 
     
     
         6 . The polynucleotide of  claim 1 , comprising the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127. 
     
     
         7 . The polynucleotide of  claim 1 , comprising the nucleotide sequence of SEQ ID NO: 127. 
     
     
         8 . A vector comprising a polynucleotide that comprises:
 a) a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;   b) the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;   c) the nucleotide sequence of SEQ ID NO: 130;   d) a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127;   e) the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127; or   f) the nucleotide sequence of SEQ ID NO: 127.   
     
     
         9 . The vector of  claim 8 , which is an adeno-associated virus (AAV) vector. 
     
     
         10 . A cell comprising a polynucleotide that comprises:
 a) a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;   b) the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;   c) the nucleotide sequence of SEQ ID NO: 130;   d) a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127;   e) the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127; or   f) the nucleotide sequence of SEQ ID NO: 127.   
     
     
         11 . A cell comprising the vector of  claim 8 . 
     
     
         12 . A cell comprising the vector of  claim 9 . 
     
     
         13 . The cell of  claim 10 , which is a mammalian cell or an insect cell. 
     
     
         14 . The cell of  claim 11 , which is a mammalian cell or an insect cell. 
     
     
         15 . The cell of  claim 12 , which is a mammalian cell or an insect cell.

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