US2019008931A1PendingUtilityA1

Aadc polynucleotides for the treatment of parkinson's disease

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Assignee: VOYAGER THERAPEUTICS INCPriority: Nov 5, 2014Filed: Sep 20, 2018Published: Jan 10, 2019
Est. expiryNov 5, 2034(~8.3 yrs left)· nominal 20-yr term from priority
Inventors:Robert KotinAdrian Philip KellsBernard Ravina
C12N 15/86C12N 2830/42C12N 9/88A61K 38/51C12Y 401/01028A61K 48/005A61P 25/14C12N 2830/50C12N 2750/14121C12N 2750/14143C12N 7/00A61K 35/76C12N 2800/22
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Claims

Abstract

The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.

Claims

exact text as granted — not AI-modified
We claim: 
     
         1 . An AAV vector genome comprising, in order:
 a) a 5′ inverted terminal repeat (ITR), wherein said 5′ ITR is 141 nucleotides in length;   b) an AADC sequence region, said AADC sequence region comprising a nucleotide sequence encoding SEQ ID NO: 1,   c) a 3′ ITR, wherein said 3′ ITR is 141 nucleotides in length.   
     
     
         2 . The AAV vector genome of  claim 1 , wherein the 5′ ITR and the 3′ ITR are derived from AAV2. 
     
     
         3 . The AAV vector genome of  claim 2 , wherein the AAV vector genome comprises a cytomegalovirus (CMV) sequence region derived from a CMV gene; wherein the CMV sequence region comprises an enhancer region and promoter region. 
     
     
         4 . The AAV vector genome of  claim 3 , wherein the CMV sequence region is 507 nucleotides in length. 
     
     
         5 . The AAV vector genome of  claim 2 , wherein the AAV vector genome comprises an immediate early 1 (IE1) sequence region derived from an IE1 gene. 
     
     
         6 . The AAV vector genome of  claim 5 , wherein the IE1 sequence region comprises a nucleotide sequence from IE1 exon1. 
     
     
         7 . The AAV vector genome of  claim 6 , wherein the IE1 sequence region comprises a nucleotide sequence from IE1 intron 1 or a fragment thereof. 
     
     
         8 . The AAV vector genome of  claim 7 , wherein the IE1 sequence region is 166 nucleotides in length. 
     
     
         9 . The AAV vector genome of  claim 2 , wherein the AAV vector genome comprises a human beta globin (BB) sequence region derived from a HB gene. 
     
     
         10 . The AAV vector genome of  claim 9 , wherein the HB sequence region comprises a nucleotide sequence from HB intron 2. 
     
     
         11 . The AAV vector genome of  claim 10 , wherein the HB sequence region comprises a nucleotide sequence from HB exon 3. 
     
     
         12 . The AAV vector genome of  claim 11 , wherein the HB sequence region is 400 nucleotides in length. 
     
     
         13 . The AAV vector genome of  claim 2 , wherein the AAV vector genome comprises a poly(A) signal sequence region derived from the human growth hormone (hGH) gene. 
     
     
         14 . The AAV vector genome of  claim 13 , wherein the poly(A) signal sequence region is 477 nucleotides in length. 
     
     
         15 . The AAV vector genome of  claim 1 , wherein the AAV vector genome comprises a sequence selected from the group consisting of SEQ ID NO: 10-13, 15, 17-20 and 22 and variants having at least 95% identity thereto. 
     
     
         16 . The AAV vector genome of  claim 2 , wherein, the AADC region comprises a codon optimized nucleotide sequence encoding SEQ ID NO: 1. 
     
     
         17 . The AAV vector genome of  claim 16 , wherein the AAV vector genome comprises a sequence selected from the group consisting of SEQ ID NO: 7 and 12 or variants having at least 95% identity thereto. 
     
     
         18 . A recombinant AAV virus comprising the AAV vector genome of  claim 1 . 
     
     
         19 . The recombinant AAV virus of  claim 18 , comprising a capsid serotype selected from the group consisting of AAV2, AAV1, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV9.47, AAV9(hu14), AAV10, AAV11, AAV12, AAVrh8, AAVrh10, AAV-DJ, and AAV-DJ8. 
     
     
         20 . The recombinant AAV virus of  claim 19 , wherein the capsid serotype is AAV2. 
     
     
         21 . A pharmaceutical composition comprising the recombinant AAV virus of  claim 20 . 
     
     
         22 . The pharmaceutical composition of  claim 21 , wherein at least 70% of the recombinant AAV vectors contain an AAV vector genome.

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