US2019008932A1PendingUtilityA1

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 15/86A61K 48/005A61K 38/51C12N 2800/22A61P 25/14C12N 7/00C12Y 401/01028C12N 9/88C12N 2830/42C12N 2750/14143C12N 2830/50C12N 2750/14121A61K 35/76
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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 130 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 119 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 5′ ITR consists of a nucleotide sequence consisting of nucleotides 1 to 130 of SEQ ID NO 6. 
     
     
         4 . The AAV vector genome of  claim 2 , wherein the 5′ ITR consists of a nucleotide sequence which has at least 99% sequence identity with nucleotides 1 to 130 of SEQ ID NO 6. 
     
     
         5 . The AAV vector genome of  claim 3 , wherein the 3′ ITR consists of a nucleotide sequence consisting of nucleotides 3417 to 3535 of SEQ ID NO 6. 
     
     
         6 . The AAV vector genome of  claim 4 , wherein the 3′ ITR consists of a nucleotide sequence which has at least 99% sequence identity with nucleotides 3417 to 3535 of SEQ ID NO 6. 
     
     
         7 . The AAV vector genome of  claim 6 , 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. 
     
     
         8 . The AAV vector genome of  claim 7 , wherein the CMV sequence region comprises a nucleotide sequence consisting of nucleotides 263 to 769 of SEQ ID NO 6. 
     
     
         9 . The AAV vector genome of  claim 7 , wherein the CMV sequence region comprises a nucleotide sequence which has at least 98% sequence identity with nucleotides 263 to 769 of SEQ ID NO 6. 
     
     
         10 . The AAV vector genome of  claim 8 , wherein the CMV enhancer region comprises a nucleotide sequence consisting of nucleotides 263 to 566 of SEQ ID NO 6. 
     
     
         11 . The AAV vector genome of  claim 9 , wherein the CMV enhancer region comprises a nucleotide sequence which has at least 98% sequence identity with nucleotides 263 to 566 of SEQ ID NO 6. 
     
     
         12 . The AAV vector genome of  claim 10 , wherein the CMV promoter region comprises a nucleotide sequence consisting of nucleotides 567 to 769 of SEQ ID NO 6. 
     
     
         13 . The AAV vector genome of  claim 11 , wherein the CMV promoter region comprises a nucleotide sequence which has at least 99% sequence identity with nucleotides 567 to 769 of SEQ ID NO 6. 
     
     
         14 . The AAV vector genome of  claim 13 , wherein the CMV sequence region is 507 nucleotides in length. 
     
     
         15 . The AAV vector genome of  claim 13 , wherein the AAV vector genome comprises an immediate early 1 (IE1) sequence region derived from an IE1 gene; wherein the IE1 sequence region comprises a nucleotide sequence from IE1 exon1, and a nucleotide sequence from IE1 intron 1 or a fragment thereof. 
     
     
         16 . The AAV vector genome of  claim 15 , wherein the IE1 sequence region comprises a nucleotide sequence consisting of nucleotides 784 to 949 of SEQ ID NO 6. 
     
     
         17 . The AAV vector genome of  claim 15 , wherein the IE1 sequence region comprises a nucleotide sequence which has at least 99% sequence identity with nucleotides 784 to 949 of SEQ ID NO 6. 
     
     
         18 . The AAV vector genome of  claim 17 , wherein the IE1 sequence region is 166 nucleotides in length. 
     
     
         19 . The AAV vector genome of  claim 17 , wherein the AAV vector genome comprises a human beta globin (BB) sequence region derived from a HB gene; wherein the the HB sequence region comprises a nucleotide sequence from HB intron 2 and a nucleotide sequence from HB exon 3. 
     
     
         20 . The AAV vector genome of  claim 19 , wherein the HB sequence region comprises a nucleotide sequence consisting of nucleotides 950 to 1349 of SEQ ID NO 6. 
     
     
         21 . The AAV vector genome of  claim 19 , wherein the HB sequence region comprises a nucleotide sequence which has at least 99% sequence identity with nucleotides 950 to 1349 of SEQ ID NO 6. 
     
     
         22 . The AAV vector genome of  claim 21 , wherein the HB sequence region is 400 nucleotides in length. 
     
     
         23 . The AAV vector genome of  claim 21 , wherein the AAV vector genome comprises a poly(A) signal sequence region derived from the human growth hormone (hGH) gene. 
     
     
         24 . The AAV vector genome of  claim 23 , wherein the poly(A) signal sequence region comprises a nucleotide sequence consisting of nucleotides 2841 to 3317 of SEQ ID NO 6. 
     
     
         25 . The AAV vector genome of  claim 23 , wherein the poly(A) signal sequence region comprises a nucleotide sequence which has at least 98% sequence identity with nucleotides 2841 to 3317 of SEQ ID NO 6. 
     
     
         26 . The AAV vector genome of  claim 25 , wherein the poly(A) signal sequence region is 477 nucleotides in length. 
     
     
         27 . The AAV vector genome of  claim 1 , wherein the AAV vector genome comprises SEQ ID NO 6, or variants having at least 98% identity thereto. 
     
     
         28 . A recombinant AAV virus comprising the AAV vector genome of  claim 1 . 
     
     
         29 . The recombinant AAV virus of  claim 28 , comprising a capsid serotype selected from the group consisting of AAV2, AAV1, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV9.47, AAV9(hul4), AAV10, AAV11, AAV12, AAVrh8, AAVrh10, AAV-DJ, and AAV-DJ8. 
     
     
         30 . The recombinant AAV virus of  claim 29 , wherein the capsid serotype is AAV2.

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