US2019060425A1PendingUtilityA1

Aadc polynucleotides for the treatment of parkinson's disease

55
Assignee: VOYAGER THERAPEUTICS INCPriority: Jun 15, 2017Filed: Nov 8, 2018Published: Feb 28, 2019
Est. expiryJun 15, 2037(~10.9 yrs left)· nominal 20-yr term from priority
Inventors:Maria ScheelBernard Ravina
A61K 48/0075C12N 15/86C12N 2750/14143A61K 48/005A61P 25/16C12Y 401/01028C12N 15/62A61K 35/761A61K 48/0066C12N 9/88A61K 38/51C12N 15/85C12N 15/8645C12N 15/11A61P 25/20
55
PatentIndex Score
0
Cited by
0
References
0
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 aromatic L-amino acid decarboxylase (AADC) polynucleotide comprising an AADC sequence region which has at least 95% identity to SEQ ID NO: 979. 
     
     
         2 . The AADC polynucleotide of  claim 1 , wherein the AADC sequence region comprises a promoter region, an enhancer region, a multiple cloning site (MCS) region and a polyadenylation (poly(A)) signal region. 
     
     
         3 . The AADC polynucleotide of  claim 2 , wherein the AADC sequence region comprises at least one 5′ inverted terminal repeat (ITR) region and one 3′ ITR region. 
     
     
         4 . The AADC polynucleotide of  claim 3 , wherein one or more of the 5′ ITRs are located 5′ to the MCS region and one or more of the 3′ ITRs are located 3′ to the poly(A) signal. 
     
     
         5 . The AADC polynucleotide of  claim 4 , wherein the AADC sequence region comprises a first exon region, a first intron region, a second intron region and a second exon region. 
     
     
         6 . The AADC polynucleotide of  claim 5 , wherein the enhancer region and the promoter region are derived from CMV. 
     
     
         7 . The AADC polynucleotide of  claim 6 , wherein the first exon region is immediate-early 1 (ie1) exon 1 or fragments thereof, the first intron region is ie1 intron 1 or fragments thereof, the second intron region is human beta-globin (hBglobin) intron 2 or fragments thereof and the second exon region is hBglobin exon 3 or fragments thereof. 
     
     
         8 . The AADC polynucleotide of  claim 7 , wherein the poly(A) signal is derived from human growth hormone. 
     
     
         9 . The AADC polynucleotide of  claim 8 , wherein the AADC sequence region consists of 3520-3530 nucleotides from the 5′ end of the 5′ ITR to the 3′ end of the 3′ ITR. 
     
     
         10 . The AADC polynucleotide of  claim 8 , wherein the AADC sequence region consists of 3526 nucleotides from the 5′ end of the 5′ ITR to the 3′ end of the 3′ ITR. 
     
     
         11 . A recombinant adeno-associated virus (rAAV) comprising the AADC polynucleotide of  claim 8 . 
     
     
         12 . The rAAV virus of  claim 11 , wherein the capsid serotype of the rAAV is AAV2. 
     
     
         13 . The AADC polynucleotide of  claim 3 , wherein the AADC sequence region has at least 99% identity to SEQ ID NO: 979. 
     
     
         14 . The AADC polynucleotide of  claim 13 , wherein the AADC sequence region consists of 3526 nucleotides from the 5′ end of the 5′ ITR to the 3′ end of the 3′ ITR. 
     
     
         15 . A recombinant adeno-associated virus (rAAV) comprising the AADC polynucleotide of  claim 13 . 
     
     
         16 . The rAAV virus of  claim 15 , wherein the capsid serotype of the rAAV is AAV2. 
     
     
         17 . A pharmaceutical composition comprising an adeno-associated virus (AAV) particle, wherein the AAV particle comprises an AAV capsid and a vector genome, and wherein the vector genome comprises at least one AADC sequence region with at least 95% identity to SEQ ID NO: 979. 
     
     
         18 . The pharmaceutical composition of  claim 17 , wherein the AADC sequence region has at least 99% identity SEQ ID NO: 979. 
     
     
         19 . The pharmaceutical composition of  claim 18 , wherein the AAV capsid serotype is AAV2. 
     
     
         20 . The pharmaceutical composition of  claim 19 , wherein at least 70% of the AAV particles contain a vector genome.

Join the waitlist — get patent alerts

Track US2019060425A1 — get alerts on status changes and closely related new filings.

We store only your email — no account needed. See our privacy policy.