AADC polynucleotides for the treatment of Parkinson's disease

Patent metadata

Field	Value
Publication number	US-11759506-B2
Application number	US-201916523567-A
Country	US
Kind code	B2
Filing date	Jul 26, 2019
Priority date	Jun 15, 2017
Publication date	Sep 19, 2023
Grant date	Sep 19, 2023

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.

First claim

The invention claimed is:

1. A polynucleotide comprising a sequence at least 99% identical to SEQ ID NO: 979.

2. An adeno-associated virus (AAV) particle comprising the polynucleotide of claim 1 and an AAV2 capsid.

3. A pharmaceutical composition comprising one or more of the AAV particles of claim 2 and one or more pharmaceutically acceptable excipients.

4. A pharmaceutical composition comprising adeno-associated virus (AAV) particles, wherein at least 70% of the AAV particles contain a polynucleotide comprising a sequence at least 99% identical to SEQ ID NO: 979.

5. The pharmaceutical composition of claim 4 , wherein at least 90% of the AAV particles contain a polynucleotide comprising SEQ ID NO: 979.

6. A method of treating a symptom of Parkinson's Disease (PD) in a patient in need of treatment, the method comprising contacting the patient with a pharmaceutical composition comprising an AAV2 particle comprising a polynucleotide comprising a sequence at least 99% identical to SEQ ID NO: 979 at the putamen or substantia nigra (SN) of the patient, wherein the polynucleotide is expressed for a sufficient amount of time and amount to alleviate at least one symptoms of PD in the patient.

7. A method of treating one or more symptoms of Parkinson's Disease (PD) in a patient in need of treatment, the method comprising contacting the patient with a pharmaceutical composition comprising AAV2 particles at the putamen or substantia nigra (SN) of the patient, wherein at least 70% of said AAV2 particles comprises a polynucleotide comprising a sequence at least 99% identical to SEQ ID NO: 979, wherein the polynucleotide is expressed for a sufficient amount of time and amount to alleviate at least one symptoms of PD in the patient.

8. A method of producing an AAV particle of claim 2 , comprising transfecting a cell with a construct which comprises a polynucleotide comprising a sequence at least 99% identical to SEQ ID NO: 979 and a construct which comprises a polynucleotide encoding rep and cap genes; and harvesting the AAV particle from the cell.

9. A pharmaceutical composition comprising AAV particles, wherein at least 70% of the AAV particles contain a polynucleotide consisting of a sequence at least 99% identical to SEQ ID NO: 979.

10. The pharmaceutical composition of claim 9 , wherein at least 90% of the AAV particles contain a polynucleotide consisting of SEQ ID NO: 979.

11. The method of claim 7 , wherein the polynucleotide consists of a sequence at least 99% identical to SEQ ID NO: 979.

12. A method of producing an AAV particle of claim 2 , comprising transfecting a cell with a construct which comprises a polynucleotide consisting of a sequence at least 99% identical to SEQ ID NO: 979 and a construct which comprises a polynucleotide encoding rep and cap genes; and harvesting the AAV particle from the cell.

13. The polynucleotide of claim 1 , wherein the polynucleotide comprises SEQ ID NO: 979.

14. A pharmaceutical composition of claim 4 , wherein the polynucleotide comprises SEQ ID NO: 979.

15. The method of claim 8 , wherein the polynucleotide comprises SEQ ID NO: 979.

16. The pharmaceutical composition of claim 9 , wherein the polynucleotide comprises SEQ ID NO: 979.

17. The method of claim 12 , wherein the polynucleotide comprises SEQ ID NO: 979.

18. The method of claim 6 , wherein the polynucleotide consists of a sequence at least 99% identical to SEQ ID NO: 979.

Assignees

• Voyager Therapeutics Inc

Inventors

• Scheel Maria
• Ravina Bernard

Classifications

• A61K38/51 Primary

  Lyases (4) · CPC title

• C12N9/88 Primary

  Lyases (4.) · CPC title

• C12N15/86

  Viral vectors · CPC title

• A61K48/005

  characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered · CPC title

• A61K48/0075

  characterised by an aspect of the delivery route, e.g. oral, subcutaneous · CPC title