Granted Patent B2

US 12,319,929 · App. 17/055,378 · Granted Jun 3, 2025

Inventors: Bernard Ravina (Newton, MA); Adrian Philip Kells (Upper Arlington, OH)

Assignee: Voyager Therapeutics, Inc.

C12N15/86A61K38/51C12N2750/14143C12Y401/01028

Abstract

Claims (19)

1. A method of administering a pharmaceutical composition to a subject, the method comprising: administering to the subject the pharmaceutical composition, said pharmaceutical composition comprising an adeno-associated virus (AAV) which comprises an AAV2 capsid and a vector genome, wherein the vector genome comprises a nucleotide sequence which has at least 99% nucleotide sequence identity to SEQ ID NO: 979.

2. The method of claim 1 , wherein the nucleotide sequence which has at least 99% nucleotide sequence identity to SEQ ID NO: 979 comprises SEQ ID NO: 980.

3. The method of claim 1 , wherein the nucleotide sequence which has at least 99% nucleotide sequence identity to SEQ ID NO: 979 comprises SEQ ID NO: 991.

4. The method of claim 1 , wherein the pharmaceutical composition is administered to the subject by posterior surgical infusion into at least one putamen of the subject; and wherein the average total putaminal coverage from the posterior administration is at least 50%.

5. The method of claim 4 , wherein the posterior surgical infusion of the pharmaceutical composition is bilateral to both the right putamen and the left putamen of the subject during a single procedure.

6. The method of claim 5 , wherein the average total putaminal coverage of the pharmaceutical composition after the posterior surgical infusion is 50-65%.

7. The method of claim 4 , wherein surgical time for the posterior surgical infusion is 7-10 hours.

8. The method of claim 4 , wherein infusion time for the posterior surgical infusion is 2.5-4.5 hours.

9. The method of claim 1 , wherein the pharmaceutical composition is administered to the subject by transfrontal surgical infusion into at least one putamen of the subject; and wherein the average total putaminal coverage from the transfrontal administration is 30-50%.

10. The method of claim 9 , wherein the transfrontal surgical infusion of the pharmaceutical composition is bilateral to both the right putamen and the left putamen of the subject during a single procedure.

11. The method of claim 10 , wherein the average total putaminal coverage from the transfrontal surgical infusion is 40-50%.

12. The method of claim 1 , wherein the pharmaceutical composition comprises an AAV concentration of between 2.0×10 12 vg/ml and 3.0×10 12 vg/ml.

13. The method of claim 1 , wherein the pharmaceutical composition comprises an AAV concentration of about 2.6×10 12 vg/ml.

14. The method of claim 1 , wherein the pharmaceutical composition is administered at a volume of up to 1800 μL per putamen.

15. The method of claim 1 , wherein the pharmaceutical composition is administered at a total viral dosage of 2.0×10 12 vg to 9.4×10 12 vg.

16. The method of claim 1 , wherein the pharmaceutical composition is a formulation comprising sodium chloride, sodium phosphate and pluronic acid F-68, and wherein the formulation has a pH between 7.0-7.5.

17. The method of claim 16 , wherein the formulation comprises 150-200 mM sodium chloride, 8-12 mM sodium phosphate and 0.001-0.01% w/v pluronic acid F-68, and wherein the formulation has a pH between 7.2-7.4.

18. The method of claim 16 , wherein the formulation comprises 180 mM sodium chloride, 10 mM sodium phosphate and 0.001% w/v pluronic acid F-68, and wherein the formulation has a pH of 7.3.

19. A method of treating Parkinson's Disease in a subject, the method comprising: administering to the subject a pharmaceutical composition comprising an adeno-associated virus (AAV) which comprises an AAV2 capsid and a vector genome, wherein the vector genome comprises a nucleotide sequence which has at least 99% nucleotide sequence identity to SEQ ID NO: 979.

Assignments (1)

Continuity (12)

References Cited (400)