IP Library › Granted Patent US 12,336,986
Granted Patent B2
US 12,336,986 · App. 17/862,668 · Granted Jun 24, 2025

Treatment and prevention of ocular neurodegenerative disorder

Inventors: Simon W. M. John (Bar Harbor, ME); Peter Alexander Williams (Bar Harbor, ME)
Assignee: The Jackson Laboratory
A61K31/455A61K31/19A61K31/4745A61K31/706A61K48/00A61K48/005A61K48/0075A61P27/06C12Y207/07001A61K38/00C12N2750/14143
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Quick Facts
Patent No.
US 12,336,986
App. No.
17/862,668
Granted
Jun 24, 2025
Kind
B2
Abstract

The invention relates to the use of a pharmaceutical composition containing nicotinamide (NAM) and/or pyruvate as a neuroprotective medicament or gene therapy in the treatment of neurodegenerative disorders, in particular axon degeneration of neuronal tissue in ocular-related neurodegeneration diseases including glaucoma.

Claims (19)

1. A method of reducing axonal degeneration of a retinal ganglion cell and treating glaucoma in a subject in need thereof, comprising the step of orally administering to the subject a therapeutically effective amount of nicotinamide (NAM) and pyruvate wherein the subject is a mammal and administered between 1 and 5 g/day of NAM and between 1 and 5 g/day of pyruvate, wherein axonal degeneration in a retinal ganglion cell is reduced.

2. The method of claim 1 , wherein the subject is also administered one or more compounds selected from the group consisting of nicotinamide mononucleotide (NMN), pyrroloquinoline quinone (PQQ), nicotinamide adenine dinucleotide (NAD) and nicotinamide ribose (NR).

3. The method of claim 1 , wherein the NAM is present in a therapeutically effective amount to reduce intraocular pressure.

4. The method of claim 1 , wherein the NAM and pyruvate are present in therapeutically effective amounts to reduce neurodegeneration in a retinal ganglion cell or to reduce intraocular pressure.

5. The method of claim 1 , wherein the subject is a human subject.

6. The method of claim 1 , the method further comprises the step of administering a gene composition, wherein said gene composition comprises a polynucleotide encoding NMNAT1.

7. The method of claim 6 , wherein the polynucleotide is in a viral vector.

8. The method of claim 7 , wherein the viral vector is an adeno-associated virus (AAV) vector, an adenoviral vector, a lentiviral vector, or a retroviral vector.

9. The method of claim 7 , wherein the viral vector is an AAV vector.

10. The method of claim 7 , wherein the viral vector is a lentiviral vector.

11. The method of claim 6 , wherein the gene composition is administered intravitreally or intraocularly.

12. The method of claim 6 , wherein the gene composition is administered intravitreally.

13. The method of claim 1 , further comprising administering to the subject an additional therapeutic agent.

14. The method of claim 13 , wherein the additional therapeutic agent is a beta blocker, a nonselective adrenergic agonist, a selective a-2 adrenergic agonist, a carbonic anhydrase inhibitor, a prostaglandin analog, a para-sympathomimetic agonist, a carbachol or a combination thereof.

15. The method of claim 13 , wherein the additional therapeutic agent is timoiol, levobunolol, metipranolol carteolol, betaxolol, epinephrine, apraclonidine, brirnonidine, acetazolamide, methazolamide, dorzolamide, brinzolamide, latanoprost, travaprost, bimataprost, pilocarpine, echothiophate iodide, carbachol, or a combination thereof.

16. A method of preventing glaucoma in a subject in need thereof, comprising the step of orally administering to the subject a pharmaceutical composition containing a therapeutically effective amount of nicotinamide (NAM) and pyruvate to reduce axonal degeneration in a retinal ganglion cell and prevent glaucoma, wherein the subject is a mammal and is administered between 1 and 5 g/day of NAM and between 1 and 5 g/day of pyruvate.

17. A method of improving visual function in a subject in need thereof, comprising the step of orally administering to the subject a pharmaceutical composition containing a therapeutically effective amount of nicotinamide (NAM) and pyruvate to reduce axonal degeneration in a retinal ganglion cell and improve visual function, wherein the subject is a mammal and is administered between 1 and 5 g/day of NAM and 10 between 1 and 5 g/day of pyruvate.

18. The method of claim 16 , wherein the subject is a human subject.

19. The method of claim 17 , wherein the subject is a human subject.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 11, 2022
From: JOHN, SIMON W.M.; WILLIAMS, PETER ALEXANDER
To: THE JACKSON LABORATORY
Reel/Frame 060778/0594 →
Continuity (5)
Continuation 15959379 · Apr 23, 2018
Continuation PCTUS2016058388 · Oct 24, 2016
Provisional Application 62366211 · Jul 25, 2016
Provisional Application 62245467 · Oct 23, 2015
Related Publication 20220354838A1 · Nov 10, 2022
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