Claims for Patent: 11,400,067

Summary for Patent: 11,400,067

Title:	Pharmaceutical compositions and uses directed to lysosomal storage disorders
Abstract:	The present disclosure provides for treating lysosomal storage disorders (LSDs) comprising administering acetyl-leucine or a pharmaceutically acceptable salt thereof.
Inventor(s):	Michael Strupp
Assignee:	Intrabio Ltd
Application Number:	US16/324,301
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 11,400,067
Patent Claims:	1. A method of treating a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years, wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria. 2. A method of delaying progression of a lysosomal storage disorder (LSD) or more symptoms associated with the LSD over time as compared to typical disease progression in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years. 3. A method of reversing progression of a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD over time in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years. 4. A method of improving in a subject in need thereof a biochemical marker of a lysosomal storage disorder (LSD) over time comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years. 5. A method of reducing the severity of a lysosomal storage disorder (LSD) or reducing the severity of or eliminating one or more existing symptoms associated with the LSD in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject, wherein the LSD is not Niemann-Pick Type C. 6. A method of providing neuroprotection in a subject having, suspected of having, or at risk of having a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years. 7. A method of delaying or reversing progression of a lysosomal storage disorder (LSD) or reducing the severity of the LSD in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject, wherein the LSD is not Niemann-Pick Type C and the subject is asymptomatic. 8. The method according to claim 4, wherein the biochemical marker is increased lysosomal volume. 9. The method according to claim 1, wherein the method comprises initiating administration of a therapeutically effective amount of acetyl-leucine to the subject in need thereof when the subject is asymptomatic. 10. The method according to claim 4, wherein the initial administration occurs after the subject has been found to have a genetic and/or biochemical marker of the LSD. 11. The method according to claim 1, wherein the acetyl-leucine is acetyl-DL-leucine. 12. The method according to claim 1, wherein the acetyl-leucine has an enantiomeric excess of the L-enantiomer or the D-enantiomer. 13. The method according to claim 1, wherein the acetyl-leucine is in a single enantiomeric form of either the L-enantiomer or the D-enantiomer. 14. The method according to claim 13, wherein the single enantiomeric form is the L-enantiomer. 15. The method according to claim 1, wherein the method comprises administering the acetyl-leucine to the subject in need thereof at a therapeutically effective amount of from about 1 g to about 15 g per day. 16. The method according to claim 1, wherein the LSD is chosen from Niemann-Pick type A disease, Niemann-Pick type B disease, and Niemann-Pick type C disease. 17. The method according to claim 15, wherein the method comprises administering the acetyl-leucine to the subject in need thereof at a therapeutically effective amount chosen from about 1 g to about 10 g per day, about 1.5 g to about 7 g per day, about 4 g to about 6 g per day, or about 4 g to about 5 g per day. 18. The method according to claim 1, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis. 19. The method according to claim 2, wherein the method comprises initiating administration of a therapeutically effective amount of acetyl-leucine to the subject in need thereof when the subject is asymptomatic. 20. The method according to claim 2, wherein the therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof ranges from about 1 g to about 15 g per day. 21. The method according to claim 2, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis. 22. The method according to claim 3, wherein the therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof ranges from about 1 g to about 15 g per day. 23. The method according to claim 3, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis. 24. The method according to claim 4, wherein the method comprises initiating administration of a therapeutically effective amount of acetyl-leucine to the subject in need thereof when the subject is asymptomatic. 25. The method according to claim 4, wherein the therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof ranges from about 1 g to about 15 g per day. 26. The method according to claim 4, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis. 27. The method according to claim 5, wherein the therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof ranges from about 1 g to about 15 g per day. 28. The method according to claim 5, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis. 29. The method according to claim 6, wherein the method comprises initiating administration of a therapeutically effective amount of acetyl-leucine to the subject in need thereof when the subject is asymptomatic. 30. The method according to claim 6, wherein the therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof ranges from about 1 g to about 15 g per day. 31. The method according to claim 6, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis. 32. The method according to claim 7, wherein the therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof ranges from about 1 g to about 15 g per day. 33. The method according to claim 7, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, mucolipidosis II, MPS III, and GM1 gangliosidosis. 34. The method according to claim 7, wherein the therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof is administered to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years.