US 12,458,614 B2
Therapeutic agents for improved mobility and cognitive function and for treating neurodegenerative diseases and lysosomal storage disorders
Mallory Factor, Oxford (GB)
Assigned to Intrabio Ltd, Yarnton (GB)
Appl. No. 16/756,990
Filed by INTRABIO LTD, Yarnton (GB)
PCT Filed Oct. 18, 2018, PCT No. PCT/US2018/056420
§ 371(c)(1), (2) Date Apr. 17, 2020,
PCT Pub. No. WO2019/079536, PCT Pub. Date Apr. 25, 2019.
Claims priority of provisional application 62/574,132, filed on Oct. 18, 2017.
Claims priority of provisional application 62/574,137, filed on Oct. 18, 2017.
Claims priority of provisional application 62/574,141, filed on Oct. 18, 2017.
Prior Publication US 2020/0338034 A1, Oct. 29, 2020
Int. Cl. A61K 31/198 (2006.01); A61P 25/28 (2006.01)
CPC A61K 31/198 (2013.01) [A61P 25/28 (2018.01)] 13 Claims
OG exemplary drawing
 
1. A method for treating a lysosomal storage disorder (LSD) comprising:
directly delivering leucine, or a pharmaceutically acceptable salt thereof, to the central nervous system of a subject having LSD or one or more symptoms associated with a LSD;
wherein the leucine, or pharmaceutically acceptable salt thereof, is delivered in a therapeutically effective amount to treat the LSD or the one or more symptoms associated with the LSD;
wherein the direct delivery is by an administration chosen from intrathecal administration and intracranial administration;
wherein the LSD is chosen from Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Niemann-Pick type C 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; and
wherein the leucine is not acetyl-leucine and wherein the therapeutically effective amount of leucine 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.