	US 12,076,332 B2
	Treatment of lysosomal storage disorders
Gal Bitan, Encino, CA (US); Alessandro Fraldi, Rome (IT); Irene Sambri, Rome (IT); and Antonio Monaco, Rome (IT)
Assigned to The Regents of the University of California, Oakland, CA (US); and FONDAZIONE TELETHON ETS, Rome (IT)
Appl. No. 17/050,406
Filed by The Regents of the University of California, Oakland, CA (US); and Fondazione Telethon, Rome (IT)
PCT Filed Apr. 25, 2019, PCT No. PCT/US2019/029222 § 371(c)(1), (2) Date Oct. 23, 2020, PCT Pub. No. WO2020/023094, PCT Pub. Date Jan. 30, 2020.
Claims priority of provisional application 62/663,964, filed on Apr. 27, 2018.
Prior Publication US 2021/0052611 A1, Feb. 25, 2021
Int. Cl. A61K 31/663 (2006.01); A61K 31/10 (2006.01); A61K 31/19 (2006.01); A61K 31/6615 (2006.01); A61P 25/00 (2006.01)

CPC A61K 31/663 (2013.01) [A61K 31/10 (2013.01); A61K 31/19 (2013.01); A61K 31/6615 (2013.01); A61P 25/00 (2018.01)]

12 Claims

1. A method of treating a lysosomal storage disease in a mammal, said method comprising:

administering to said mammal an effective amount of a molecular tweezers that is capable of inhibiting protein aggregation, wherein said effective amount is an amount effective to slow the progression, or stop, or reverse protein accumulation/aggregation associated with said lysosomal storage disease, and/or said effective amount is an amount effective to ameliorate one or more symptoms of the pathology associated with said lysosomal storage disease and/or to reduce neurodegeneration and/or neuro-inflammation associated with said lysosomal storage disease;

wherein said lysosomal storage disease is Sanfilippo syndrome, and said method is effective to reactivate autophagic flux in the neurons of the mammal.