Invention Grant
- Patent Title: Bicalutamide analogs or (S)-bicalutamide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis
-
Application No.: US15102358Application Date: 2014-12-19
-
Publication No.: US11571407B2Publication Date: 2023-02-07
- Inventor: Josep Farrera-Sinfreu , Leslie Matalonga Borrel , Laura Gort Mas , Roberto Pascual Martínez , Antonio Ferrer Montiel , Antonia Ribes Rubió , Berta Ponsati Obiols
- Applicant: BCN PEPTIDES, S.A.
- Applicant Address: ES Sant Quinti De Mediona Barcelona
- Assignee: BCN PEPTIDES, S.A.
- Current Assignee: BCN PEPTIDES, S.A.
- Current Assignee Address: ES Sant Quinti De Mediona Barcelona
- Agency: Harness, Dickey & Pierce, P.L.C.
- Priority: EP13382541 20131223
- International Application: PCT/EP2014/078745 WO 20141219
- International Announcement: WO2015/097088 WO 20150702
- Main IPC: A61K31/277
- IPC: A61K31/277 ; A61K31/167 ; A61K47/42 ; G01N33/566
Abstract:
The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds that promote exocytosis, preferably lysosomal exocytosis. The treatment of cells from patients affected by different lysosomal storage disorders with exocytosis activating compounds leads to a decrease in the accumulation of toxic substrate in the lysosomes, thus allowing the treatment, prevention and relief of the symptoms of many lysosomal storage disorders.
Public/Granted literature
Information query
