CYCLODEXTRIN DERIVATIVES FOR THE TREATMENT OR PREVENTION OF LYSOSOMALIC STORAGE DISEASES
Patent Information
- Authority / Receiving Office
- DE · DE
- Patent Type
- Patents
- Current Assignee / Owner
- CYCLOLAB CYCLODEXTRIN RES & DEV LAB
- Filing Date
- 2021-09-29
- Publication Date
- 2026-06-10
AI Technical Summary
Current treatments for lysosomal storage diseases, particularly Niemann-Pick type C disease, rely on high doses of composite isomer mixtures of hydroxypropyl-beta-cyclodextrin (HPBCD) that are ototoxic and have unclear mechanisms of action, necessitating the identification of safer and more effective single isomer cyclodextrin derivatives.
The use of chemically pure, single isomer S-(carboxyalkyl)-thio-cyclodextrin salts, such as Sualfadex sodium, Subetadex sodium, and Sugammadex sodium, which demonstrate efficacy in reducing cholesterol accumulation and improving motor activity in zebrafish models of lysosomal storage diseases, even at lower doses compared to HPBCD.
These single isomer cyclodextrin salts effectively reduce cholesterol accumulation and improve motor function in zebrafish models of lysosomal storage diseases, offering a safer and potentially more effective treatment alternative to HPBCD.