Compositions and methods for treating huntington's disease
Inventors
Assignees
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Publication Number
US-9572825-B2
Publication Date
2017-02-21
Expiration Date
Abstract
The present invention provides intravenous compositions of trehalose for the treatment of signs and symptoms of Huntington's disease.
Core Innovation
The invention describes treating Huntington’s Disease by intravenously administering trehalose. The method includes identifying a subject with Huntington’s Disease and administering an aqueous formulation for intravenous injection comprising a single active ingredient consisting of trehalose. The overall context is the alleviation of a sign or symptom of Huntington’s Disease using the defined intravenous trehalose formulation.
The formulation and administration are defined by specified properties, including a pH about 4.5 to 7.0 and less than 0.75 endotoxin units per mL. The per administration dose is between 5 and 50 grams trehalose, and administration is completed within less than 120 minutes. The described approach is associated with trehalose acting as a protein aggregation inhibitor.
The described content further states that rapid intravenous administration supports cellular uptake and retention for 48 to 72 hours. The documented technical discussion includes evaluation of intravenous versus oral pharmacokinetics and safety rationale related to trehalose.
Claims Coverage
The independent claim covers one method of alleviating a sign or symptom of Huntington’s Disease using an intravenous aqueous trehalose formulation under defined formulation quality and administration time/dose constraints. The claim includes 4 core inventive feature areas.
Intravenous trehalose for alleviating Huntington’s Disease signs or symptoms
A method of alleviating a sign or symptom of Huntington’s Disease by identifying a subject with Huntington’s Disease and intravenously administering an aqueous formulation for intravenous injection comprising a single active ingredient consisting of trehalose.
Single-active ingredient aqueous trehalose formulation with defined pH and endotoxin
The aqueous trehalose formulation has a pH about 4.5 to 7.0 and contains less than 0.75 endotoxin units per mL.
Constrained per-administration dose and administration completion within 120 minutes
The formulation is administered at a per administration dose of between 5 to 50 grams trehalose, with administration completed within less than 120 minutes.
Across the covered inventive features, the claim requires an identified Huntington’s Disease subject receiving an aqueous intravenous formulation containing only trehalose, with specified pH and endotoxin limits, and dosing that is completed in less than 120 minutes within the stated per-administration trehalose dose range.
Stated Advantages
Alleviating a sign or symptom of Huntington’s Disease.
Rapid intravenous administration supports cellular uptake and retention for 48 to 72 hours.
Documented Applications
Treatment of Huntington’s Disease by alleviating a sign or symptom using an intravenous aqueous trehalose formulation under specified formulation and administration constraints.
Intravenous trehalose as a treatment for aggregation diseases, notably OPMD (oculopharyngeal muscular dystrophy) and Huntington’s disease among others.
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