Use of IGF-2 receptor agonist ligands for treatment of Angelman Syndrome and autism
Inventors
ALBERINI, CRISTINA MARIA • Trauner, Dirk • ARP, CHRISTOPHER JAMES
Assignees
Publication Number
US-12083139-B2
Publication Date
2024-09-10
Expiration Date
2039-08-12
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Abstract
Provided are methods for treatment of neurodevelopmental disorders, such as Angelman Syndrome and autism comprising administering to an individual a composition comprising an agonist ligand of IGF-2 receptor. The agonist ligand of IGF-2 receptor may be IGF-2, or mannose-6-phosphate or a derivative thereof. Compositions comprising mannose-6-phosphate derivatives are also disclosed.
Core Innovation
The present invention provides methods for the treatment of neurodevelopmental disorders, including Angelman Syndrome (AS) and autism, by administering a composition comprising an agonist ligand of the IGF-2 receptor. The agonist ligands may include IGF-2, mannose-6-phosphate (M6P), or derivatives of M6P where specific modifications are made to carbon 1 and/or carbon 6 of mannose. These compositions are designed to be specific for the IGF-2 receptor, and include both the native compounds and their pharmaceutically acceptable salts and derivatives.
The problem addressed by this invention is the lack of effective therapies for AS and autism, which are associated with developmental delays, intellectual disabilities, motor dysfunction, and behavioral abnormalities. Current medical options do not provide a cure or viable means to ameliorate the various symptoms of these disorders, which include speech and movement difficulties, seizures, and repetitive behaviors.
The invention solves these problems by providing compositions and methods that involve administration of IGF-2 receptor agonist ligands, which have been shown, in animal models, to reverse or ameliorate memory, motor, and behavioral deficits characteristic of Angelman Syndrome. The invention also details the preparation and pharmaceutical formulation of these ligands and their derivatives, establishing a platform for therapeutic intervention in AS and autism via IGF-2 receptor targeting.
Claims Coverage
There are two independent claims defining two core inventive features.
Compound having the specified structure
A compound possessing a structure as detailed in the patent, specifically a derivative of mannose-6-phosphate (M6P) with modifications at carbon 1 and/or carbon 6 as described in the patent, suitable as an IGF-2 receptor agonist ligand for therapeutic use.
Pharmaceutically acceptable salt of the specified compound
A pharmaceutically acceptable salt form of the compound having the defined structure, enabling formulation and use as a therapeutic agent targeting the IGF-2 receptor.
The inventive features encompass both the specific compound structure and its pharmaceutically acceptable salt form for use as IGF-2 receptor agonist ligands.
Stated Advantages
The compositions and methods described can reduce or delay one or more symptoms associated with Angelman Syndrome or autism, including cognitive, developmental, and motor impairments.
Systemic administration of IGF-2 receptor agonist ligands such as IGF-2, M6P, or M6P derivatives can reverse memory deficits, motor dysfunction, and repetitive behavior in animal models of Angelman Syndrome.
Documented Applications
Treatment of neurodevelopmental disorders, specifically Angelman Syndrome and autism spectrum disorders, by administration of IGF-2 receptor agonist ligands.
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