Erythritol Phenylbutyrate Esters for Improved Bioavailability
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
Existing phenylbutyrate esters, such as those of glycerol, do not address disorders associated with mutations in SLC6A1, STXBP1, SYNGAP, FOXG1 genes, or endoplasmic reticulum stress, and lack enhanced therapeutic efficacy.
Innovation Solution
Development of phenylbutyrate esters of erythritol, specifically (2R,3S)-butane-1,2,3,4-tetrayl tetrakis(4-phenylbutanoate) and (2S,3R)-4-hydroxybutane-1,2,3-triyl tris(4-phenylbutanoate), which demonstrate enhanced pharmacokinetics and therapeutic efficacy.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If phenylbutyrate esters of glycerol are used for treatment, then therapeutic effect is achieved, but therapeutic efficacy is limited and does not address disorders associated with SLC6A1, STXBP1, SYNGAP, FOXG1 gene mutations or endoplasmic reticulum stress
Solution Approach 1:
The patent changes the chemical parameter by substituting glycerol with erythritol as the alcohol component in phenylbutyrate esters. This structural modification creates new compounds with enhanced therapeutic efficacy that can address disorders associated with SLC6A1, STXBP1, SYNGAP, FOXG1 gene mutations and endoplasmic reticulum stress, while maintaining the core phenylbutyrate therapeutic mechanism.
Solution Approach 2:
The patent develops composite chemical structures by combining phenylbutyrate moieties with erythritol backbone, creating tetra-phenylbutyrate ester of erythritol and other derivatives. These composite molecules exhibit synergistic properties that enhance therapeutic effectiveness across multiple disorder types compared to conventional glycerol-based esters.
2Reliability
If conventional phenylbutyrate esters are administered, then treatment is provided, but bioavailability and pharmacokinetic properties are suboptimal
Solution Approach 1:
The patent optimizes pharmacokinetic parameters by modifying the molecular structure from glycerol-based to erythritol-based phenylbutyrate esters. This change improves bioavailability, extends mean residence time (MRT), and optimizes clearance characteristics, thereby enhancing the overall pharmacokinetic performance and therapeutic reliability of the compound.
Data Source
AI summary
The present application discloses an erythritol analog of phenylbutyrate, and compositions including same. Also disclosed are methods of using the erythritol analog of phenylbutrate.