SCN1B Mimetic Peptides for Sodium Channel Activity Modulation
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Solution Overview
Problem
Current therapeutics are lacking for voltage-gated sodium channel β subunits, which are linked to diseases such as epilepsy, sudden death syndromes, and cardiac arrhythmia.
Innovation Solution
Development of engineered βadp1 mimetic peptides and polynucleotides that encode these peptides, which can be delivered via vectors or vehicles like liposomes, micelles, or exosomes, to target and modulate β subunits, affecting their function and regulation.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If β subunit-specific therapeutics are developed, then treatment efficacy for SCN1B-linked diseases is improved, but device complexity and manufacturing difficulty increase due to the need for engineered peptides and delivery systems
Solution Approach 1:
The patent creates simplified copies of the βadp1 peptide sequence (SEQ ID NO: 2) and its functional domains to develop therapeutics that mimic the natural peptide's ability to bind and modulate SCN1B β subunits. These mimetic peptides replicate the therapeutic effect while being more stable and suitable for drug development
Solution Approach 2:
The patent employs nested delivery systems where engineered peptides are encapsulated within delivery vehicles such as liposomes, micelles, or exosomes. This nested structure protects the peptide payload while enabling targeted delivery to specific tissues, thereby improving treatment efficacy without requiring complex external delivery infrastructure
2Reliability
If engineered peptides are used to target β subunits, then therapeutic benefit is improved, but ease of manufacture deteriorates due to peptide engineering requirements
Solution Approach 1:
The patent extracts the essential functional domains from the full-length βadp1 peptide to create smaller mimetic peptides that retain therapeutic activity. This extraction simplifies the manufacturing process by reducing peptide length and complexity while maintaining the ability to bind and modulate SCN1B β subunits effectively
Solution Approach 2:
The patent modifies peptide parameters such as amino acid sequence, length, and chemical stability to optimize manufacturability. These parameter changes include creating derivatives with improved pharmacokinetic properties that are easier to synthesize and formulate while preserving therapeutic benefit
3Productivity
If delivery vehicles like liposomes or exosomes are used, then peptide delivery efficiency is improved, but loss of substance increases due to formulation complexity
Solution Approach 1:
The patent utilizes liposomal delivery vehicles with flexible phospholipid bilayer shells that encapsulate the engineered peptides. These flexible shells protect peptides from degradation during storage and delivery while allowing controlled release at the target site, thereby improving delivery efficiency and reducing peptide loss
Solution Approach 2:
The patent develops composite delivery formulations combining engineered peptides with protective excipients and stabilizing agents within delivery vehicles. These composite materials protect the peptide payload from degradation and improve stability, reducing substance loss while maintaining high delivery efficiency to target tissues
Data Source
AI summary
Described in certain example embodiments herein are voltage gated sodium channel beta subunit 1 mimetic polypeptides, polynucleotides encoding the same, and formulations thereof. Described in certain example embodiments, are methods of inhibiting or otherwise modulating voltage gated sodium channel activity by administering the voltage gated sodium channel beta subunit 1 mimetic polypeptides, polynucleotides encoding the same, and/or formulations thereof to a subject in need thereof.