PPIB Antisense Oligomers for Non-Productive CYPB Splicing
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Solution Overview
Problem
Current treatments for diseases and pathologies associated with cyclophilin B are inadequate, necessitating the development of new methods to modulate CYPB levels in specific tissues and the body as a whole.
Innovation Solution
The use of antisense oligomers targeting the PPIB gene transcript to induce non-productive splicing, specifically through exon skipping and terminal intron retention, thereby reducing CYPB protein expression and function.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Quantity of substance
If conventional immunosuppressive drugs like cyclosporin A are used to modulate CYPB, then CYPB levels can be reduced, but the treatment is inadequate and lacks specificity for particular tissues
Solution Approach 1:
The invention segments the body into specific tissues and cell types, allowing targeted modulation of CYPB in particular locations (e.g., liver, kidney, brain) rather than systemic treatment. This is achieved through tissue-specific delivery mechanisms and selective binding to tissue-specific markers, enabling localized therapy that addresses the contradiction between reducing CYPB levels and maintaining treatment specificity.
Solution Approach 2:
The invention applies local quality by creating treatment formulations with different properties for different tissues. Conjugates are designed with tissue-specific targeting moieties that confer different pharmacokinetic and pharmacodynamic characteristics in different organ systems, allowing optimized CYPB modulation in each specific tissue context while avoiding unnecessary exposure in other areas.
2Reliability
If existing treatment methods are used, then CYPB can be modulated, but new treatments are needed to complement or improve current approaches
Solution Approach 1:
The invention creates a universal platform of conjugates that can be adapted for multiple disease states and tissue types. The core conjugate structure combines a targeting moiety with a CYPB-modulating agent, creating a multi-functional system that can be customized for different indications (cancer, inflammation, infection) and tissue targets, thereby expanding treatment versatility while maintaining high reliability through standardized mechanism of action.
Solution Approach 2:
The invention introduces dynamic adaptability by allowing the conjugate structure to be modified based on specific treatment requirements. The targeting moiety and CYPB-modulating agent can be selected and combined in different configurations depending on the disease state, tissue target, and patient characteristics, enabling the treatment system to adapt to diverse clinical scenarios while maintaining consistent effectiveness.
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
This approach effectively modulates CYPB expression, leading to reduced protein function and therapeutic benefits in conditions such as inflammatory diseases, viral infections, and cancers by inducing exon skipping and intron retention, without relying on RNA degradation mechanisms.
Implementation Method 1
an antisense oligomer of 10 to 50 nucleotides comprising a targeting sequence complementary to a region near or within an intron of the PPIB gene transcript
Data Source
AI summary
An isolated or purified antisense oligomer which has a modified backbone structure for modifying pre-mRNA splicing in the PPIB gene transcript or part thereof.