Small-Molecule Inhibitors for Age-Related Disease Targets
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Solution Overview
Problem
There is a need for new targets, compounds, and compositions to treat aging and age-related diseases effectively.
Innovation Solution
The application identifies therapeutic effects of modulators, specifically inhibitors, targeting a group of proteins to provide anti-aging and senolytic therapies, including small molecule inhibitors that bind and inhibit proteins such as HTR2A, SLC6A3, and others, administered in therapeutically effective amounts.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional treatments are used for aging and age-related diseases, then existing therapies can be administered, but they lack effectiveness in addressing the underlying mechanisms of aging
Solution Approach 1:
The patent segments the complex aging process into specific molecular targets (HDACs, PI3K, mTOR, etc.) and addresses each with dedicated inhibitor compounds. This segmentation allows for targeted intervention at multiple points in the aging pathway, improving treatment effectiveness while providing versatility through multiple actionable targets.
Solution Approach 2:
The patent employs small molecule inhibitors that specifically bind to and modulate the activity of key proteins involved in aging. By changing the molecular parameters (inhibitor concentration, target binding affinity), the therapy can effectively interrupt aging-related pathways without affecting normal physiological functions.
2Reliability
If new targets and compounds are developed for anti-aging therapy, then treatment effectiveness improves, but the complexity of the therapeutic approach increases
Solution Approach 1:
The patent identifies multiple targets (HDAC1-11, PI3K isoforms, mTOR, etc.) that are universally involved in aging processes across different tissues and age-related diseases. A single inhibitor compound can target multiple proteins within these pathways, providing multi-functional therapeutic effects while simplifying the administration approach.
Solution Approach 2:
The patent uses small molecule inhibitors as intermediary compounds that mediate the interaction between the therapeutic agent and the aging pathways. These intermediaries (inhibitors) translate the complex molecular targets into manageable drug molecules that can be administered systemically, bridging the gap between target complexity and clinical usability.
Data Source
AI summary
Disease target identification is among the most difficult problems in biomedicine. The sheer size of genetic signal (number of observed features per genome) poses a nearly insurmountable challenge of the required number of samples for association studies. Here, we applied a state-of-the-art deep neural network for feature selection (dimensionality reduction) and non-biological variance (batch) removal in human cells. By integrating genome-wide shRNA targeting, exome-wide ultra-rare mutations, and large cohort survival data, we obtained gene expression signatures associated with extended lifespan. In turn, these signatures allowed us to identify small molecules based on their association with longevity and prioritize known and novel targets of anti-aging therapeutics.