Desmin-related cardiomyopathy therapeutics development
Innovative therapeutics targeting genetic-driven dilated cardiomyopathy (DCM) using human induced pluripotent stem cells (hiPSC) and engineered heart tissue models. Aims to identify drug candidates that address cellular metabolism and mitochondrial perturbations.
Overview
Desmin-related cardiomyopathy therapeutics focus on addressing genetic-driven dilated cardiomyopathy (DCM), a condition that leads to heart failure or sudden cardiac death, particularly in patients with myofibrillar myopathies and desminopathies. The research leverages human induced pluripotent stem cells (hiPSC) derived from patients with mutations in DES or DMD genes to understand cellular abnormalities in cardiomyocytes. The ultimate goal is to identify effective drug candidates to mitigate these impacts.
Technical specifications
Key features include:
- Use of hiPSC lines to phenotype cellular abnormalities in cardiomyocytes.
- Identification of metabolic and mitochondrial disturbances through high content imaging.
- Development of a high throughput drug screening assay.
- Creation of a novel 3D engineered heart tissue model adjusting extracellular matrix properties to study the microenvironment's role in disease progression.
Technology readiness level
Currently, the technology is at TRL 3, indicating experimental proof of concept. Ongoing efforts within a research consortium aim to advance target and drug discovery, enhancing the path toward clinical application.
About KSILINK
Ksilink is a drug discovery company that utilizes a proprietary AI-enabled phenotypic screening platform to identify novel therapeutic candidates. The company specializes in human patient-derived cellular disease models, which it automates for large-scale, high-throughput screening. By integrating morphometric profiling, deep learning, and advanced image mining, Ksilink captures complex disease biology to identify druggable targets and efficient compounds, aiming to establish human proof-of-concept early in the development process. The company operates through a hybrid model, advancing its own proprietary CNS pipeline while also engaging in strategic collaborations across various therapeutic areas.
By leveraging these patient-based models, Ksilink addresses significant unmet medical needs and seeks to reduce high failure rates often associated with traditional, less predictive preclinical models. Their approach allows for the systematic investigation of drug candidates, including small molecules and genetic modalities, by observing phenotypic shifts in diseased cells toward healthy states. This capability is supported by a robotic screening facility and expertise in interpreting large biological datasets, enabling the company to provide robust, reproducible data to partners and accelerate the transition of candidates toward preclinical and clinical development.
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