Target Identification & Validation

Our expertise

By leveraging our physiologically relevant in vitro models and screening expertise, we reveal and validate novel therapeutic targets that the onset and progression of skeletal muscle diseases. We generate robust, functional data that supports target selection and de-risks early-stage drug discovery programs.

Our approach

We use our MyoScreen-based disease models in combination with high-content imaging and high-throughput screening capabilities to perform siRNA and CRISPR library screens.

Our large panel of patient-derived and healthy donors allows us to assess target effects across different genetic backgrounds, while our quantitative phenotypic assays support mechanistic and functional validation.

This approach enables rigorous, scalable, and clinically relevant validation of targets under pathophysiological conditions.

Case study

AI-driven target identification in DMD

Phenotypic screening platform for Duchenne muscular dystrophy (DMD) integrating AI-powered image analysis to extract subtle morphological features that standard readouts fail to detect.

This approach enabled the clear separation of diseased and healthy donor cells, revealing hidden phenotypes.

Approach validated using disease rescuers and miniaturized into a 384-well format to support high-throughput screening.

Learn more about this platform in our recent publication.

How can we work together

Everything is tailored to your needs through a flexible R&D partnership model that fosters true collaboration and innovation. We offer adaptable project structures and FTE allocation to fit your goals.

In cases of joint development, outlicensing opportunities are also available for our MyoScreen™ technology, including assays and customised image analysis scripts.