Rare Disease

Rare Disease Drug Discovery


InvivoSciences (IVS) combines its human micro tissue technology and phenotypic assay platform with the systems biology approach to achieve rare disease modeling for its drug discovery.  Our strip tissues are perfect for modeling muscle and connective tissue in micro-well plates.   Since many rare diseases affect heart muscle and skeletal muscle as well as connective tissue, we offer rare disease modeling services as well as drug discovery services including target analysis, compound screening, lead optimization, biomarker discovery, and clinical trial prioritization.

InvivoSciences Cares about Rare Disease Drug Discovery

  • There are ~6,000 rare diseases, but >230 million people are affected when combined
  • Over half of them are children
  • So many of them affect heart muscle and skeletal muscle, and some affect connective tissues, for which we can potentially develop micro tissue disease models in a dish

We Excel in Supporting Precision Medicine Development

Funding from the National Institutes of Health enabled InvivoSciences to demonstrate reconstitution of various patient-specific disease phenotypes in micro-engineered heart tissues; now we call them “NuHeart™“. In collaboration with a cardiologist, Dr. Jennifer Strande, at the Medical College of Wisconsin, we are mass-producing NuHearts™ using iPSCs obtained from patients’ cells or gene-edited iPSCs carrying only a disease-causing single mutation.

Award Winning Technologies

The 3D tissue strip culture is our patented unique 3D culture technology different from 3D spheres or 3D printed tissues.  It is an optimized approach to reconstitute muscle (skeletal, cardiac, smooth) and connective tissues including dermis of the skin in micro-well plates. Based on the invention, we fabricate NuHeartTM and conditioned (e.g., mechanical stretching) to mature for analyzing their electrophysiology, contractility, and profibrotic phenotypes using our high-throughput and high-content phenotypic compound screening platform; we call it, Live-in-Tissue Analyzer (LITATM).

To improve the assay efficiency and reproducibility, processes of cell derivation (e.g., iPSC culture and differentiation) can be optimized for each iPSC line by screening culture conditions.

These award-winning technologies help de-risk the therapeutic development efficiently and quickly. InvivoSciences has won numerous awards since its launching in 2001. A full list of awards can be seen here.