Regenerative medicine has been called the “vanguard of 21st century healthcare” by the U.S. Department of Health and Human Services. The global market for regenerative medicine therapies is predicted to reach $67 billion by 2020 and financial analysts believe that the field is at a positive growth inflection point similar to where the field of therapeutic monoclonal antibodies was about 15 years ago.
Over 90 percent of regenerative medicine R&D has focused on stem cell-based therapies and tissue engineering aimed at growing replacement organs in vitro for transplantation. Stem cell therapies for treatment of damaged heart and skeletal muscle and other tissues remain unproven and fraught with controversy, despite over 15 years of research and patient trials. Both stem cell and tissue engineering therapies remain experimental and will be very costly to implement even if efficacy is eventually demonstrated.
The development of small molecules for stimulating endogenous tissue repair and regeneration mechanisms is in its infancy due to the lack of detailed molecular understanding of regenerative mechanisms. Without knowledge of these pathways, druggable targets cannot be identified. Novo Biosciences has pioneered two highly complementary approaches to address this pressing unmet need.
Identifying drug targets
Many lower animals like the zebrafish can rapidly regenerate the form and function of almost any lost or damaged body part. Scientists at Novo Biosciences use zebrafish and other lower animal models to define the genes and genetic pathways required for tissue regeneration. We now know that these pathways are conserved in humans, but have been inactivated for reasons not yet fully understood. By first defining the genetic mechanisms underlying regeneration, our scientists can then identify druggable targets and develop lead small molecules that alter their activity and activate repair and regeneration processes.
Using the zebrafish as a novel drug screening platform
Novo Biosciences is the only regenerative medicine R&D company using the zebrafish as a phenotypic screening platform to identify lead small molecules for regenerative medicine. We screen small molecules with known targets using a simple and rapid tissue regeneration assay. By screening molecules with known targets, we greatly reduce the time and costs associated with defining the mechanisms of drug action and advancing promising leads towards clinical trials. This highly focused approach has led to the discovery of our lead small molecule MSI-1436.