For decades, drug discovery has centered on targeting proteins to treat disease. While we’ve made great strides with this approach, we are quickly reaching the limits of what is easily druggable. It’s time for a new set of tools to not only treat disease in new ways, but treat diseases more effectively.
RNA Processing – An Untapped Opportunity
Just as an instrument can be tuned to create an entirely different sound, RNA transcripts can also be tuned in a multitude of ways, extending far beyond simply splicing out non-coding introns. Development of new molecular tools and the advent of next-generation, multi-omic disease datasets has given us better insight into RNA’s vast intricacies and unveiled new, exciting insights into how RNA is processed and dysregulated after transcription. By unlocking these tools and controlling RNA processing we can target the drivers of disease at their origin, affording rich therapeutic opportunities.
RNA Drug Discovery, Remixed
Even with these large datasets, it has still proved challenging to decipher the language of how cells fine-tune RNA. Remix is applying data in new ways, seeing opportunities where others have traditionally seen hurdles to create small molecule modulators that target and reprogram the RNA processing machinery. Using our REMaster platform, we are able to clearly identify patterns and rapidly arrive at key insights to modify expression of disease-causing genes, delivering breakthrough therapies for patients.
The REMaster platform enables the design of molecules that can selectively degrade RNA, enhance RNA expression, induce exon skipping or rescue genetic lesions. Our technology can address undrugged disease drivers across therapeutic areas to rapidly bring new medicines to patients.
Expanding the Universe of Druggable Targets
Realizing the vast, untapped opportunities before us, Remix is making this vision a reality by advancing a pipeline of novel, small molecule therapeutics that can alter RNA processing in a multitude of ways, unlocking new strategies to provide treatments across the disease landscape, including cancers, central nervous system diseases, rare genetic diseases and beyond.