Cancer is one of the most important health problems facing the world. It is the second leading cause of death and most costly illness in the United States.1
Biosplice’s goal in oncology is to broadly impact cancer therapy through pharmacological modulation of corrupted proteomes at the level of RNA-recoding.
Our scientific platform is rooted in the biological systems that govern alternative splicing (AS). Endowing the human genome with enormous protein coding potential, AS can also commonly be “highjacked” by cancers to enable their growth, survival, dissemination throughout the body, and evasion of current standard-of-care therapies. Biosplice has designed small molecules that inhibit CLK/DYRK family kinases to selectively disrupt alternative splicing in a manner that is tuned to disease and tissue context. This can be lethal to cancer cells that rely on dysregulation of AS for survival, and lead to reversal of drug tolerance mechanisms, and loss of oncogene pathway activation.
A monumental challenge facing durable cancer therapy is the extreme mechanistic diversity and adaptive capacity of tumors within and among cancer patients. For any given drug, benefit is increasingly limited by the subtype of disease and by rapid emergence of drug resistance. Our goal is to deliver broadly active and durable therapy by exploiting AS as a common cancer vulnerability within the ocean of confounding diversity.
- American Cancer Society. Cancer Facts &Figures 2021. Atlanta: American Cancer Society; 2021.