Aspera Biomedicines, Inc. (“Aspera”) is a privately-held platform company that focuses on developing RNA modulatory and immuno-oncology drug assets to treat patients with refractory malignancies that have hijacked stem cell properties including the capacity to self-renew (divide without differentiating) and become dormant, albeit in a dysregulated manner. This property of “cancer stemness” is the main target of our drug development platforms.

 

The first asset, Rebecsinib (17S-FD-895), is currently undergoing IND enabling studies as a selective splicing modulator that inhibits both ADAR1-mediated immune silencing and pro-survival MCL1-L expression that collectively drive therapy resistant cancer stem cell generation in a broad array of malignancies.  

 

Other selective ADAR1 inhibitors are uniquely enabled by our purified human ADAR1 and lentiviral reporter technologies.

 

Because ADAR1 inhibition overcomes immune checkpoint blockade resistance and prevents self-renewal of cancer stem cells (CSCs) while MCL1 inhibition prevents CSC survival, initial clinical trials will focus on relapsed/refractory myelofibrosis (MF), myelodysplastic syndrome (MDS), and secondary AML that have high levels of ADAR1 and MCL1.  

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The Aspera management team founded Impact Biomedicines, Inc, which translated a $22 million investment into a $7 billion milestone-driven acquisition and resurrection of a cancer stemness blocking JAK2 inhibitor, Inrebic ®, which is now FDA and EMA approved to eradicate cancer stem cells in myelofibrosis.