Stoking Protein Production
We believe Stoke’s proprietary research platform, TANGO, can be used to treat a broad spectrum of serious diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies.
Using our proprietary platform, we aim to increase protein production from the remaining healthy copy of a gene and to restore the protein to near normal levels, thereby slowing or stopping disease progression.
A Vision for Our Science
Our novel approach will allow us to pursue disease-modifying treatments for severe diseases. Using the TANGO approach and a deep understanding of RNA science, Stoke researchers design antisense oligonucleotides (ASOs) that bind to pre-mRNA and help the target genes produce more protein.
Stoke’s initial focus is haploinsufficiencies and diseases of the central nervous system and the eye, although proof of concept has been demonstrated in other organs, tissues, and systems, supporting the Company’s belief in the broad potential for its proprietary approach.
“Preclinical data have shown our therapies to be highly precise, durable and controllable. We believe we have the science, the strategy and the team to tackle this tremendous unmet need.”
– Gene Liao, Ph.D., Chief Scientific Officer