Stoke Therapeutics completed a series A round to support the development of its antisense approach to boost gene expression and treat disease caused by genetic insufficiency.
Apple Tree Partners ponied up the $40 million, which will drive Stoke’s antisense oligonucleotide preclinical development programs. The company’s focus is upregulating RNA splicing to ramp up production of messenger RNA that can be translated into protein.
“Stoke Therapeutics represents a bold step forward in opening up a vast new area of drug development focused on upregulation of gene expression,” new CEO Ed Kaye, who takes the helm in the briefest of periods after leaving his former DMD biotech Sarepta, said in a release. “By restoring gene dosage using target-specific antisense approaches, we have the opportunity to create a new way of treating diseases that are not amenable to enzyme replacement, gene therapy or other existing modalities.”
Stoke licensed its tech, dubbed Targeted Augmentation of Nuclear Gene Output (TANGO), from scientific founder Adrian Krainer of Cold Spring Harbor Laboratory.
"Our plan for 2018 is to identify at least three preclinical programs that we can bring for IND studies in 2019," Kaye said.
"Right now, we are focusing on the central nervous system," he said. "We are looking at some of the genetic epilepsies and also some of the autosomal dominant forms of blindness. ... We are looking at other organs, but those [programs] are not as well-developed at this time."
The company will initially zero in on diseases caused by a single malfunctioning gene, but TANGO could potentially be used to modulate a nonmutated gene in order to halt or reverse disease progression, the company said.
"We're focusing on smaller indications and on organs we think we can handle as a small company, but I think the potential for this platform could be much larger," Kaye said.
“We are tremendously impressed by the broad potential of this approach to address so many debilitating diseases and have assembled the team, platform and funding to thoroughly exploit this opportunity,” said Seth L. Harrison, M.D., founder and managing partner of Apple Tree Partners and chairman of the Stoke board of directors.
Stoke joins Atlantic Healthcare and Ionis Pharma in the antisense field. The former kicked off a rolling FDA submission for its inflammatory bowel disease candidate in May, while the latter recently licensed a second gastrointestinal antisense drug to J&J’s Janssen.