Stoke Therapeutics Comes Out of Seed Mode With $40M and Ex-Sarepta CEO at Its Helm

Stoke Therapeutics launched with a $40 million Series A financing and former Sarepta chief executive officer Edward Kaye at the helm.

The Series A round was entirely funded by founding investor Apple Tree Partners. The company’s technical approach uses antisense oligonucleotides developed by the company’s scientific founder, Adrian Krainer, of Cold Spring Harbor Laboratory. Krainer was also the inventor of Biogen’s Spinraza (nusinersen) for Spinal Muscular Atrophy (SMA), which uses antisense technology. The initial focus is on modulating RNA splicing to improve the production of messenger RNA.

The company has already validated hundreds of genes that can be up-regulated with its TANGO (Targeted Augmentation of Nuclear Gene Output) platform. As such, its priorities include tissues of the central nervous system, eye, and liver. The initial focus will be on inherited diseases caused by a single gene mutation.

“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 Sam Hall, principal of Apple Tree Partners and a Stoke board director, in a statement.

When Kaye left Sarepta in August, he planned to take time off before tackling another company. But within a matter of weeks, Kaye told Endpoints News’ John Carroll, he had six offers.

He also already knew Krainer. Carroll writes, “Krainer, an RNA splicing expert who played a key role in the discovery of Spinraza, had been studying the body’s machinery for making proteins and a method using antisense olignonucleotides to increase protein expression. Adding a bioinformatics approach, the fledgling company was able to identify key diseases driven by missing protein—and now they’re out to use the tech to address diseases that seem outside the realm of such technologies as gene editing or gene therapy.”

At the moment, Stoke has 14 employees, and is expected to double that in the near future. Kaye hopes to take the company’s lead candidate into the clinic in 2019. “My goal,” he told Carroll, “is to take this all the way to completion and build a company that can be sustainable.”

In addition to Kaye, Huw Nash is chief operating officer and chief business officer, and founding chief executive officer. He is an Entrepreneur-in-Residence at Apple Tree Partners. Barry Ticho is chief medical officer. He was previously at Moderna Therapeutics, Pfizer and Biogen. Isabel Aznarez is co-founder and vice president, Head of Biology. She was previously at Krainer’s laboratory and has significant experience in human genetics, RNA metabolism, and RNA modulation. Charles Allerson is vice president, head of chemistry. He previously held senior roles at Regulus Therapeutics and Ionis Pharmaceuticals.

The company’s board of directors includes Kaye, Krainer, Arthur Levin, currently executive vice president, R&D at Avidity Biosciences, Seth Harrison, founder and managing partner of Apple Tree Partners, and Sam Hall.