Our Story

Meet our founders

Stoke’s co-founders, Professor Adrian Krainer, Ph.D., and Isabel Aznarez, Ph.D., launched the company with the goal of targeting pre-mRNA splicing to develop precision medicines to treat the underlying cause of genetic diseases. Now, Stoke’s chief executive officer, Ed Kaye, M.D., and chief operating and business officer, Huw Nash, Ph.D., are leading the team as they develop new medicines to precisely upregulate protein expression. Explore the story below.

Passion for genetic disease research

“Everything I do, every project I’ve had, I’m always thinking of it in the context of how it can be used to treat the underlying cause of diseases. That goal has never changed.”

– Dr. Aznarez

Dr. Aznarez and Professor Krainer began working together in 2008 at Cold Spring Harbor Laboratory in New York. Their focus has been on applying antisense oligonucleotides to modulate RNA splicing and RNA metabolism pathways. They share a passion for addressing genetic diseases, and also share a common heritage, as both were born in Uruguay. TANGO is the name of Stoke’s foundational technology and is a nod to their native country. Today, Dr. Aznarez continues to guide research as Stoke’s head of biology, Professor Krainer remains closely involved as a member of Stoke’s board of directors, Dr. Nash is the chief operating officer and business officer and Dr. Kaye is the chief executive officer.

A revolutionary technology

“We discovered an innovative scientific approach to restore protein expression in patients with severe genetic diseases. This novel approach addressed many of the limitations of existing modalities, such as gene therapy or enzyme replacement, and opened up a vast new area of drug development.”

– Professor Krainer

Dr. Aznarez and Professor Krainer’s early work led to the development of our proprietary TANGO platform, which is designed to allow us to deliver disease-modifying therapies to a wide-range of relevant tissues in a highly precise, durable and controlled manner.  While the TANGO mechanisms are novel, Stoke is able to leverage antisense chemistry that has been clinically validated for the central nervous system (CNS), which we believe minimizes the safety and biodistribution risks for Stoke’s CNS programs, including Dravet syndrome and other genetic epilepsies.

The birth of a biotech startup

“At a time when many companies were pursuing downregulation of protein expression with antisense oligonucleotide (ASO) and RNA interference technologies, Adrian and Isabel came to Apple Tree Partners with a very unique and gene-specific approach to upregulate protein expression with ASOs. We quickly realized the potential of this approach to treat a significant and underserved set of genetic diseases, those caused by haploinsuffiency, and Stoke was born.”

– Dr. Nash

Dr. Aznarez and Professor Krainer recognized that their insights on RNA splicing and RNA metabolism pathways could potentially be translated into medicines to improve the lives of people living with serious diseases. Teaming up with Dr. Nash, the three founded Stoke Therapeutics. Backed by a veteran leadership team and $40 million from Apple Tree Partners, Stoke emerged from stealth mode in January 2018. Stoke subsequently added $90 million in Series B financing from a syndicate of investors in October 2018.

Rapid progress toward the clinic

“Early on, Stoke wanted to combine proven antisense chemistries with novel biology to tackle genetic epilepsies, an area in which the regulatory path is relatively well defined and yet the unmet need is still very high. We have a chance to make a profound difference in patients’ lives, and that’s what all of us are working toward.”

– Dr. Kaye

We are using our proprietary TANGO platform to build a pipeline of potential new medicines to help people with severe genetic epilepsies and other diseases that are caused by an individual gene. Stoke’s lead program is a potentially disease-modifying treatment for Dravet syndrome, a severe and progressive form of genetic epilepsy. Beyond genetic epilepsies, we are advancing several other early programs, including haploinsufficiencient diseases of the CNS, eye, kidney and liver.