Editor’s note: This story is part of our 2022 PharmaVoice 100 feature.
Our honoree: Dr. Edward Kaye
Title: CEO, Stoke Therapeutics
The company’s focus: Through its proprietary technology platform, TANGO, Stoke Therapeutics aims to increase protein production from healthy genes to stop or slow disease progression. Currently, the company is focusing on two therapeutic areas: the central nervous system and ophthalmology.
Its lead product — STK-001 — is in clinical testing for the genetic disorder Dravet syndrome, a severe and progressive form of epilepsy that impacts infants usually within the first year of life. The company is also investigating treatments for SYNGAP1 and Rett syndromes in collaboration with Acadia Pharmaceuticals.
Under its ophthalmology umbrella, Stoke has a target in preclinical stages to address autosomal dominant optic atrophy (ADOA), a progressive and severe genetic disorder impacting the optic nerve leading to irreversible vision loss usually before patients reach age 10. Currently, there are no approved treatment options for ADOA.
Kaye’s biggest wins: Kaye’s roots as a biochemical geneticist and pediatric neurologist are the foundation of his success as a drug developer and industry influencer. Over the course of his career at both Genzyme and Sarepta Therapeutics, Kaye has been involved in bringing seven medicines to market that have helped change the lives of tens of thousands of people.
“From disillusioned clinician to unstoppable drug hunter, Kaye is driven by an underlying desire and commitment to help more patients. Early in his career as a pediatric neurologist, he became frustrated with routinely seeing patients leave his clinic without an official diagnosis. He had no treatments to offer and little hope. An eternal optimist, he saw a future in which patients had more options, and maybe even cures, and realized he could have a much greater impact by being involved in the development of new medicines,” one of his nominators says.
As group vice president of clinical research at Genzyme, where he spent 10 years, Kaye played a key leadership role in leading several drugs to clinical success and eventual approval, including Myozyme in 2006 for Pompe disease.
“Be the last person in the room to speak.”
Dr. Edward Kaye
CEO, Stoke Therapeutics
After Genzyme, Kaye took on the role of chief medical officer at Sarepta, eventually landing the CEO position overseeing the development of Exondys51, the first genetic medicine approved for Duchenne muscular dystrophy.
“Upon leaving Sarepta, Kaye attempted to retire, but realized after a few mornings at Starbucks that he couldn’t let go of the desire to help bring new medicines to people who need them,” one of his nominators says. “In 2017, he joined Stoke Therapeutics as CEO. He was attracted by the science and the potential to develop the first disease-modifying medicine for the treatment of Dravet syndrome.”
Why Kaye is inspiring: “Not every scientist can make a medicine and not every clinician can understand and work the science. Kaye has proven he knows how to translate science into medicine, and he has a unique ability to follow the science but not get lost in it,” one colleague says. “Through it all, Kaye’s commitment to patients is unwavering and indefatigable.”
Kaye’s commitment to patients and families makes him a “trusted partner and a thought leader within the patient community” and inspires his teams to think differently about how to tackle tough challenges. One nominator notes that “he has rescheduled meetings in order to take a call from a patient, a parent or an advocate. He has been beaten up by regulatory authorities and stepped in to mediate conflicts that stand in opposition to the ultimate goal of getting life-changing medicines to patients faster.”
Kaye’s approach to building high-performing teams and creating an inclusive culture based on serious science, hard work and collegiality attracts some of the best and brightest talent.
“In just four and a half years, Kaye has grown the Stoke team from a small privately funded startup with 14 people to a publicly traded company with a high-functioning team of more than 100 people,” a nominator says. “He has helped cultivate a culture where team members are serious about science but don’t take themselves too seriously. He is passionate about coaching and mentoring people, whether they are peers and colleagues, young scientists or up-and-coming biotech professionals embarking on their careers. He powers through 16-hour days, starting with a morning workout, and spending days on calls with investors and in team meetings. Before he goes home, he can often be found socializing with a colleague in Kendall Square.”
A noted industry influencer and speaker, Kaye isn’t shy about sharing the four factors he considers vital to a success in biotech: “Stay focused on the people counting on you — patients and their families; don’t stop innovating; experience leads with a steady hand; and funding matters — raise money when you can, because you may need it tomorrow.”
In his own words: “I was drawn into the life sciences through my pediatric training. I brought my love of taking care of sick children into the biotech industry by developing novel medicines for genetic diseases.”