Expanded Access Policy

Stoke is a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases. Inherent in our pursuit is a deep commitment to the people who are living with these diseases. To them and to their families, we commit to working with a sense of urgency and scientific rigor to discover and develop new medicines.

It is our duty to carefully evaluate the safety and efficacy of any of our potential new medicines. We believe that clinical trials are the best and most appropriate way to do that. The process is a team effort and we will work closely with researchers, physicians, clinical trial site teams, patients and their families to conduct our studies efficiently and effectively. When successful, data from these studies form the basis of regulatory (i.e. FDA, EMA, or similar regulatory agencies) approvals, which ensure the most equitable and sustainable access to medicines for the people who need them.

We know that patients who are living with severe genetic diseases and their families are counting on us and that every day matters. Given this, we understand that some patients and their families may be interested in accessing our investigational medicines prior to regulatory approval, and outside of the clinical trial setting, through an expanded access program (sometimes referred to as “early access” or “compassionate use”).  Stoke does not offer programs that allow access to our investigational medicines prior to regulatory approval. For now, participation in a clinical trial is the only way to access our investigational medicines.

More information about Stoke’s current clinical trials is available online (see links below).  If you have additional questions, please talk to your doctor or contact clinicaltrials@stoketherapeutics.com. We anticipate acknowledging receipt of requests sent to this email within five business days.

Current trials: