Patients & Families

Patients are our guiding force. On a global scale we strive to understand their individual journeys and unique needs. As we work to advance our pipeline of investigational therapies, we recognize the essential need to gain important perspectives from patients, families and leaders in patient advocacy.

Read more read more

Meet our inspiration

The resilience of the people we serve is our inspiration to push through barriers to create a world where no life is limited by genetic disease. These are just some of the individuals who drive us to do more.

Our commitment to the patient community

As our scientific programs progress, we are grateful to work alongside advocacy groups, raising awareness of and supporting people affected by rare genetic diseases. Learn more about our disease areas of focus, and hear from members of our scientific team about what motivates them every day to propel Spark Therapeutics’ gene therapy research programs forward.

Check out these rare genetic disease organizations for more information

Meet our patient advocacy team

As a bridge between Spark Therapeutics and patient communities, we aim to create meaningful relationships that help us challenge the inevitability of genetic disease and address the needs of those communities we serve.

Get in touch

For questions about all of Spark Therapeutics’ programs,
email us at or
call 1-855-SPARKTX / +1 215-220-9300 in the United States.
We also invite you to sign up here to receive updates on Spark Therapeutics’ progress.

Spark Therapeutics Generation Patient Services℠

Providing a caring support team,
from confirmed diagnosis through postsurgery follow-up.

Visit for more information

For questions, contact us at 1-833-SPARK-PS (833-772-7577).

Spark Therapeutics clinical trials

Spark Therapeutics is discovering and developing investigational gene therapies for debilitating inherited diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia and lysosomal storage disorders, and neurodegenerative diseases. To see our current pipeline of investigational therapies, click here. For more information about our ongoing clinical trials, visit and search for “Spark Therapeutics.”

Go to

To learn more about how clinical trials work, visit the National Institutes of Health (NIH) website.