Healthcare Professionals

At Spark Therapeutics, we are leveraging our unique technical and R&D expertise as we strive to bring gene therapies to patients. We are committed to working with the healthcare community as we expand our partnership to continue advancing state-of-the-art gene therapy research, education and support.




Scientific publications

Adeno-associated viral (AAV) technology

Inherited retinal diseases (IRDs)

Access to genetic testing for IRDs

Inherited retinal diseases (IRDs, also known as inherited retinal dystrophies) are a group of rare blinding conditions caused by one of more than 220 different genes. It is estimated that more than two million people live with inherited retinal diseases (IRDs) globally, but only a small percentage of them have been genetically screened because of limited access to and information about genetic testing. In response to feedback from advocates, families affected by IRDs and healthcare professionals about the current barriers preventing access to genetic screening for IRDs in a consistent manner, Spark Therapeutics has been working closely with physicians in the U.S., South America and Europe to help facilitate access to genetic screening for people with IRDs.

For more information about a treatment for appropriate patients with a specific IRD, click here.


Berger W, Kloeckener-Gruissem B, Neidhardt J. The molecular basis of human retinal and vitreoretinal diseases. Progress in Retinal and Eye Research. 2010;29(5):335-375.

Sahel J-A, Marazova K, Audo I. Clinical Characteristics and Current Therapies for Inherited Retinal Degenerations. Cold Spring Harbor Perspectives in Medicine. 2014;5(2).

Veleri S, Lazar CH, Chang B, Sieving PA, Banin E, Swaroop A. Biology and therapy of inherited retinal degenerative disease: insights from mouse models. Dis Model Mech Disease Models & Mechanisms. 2015;8(2):109-129.

Maintaining an active dialogue with the scientific community

Spark Therapeutics seeks to actively engage with physicians, researchers and other healthcare professionals to better understand and provide education about diseases related to our therapeutic areas of interest.

The urgent needs of patients and the challenges before us demand uncompromising focus, tenacity and determination to create a fully integrated company built on world-class science, high-quality manufacturing and thoughtful, sustainable access. We embrace our ethical obligation to develop innovative, investigational treatments for life-altering and life-threatening genetic diseases as rapidly as possible without compromising product quality or patient safety.

To learn more, please click on one of the topics below.

Adverse Events in U.S.
and Product Complaints

Medical Questions

Expanded Access

Other Requests

To report an adverse event or a product complaint, click here and complete the submission form, or contact us by calling 1-855-SPARKTX/+1 855-772-7589.