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.
Inherited retinal diseases (IRDs, also known as inherited retinal dystrophies) are a group of rare blinding conditions caused by one of 250+ 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 to help facilitate access to genetic screening for people with IRDs.
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.
RetNet: Summaries of Genes and Loci Causing Retinal Diseases. RetNet. https://sph.uth.edu/retnet/sum-dis.htm. Updated January 4, 2019. Accessed January 9, 2019.
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.
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