Our Scientific Platform and Programs
Spark® Therapeutics has built a leading integrated gene therapy platform as we strive to turn genes into medicines for patients with inherited diseases, including inherited retinal diseases (IRDs), liver-directed diseases including hemophilia, and neurodegenerative diseases. Learn more about our platform below.
Cutting-edge vector
design
We are advancing research programs against cell targets in the retina, liver, and central nervous system using adeno-associated viral (AAV) vectors. Our viral vectors are designed to encapsulate genetic material for the potential treatment of genetic diseases. The vectors used in our research programs have been engineered using Spark’s cutting-edge proprietary platform, selected through vigorous preclinical testing and validated in several clinical trials.
State-of-the-art, in-house expertise in vector manufacturing
With adeno-associated viral (AAV) vector manufacturing capabilities in-house, investigational clinical-grade vectors developed and manufactured by our team have been delivered through six routes of administration to hundreds of patients in more than a dozen clinical trials.
Innovative scientific and regulatory strategies
We work in a spirit of collaboration with investigators and regulators to develop rigorous and novel endpoints that measure the potential impact of our investigational, potentially one-time therapies on targeted diseases and to help shape high standards for future gene therapies. At the forefront of gene therapy research for more than two decades, members of our scientific team are responsible for numerous development milestones, including the first clinical studies to evaluate AAV administration to the second eye and the first gene therapy trial for a non-lethal disorder that included pediatric participants.
Strong commitment to improve patient care
We are committed to developing an approach to patient access tailored to the unique nature of an investigational, potentially one-time, life-altering gene therapy. We are providing education and other resources to promote a shift from clinical to genetic-based disease classification, and advocating for patient access to care and incentives for innovation.
Get our news alerts delivered straight to your inbox
Sign Up Here
Our Research
Ocular
Spark Therapeutics is researching potential gene therapies for inherited ocular conditions.
Liver-Directed Therapies
Spark Therapeutics is researching liver-directed therapies to address a range of diseases.
Neurodegenerative Diseases (Central Nervous System Delivery)
Spark Therapeutics is researching neurodegenerative gene therapies to address a range of diseases including Huntington’s disease.
Other areas of interest
Along with our ongoing research across therapeutic areas, we are continuing to conduct research related to the immune system, targeted gene therapy deliveries, and enabling technologies to optimize delivery and potentially expand access for eligible patient populations.
Additionally, we are continuing to build strategic partnerships to help accelerate our progress for patients. If you have an opportunity for collaboration or an interesting technology, or if you have an asset in one of our areas of interest, we would like to hear from you. For inquiries or to submit a proposal, visit our Business Development page.
Spark® Therapeutics, Inc.
3737 Market Street
Philadelphia, PA 19104
Phone: 1-855-SPARKTX / +1 215-220-9300
N-SPK-US-700011-17
