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-mediated diseases such as 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 trials of adeno-associated viral (AAV) vectors in skeletal muscle tissue and the liver; 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.

What is gene therapy?

At Spark Therapeutics, our approach to gene therapy is to address an inherited disease at its root by augmenting, replacing or suppressing the function of a mutated gene. Once the body has the functional genetic material, it should begin to produce the necessary protein or stop making the harmful protein, with the potential of restoring function in the diseased cells and slowing the progression of disease.

To learn more about the mechanics of investigational gene therapy, visit A Shared Vision.