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

State-of-the-art, in-house expertise in vector manufacturing

Innovative scientific and regulatory strategies

Strong commitment to improve patient care

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.

Pipeline

Spark Therapeutics is working to address a range of debilitating genetic diseases, including inherited retinal diseases (IRDs), liver-mediated diseases and neurodegenerative diseases. Each of our investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and our collaborators.

INHERITED RETINAL DISEASES (IRDs)
PRECLINICAL
PHASE 1 / 2
PHASE 3
REGISTRATION
Voretigene Neparvovec: RPE65-mediated IRD
PHASE 3
SPK-7001: Choroideremia
PHASE 1/2
RHO-adRP
PRECLINICAL
Leber hereditary optic neuropathy (LHON)
PRECLINICAL
LIVER-MEDIATED DISEASES
PRECLINICAL
PHASE 1 / 2
PHASE 3
REGISTRATION
SPK-9001: Hemophilia B (in collaboration with Pfizer)
PHASE 1/2
SPK-8011: Hemophilia A
PHASE 1/2
NEURODEGENERATIVE DISEASES
PRECLINICAL
PHASE 1 / 2
PHASE 3
REGISTRATION
CLN2 disease, a form of Batten disease
PRECLINICAL
Huntington’s disease
PRECLINICAL

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