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

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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
LUXTURNA™ (voretigene neparvovec): biallelic RPE65-mediated IRD*
PHASE 3
SPK-7001: Choroideremia
PHASE 1/2
Leber hereditary optic neuropathy (LHON)
PRECLINICAL
Undisclosed
PRECLINICAL
LIVER-MEDIATED DISEASES
PRECLINICAL
PHASE 1 / 2
PHASE 3
REGISTRATION
SPK-8011: Hemophilia A
PHASE 1/2
SPK-9001: Hemophilia B (in collaboration with Pfizer)
PHASE 1/2
Undisclosed
PRECLINICAL
NEURODEGENERATIVE DISEASES
PRECLINICAL
PHASE 1 / 2
PHASE 3
REGISTRATION
CLN2 disease, a form of Batten disease
PRECLINICAL
Huntington’s disease
PRECLINICAL

* BLA filing under review in the U.S. and MAA submitted in the EU

P-SPK-ALL-790055