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 such as hemophilia and lysosomal storage disorders, 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-directed diseases such as hemophilia and lysosomal storage disorders 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.

Retinal Delivery / Inherited Retinal Diseases
DISCOVERY
CANDIDATE OPTIMIZATION / IND-ENABLING
PHASE 1/2
PHASE 3
SPK-7001: Choroideremia
Phase 1/2
Stargardt Disease
Discovery
Liver Delivery / Hemophilia and Lysosomal Storage Disorders
DISCOVERY
CANDIDATE OPTIMIZATION / IND-ENABLING
PHASE 1/2
PHASE 3
Fidanacogene elaparvovec (SPK-9001): Hemophilia B
Phase 3
SPK-8011: Hemophilia A
Phase 3
SPK-8016: Hemophilia A with inhibitors
Phase 1/2
SPK-3006: Pompe Disease
Candidate optimization / IND-Enabling
Undisclosed
Discovery
Central Nervous System Delivery / Neurodegenerative Diseases
DISCOVERY
CANDIDATE OPTIMIZATION / IND-ENABLING
PHASE 1/2
PHASE 3
SPK-1001: CLN2 Disease (a form of Batten Disease)
Candidate optimization / IND-Enabling
Huntington’s Disease
Candidate optimization / IND-Enabling
Undisclosed
Candidate optimization / IND-Enabling
Undisclosed
Discovery

A journey through a history of selected gene therapy milestones

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