Company intelligence / Profile preview

Spark Therapeutics

Philadelphia, Pennsylvania, United Statessparktx.com ↗
Ownership
Private
Employees
~800
Development stage
Commercial
01

Overview

Spark Therapeutics has successfully commercialized the first gene therapy for a genetic disease in the U.S. (Luxturna for inherited retinal disease). Their hemophilia B therapy (fidanacogene elaparvovec) received FDA approval in 2024. Other programs are in various clinical stages.

Therapeutic areas
OphthalmologyHematologyRare DiseasesNeurology
Modalities
Gene therapyAAV vectors
Industry
Biotech
02

Drug pipeline

10 assets

voretigene neparvovec

Approved

9 more assets in the full pipeline.

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03

Partnerships

Pfizer (for hemophilia B gene therapy program)Genethon (licensed SPK-3006 construct)

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