Spark Therapeutics

sparktx.com

Spark Therapeutics is a fully integrated company focused on gene therapy, combining proprietary adeno-associated viral (AAV) technology with R&D, manufacturing, and commercial operations. They aim to transform healthcare by developing potentially curative, one-time gene therapy products for inherited diseases, including inherited retinal diseases, hemophilia, and neurodegenerative diseases. Their robust pipeline includes pivotal programs like SPK-RPE65 for blindness and SPK-FIX for hemophilia B, supported by a proprietary manufacturing platform and a strong commitment to improving patient care.