Summary
Spark Therapeutics Inc (Spark) is a clinical-stage gene therapy company which provides gene therapies for inherited retinal diseases (IRDs), neurodegenerative diseases, as well as liver diseases. Its pipeline products include SPK-RPE65, SPK-CHM, rhonova, LHON, SPK-FIX, SPK-FVIII and SPK-TPP1. The company’s gene therapy vectors used in its programs are engineered using adeno-associated virus. Spark’s SPK-RPE65 is intentended to cure rare blinding conditions caused by mutations in the RPE65 gene. The company develops gene therapy products for the treatment of debilitating genetic diseases such as inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. It collaborates with pharma and biotech companies for its research and development activities. Spark is headquartered in Philadelphia, Pennsylvania, the US.

Spark Therapeutics Inc Key Recent Developments

Feb 25,2021: Spark Therapeutics further strengthens technical expertise with appointment of Cynthia Pussinen as Chief Technical Officer
Feb 01,2021: Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease
Nov 16,2020: Spark Therapeutics expands visionary leadership with appointment of Michael Retterath as Chief Strategy Officer
Aug 10,2020: Spark Therapeutics deepens drug development expertise in hematology and rare disease with appointment of Gallia G. Levy, M.D., Ph.D., as Chief Medical Officer
Jun 01,2020: Study demonstrating role of IdeS in enabling of gene therapy in the presence of neutralizing anti-AAV antibodies published in Nature Medicine

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