Spark Therapeutics announced new preclinical data for SPK-3006, an investigational liver-directed adeno-associated viral (AAV) gene therapy for Pompe disease. This was announced during the late-breakling session of the International Annual Congress of the World Muscle Society. This is a one-time investigational gene therapy that is engineered to use the liver to express the needed enzyme. Positive results have been seen at the 10-month point in mouse studies.
advocacy awards Awareness Breakthrough therapy designation business CAP chaperone clinical trials compassion CORD COVID-19 eLearning Emma Rooney ERT Extraordinary Measures Fabry fundraising Gaucher gene therapy getting treated grants guest blogger home infusions IND Int'l Pompe Day IPA late-onset law living with Pompe meetings Muscular Dystrophy Canada MyNormal newborn screening NIH Orphan Drug Framework Orphanet politics portraits pre-clinical preclinical pregnancy privacy Rare Disease Rare Disease Day video