Spark Therapeutics, Inc. announced the presentation of preclinical data for SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease, a form of Batten disease, on February 6, 2019, at the 15th Annual WORLDSymposium™ in Orlando, FL. SPK-3006 is an investigational liver-directed AAV gene therapy for Pompe disease. SPK-3006 has been engineered to produce a modified enzyme that is secreted from the liver, which may sustain GAA plasma levels and lower immunogenicity to GAA to potentially provide greater uptake in muscle tissue. The transgene was in-licensed in 2017 from Genethon, a non-profit research and development organization dedicated to the development of gene therapies for orphan genetic diseases from research to clinical validation. Spark Therapeutics retains global commercialization rights to SPK-3006. SPK-1001 is an investigational central nervous system (CNS)-directed AAV gene therapy that has demonstrated compelling preclinical proof-of-concept in a naturally occurring model of TPP1 deficiency, a form of Batten disease. Batten disease is a fatal neurological disorder involving mutations of the TPP1 gene that begins in early childhood. Spark Therapeutics has received orphan drug designation from the U.S. Food and Drug Administration for SPK-1001 for the treatment of CNL2 disease [neuronal ceroid lipofuscinosis (NCL)] caused by TPP1 deficiency. Spark Therapeutics retains global rights to SPK-1001.