Spark Reports Promising Preclinical Results for Neurodegenerative Disease Gene Therapy

Gene therapy company Spark Therapeutics has achieved promising results in preclinical experiments for treating Batten disease, a fatal, juvenile neurodegenerative disorder. Spark’s therapy called SPK-TPP1 is recombinant adeno-associated viral (AAV) vector that expresses the deficient protein in forms of the disease caused by mutations in a lysosomal protease called tripeptidyl peptidase I (TPP1). In collaboration with Beverly Davidson and colleagues from the Children’s Hospital of Philadelphia, intraventricular delivery of a single dose of SPK-TPP1 in a canine model of the disease increased TPP1 expression through the CNS, delayed disease onset and progression, prolonged lifespan, and decreased cognitive deficits. The results were published in the Nov 11 Science Translational Medicine and represent another preclinical proof-of-concept for an effective gene therapy for neurodegenerative diseases. Gene therapy approaches targeting SOD1 and C9ORF72 are also under development for ALS (see Nov 2015 news).

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Katz ML, Tecedor L, Chen Y, Williamson BG, Lysenko E, Wininger FA, Young WM, Johnson GC, Whiting RE, Coates JR, Davidson BL. AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease. Sci Transl Med. 2015 Nov 11;7(313):313ra180.
batten disease gene therapy topic-preclinical topic-randd tpp1
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