GENE THERAPY FOR NEURONAL CEROID LIPOFUSCINOSES

• Main Authors: GUIBERT, Matthieu Pierre, CARRELL, Michael James, BUSS, Nicholas Alexander Piers Sascha, GERNER, Franz, Michael, DEPAZ, Roberto, BEE, Jared, MERCER, Andrew Christopher, STADELMAN, Robert Thomas, KONDRAGUNTA, Bhargavi, MELETHIL, Subha Karumuthil, MARSHALL, Tristan James
• Format: Patent
• Language: English; French; German
• Published: 10.08.2022
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR. Also provided herein are methods of treating Batten disease comprising administering to a subject in need thereof the rAAV described herein via more than one route. Also provide herein are pharmaceutical compositions comprising the rAAV described herein and related methods of treating Batten disease.