Benitec IP Holdings, Inc.

Vanessa Strings-Ufombah

David A Suhy

Shih-Chu Kao

Petrus W Roelvink

The present disclosure relates to RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR) and short hairpin RNA (shRNA), for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto. The present disclosure also relates to the use of the RNAi reagents in combination with PABPN1 replacment reagents, such as constructs which encode functional PABPN1 protein, for treatment of OPMD, compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.

Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof

The present disclosure relates generally to modified adeno-associated virus (AAV) from serotypes other than serotype 2, which have a viral capsid protein with a subunit 1 (VP1) sequence which is modified relative to the corresponding wildtype sequence. In particular, the modified AAVs of the disclosure comprise site-specific amino acid substitutions within the phospholipase A2 (PLA2) domain and flanking sequence relative to the corresponding wild-type sequence which improve functionality of the AAV when produced in insect cells. The present disclosure also relates to methods of producing the modified AAVs, reagents therefor, baculovirus expression systems and insect cells for producing said modified AAVs.

Adeno-associated virus (AAV) with modified phospholipase domain

The present disclosure relates to RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR) and short hairpin RNA (shRNA), for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto. The present disclosure also relates to the use of the RNAi reagents in combination with PABPN1 replacement reagents, such as constructs which encode functional PABPN1 protein, for treatment of OPMD, compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.

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Hayward, CA, United States of America

Vanessa Strings-Ufombah