Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
Inventors
Sah, Dinah Wen-Yee • Goulet, Martin • Patzke, Holger • Shu, Yanqun • Hou, Jinzhao • PUCCIO, Hélène
Assignees
Centre National de la Recherche Scientifique CNRS • Institut National de la Sante et de la Recherche Medicale INSERM • Universite de Strasbourg • Voyager Therapeutics Inc
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Publication Number
US-12146150-B2
Publication Date
2024-11-19
Expiration Date
Abstract
Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.
Core Innovation
The invention relates to a method of delivering a payload to a tissue of the central nervous system (CNS) in a mammalian subject by administering a first adeno-associated virus (AAV) particle by intravenous (IV) administration. The first AAV particle comprises an AAV capsid with the amino acid sequence of SEQ ID NO: 2 and a viral genome comprising at least one inverted terminal repeat (ITR) and a polynucleotide sequence encoding the payload.
The approach is further characterized by delivering a payload in association with viral-genome structural elements that include one or more inverted terminal repeats (ITR). In additional refinements, the viral genome includes polynucleotide sequence elements encoding microRNA binding sites, including miRNA-122 binding sites and copy-number variants as defined in dependent claim features.
A specific payload is described as frataxin, including embodiments in which the payload comprises a frataxin protein. The invention also includes compositions as AAV particles comprising the capsid sequence of SEQ ID NO: 2 and a viral genome containing at least one ITR and a payload-encoding polynucleotide sequence, with the payload specified to comprise frataxin in dependent scope.
Claims Coverage
The document includes two independent claims: a method and a composition. Across the independent claims, there are three central inventive elements: the SEQ ID NO: 2 AAV capsid, the viral genome architecture with at least one ITR plus a payload-encoding polynucleotide, and, for the method claim, IV delivery to CNS tissue in a mammalian subject.
Intravenous AAV delivery to CNS tissue with SEQ ID NO: 2 capsid
The method comprises administering a first adeno-associated virus (AAV) particle by intravenous (IV) administration to a mammalian subject for delivering a payload to a tissue of the central nervous system (CNS), wherein the first AAV particle comprises an AAV capsid comprising the amino acid sequence of SEQ ID NO: 2.
Viral genome with ITRs and payload-encoding polynucleotide
The method is characterized by a first AAV particle having a viral genome comprising at least one inverted terminal repeat (ITR) and a polynucleotide sequence encoding the payload.
AAV particle composition with SEQ ID NO: 2 capsid and ITR/payload genome
An AAV particle comprising an AAV capsid and viral genome, where the AAV capsid comprises the amino acid sequence of SEQ ID NO: 2 and the viral genome comprises at least one inverted terminal repeat (ITR) and a polynucleotide sequence encoding a payload.
Overall, the claim coverage anchors the invention in AAV particles defined by the SEQ ID NO: 2 capsid and a viral genome containing at least one ITR and a payload-encoding polynucleotide, with the method claim further requiring IV administration for CNS-tissue payload delivery. Dependent features refine payload composition (frataxin) and genome elements such as microRNA binding sites, including miRNA-122 binding-site embodiments.
Stated Advantages
Not explicitly described in patent.
Documented Applications
Not explicitly described in patent.
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