Modified RNA with pseudouridine that extends in vivo gene therapy and silencing payloads without licensing constraints.
This technology builds RNA and gene-therapy vectors that incorporate pseudouridine (a modified nucleoside) to alter how the RNA behaves in cells. It’s technically significant because pseudouridine-modified RNA is engineered for stronger functional performance than unmodified RNA, enabling more effective gene replacement and transcription silencing from the same delivery format. Previously, using these specific modified-nucleoside RNA compositions and vectors required permission under the patent’s claims.
Launch pseudouridine-modified RNA therapeutics for gene replacement, CRISPR-like silencing, or in vivo protein delivery.
This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules.
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