Pseudouridine-modified RNA enables longer-lasting gene therapy and protein delivery without immediate licensing friction.
Pseudouridine (a modified nucleoside) is incorporated into RNA to reduce innate immune sensing while improving stability and translation for in vivo delivery. Technically, replacing uridine with pseudouridine can substantially extend functional RNA half-life compared with unmodified RNA, enabling higher protein expression from smaller doses. Before expiration, deploying this approach in therapies typically required licensing or rights tied to the University of Pennsylvania patent family.
Build pseudouridine-modified mRNA therapeutics for in vivo protein replacement and gene-silencing indications without licensing roadblocks.
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.
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