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Rana Therapeutics Inc patents

Recent patent applications related to Rana Therapeutics Inc. Rana Therapeutics Inc is listed as an Agent/Assignee. Note: Rana Therapeutics Inc may have other listings under different names/spellings. We're not affiliated with Rana Therapeutics Inc, we're just tracking patents.

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Cftr mrna compositions and related methods and uses

Materials, formulations, production methods, and methods for delivery of cftr mrna for induction of cftr expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.. ... Rana Therapeutics Inc

Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency

The present invention provides, among other things, methods of treating ornithine transcarbamylase (otc) deficiency, including administering to a subject in need of treatment a composition comprising an mrna encoding an ornithine transcarbamylase (otc) protein at an effective dose and an administration interval such that at least one symptom or feature of the otc deficiency is reduced in intensity, severity, or frequency or has delayed onset. In some embodiments, the mrna is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more peg-modified lipids.. ... Rana Therapeutics Inc

Encapsulation of messenger rna

The present invention provides an improved process for lipid nanoparticle formulation and mrna encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger rna (mrna) in lipid nanoparticles comprising a step of mixing a mrna solution and a lipid solution, wherein the mrna solution and/or the lipid solution are at a pre-determined temperature greater than ambient temperature.. ... Rana Therapeutics Inc

Lipid nanoparticle compositions and methods for mrna delivery

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.. ... Rana Therapeutics Inc

Multimeric oligonucleotide compounds

The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (asos)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. ... Rana Therapeutics Inc

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