Recode Therapeutics, Inc.

Daniella Ishimaru

Mirko Hennig

David J Lockhart

Brandon A Wustman

Yufeng Wang

Sakya Sing Mohapatra

Vladimir Grigor'evich Kharitonov

Shuang Li

Julia Jung-un Baek

Ali Ahmed Alfaifi

Joseph S Cefalu

Jackson Eby

Michael Torres

Dmitri Boudko

Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.

Lipid nanoparticle compositions and uses thereof

Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may encode for a cystic fibrosis transmembrane conductance regulator (CFTR) protein or a functional fragment thereof. The polynucleotide may be assembled with a lipid composition for targeted delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of CFTR protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.

Polynucleotide compositions, related formulations, and methods of use thereof

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Mountain View, CA, United States of America

Daniella Ishimaru