Flagship Pioneering Innovations Vi, LLC

William Edward Salomon

Barrett Ethan Steinberg

Robert James Citorik

Jacob Rosenblum Rubens

Zi Jun Wang

Anne Helen Bothmer

Randi Michelle Kotlar

Nathaniel Roquet

Cecilia Giovanna Silvia Cotta-Ramusino

Ananya Ray

Robert Charles Altshuler

Kyusik Kim

Geoffrey Von Maltzahn

Stephen Hoyt Cleaver

Donghui Li

Sandeep Kumar

Carlos Sanchez

Daniel Raymond Chee

Aamir Mir

Gregory David McAllister

Zhan Wang

William Querbes

Luciano Henrique Apponi

Methods and compositions for modulating a target genome are disclosed. This disclosure relates to novel compositions, systems and methods for altering a genome at one or more locations in a host cell, tissue or subject, in vivo or in vitro. In particular, the invention features compositions, systems and methods for inserting, altering, or deleting sequences of interest in a host genome.

Methods and compositions for modulating a genome

Methods and compositions for modulating a target genome are disclosed. The composition may comprise a first RNA encoding a polypeptide comprising a retrotransposase reverse transcriptase domain and a retrotransposase endonuclease domain. The composition may also comprise a second RNA comprising a sequence that binds the polypeptide and a heterologous object sequence. The composition may insert the sequence of the heterologous object sequence into a target DNA.

The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject. Gene modifying systems for treating cystic fibrosis, e.g., in subjects having a mutation resulting in F508del, are described.

CFTR-modulating compositions and methods

Methods and compositions for modulating a target genome are disclosed.

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West Roxbury, MA, United States of America

William Edward Salomon