US 12,270,029 B2 · CFTR-modulating compositions and methods

CFTR-modulating compositions and methods

AssigneeFlagship Pioneering Innovations VI, LLC

InventorsRobert Charles Altshuler, Anne Helen Bothmer, Daniel Raymond Chee, Cecilia Giovanna Silvia Cotta-Ramusino, Kyusik Kim, Randi Michelle Kotlar, Gregory David McAllister, Aamir Mir, Ananya Ray, Nathaniel Roquet, Carlos Sanchez, Barrett Ethan Steinberg, Robert James Citorik, William Edward Salomon, William Querbes, Luciano Henrique Apponi, Zhan Wang

Life of this patent

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Abstract

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.

Claims15

Dependent claims

A template RNA comprising the RNA sequence of SEQ ID NO:

19,479.

Dependencies are inferred from the claim text.

Facts

Application

18/470,687Filed 20 Sep 2023 · continuation

Priority

10 Dec 2021

Published

US 2024/0093186 A121 Mar 2024

Granted

8 Apr 2025

Term ends

Not estimatedThe source records don’t support an estimate.

Classification21 codes

C12N 15/11
Recombinant DNA-technology: dNA or RNA fragments; Modified forms thereof ; Non-coding nucleic acids having a biological activity
A61K 48/005
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy:…
C07K 14/4712
Not used: cystic fibrosis
C12N 9/1276
Nucleotidyltransferases: rNA-directed DNA polymerase , i.e. reverse transcriptase or telomerase

Family

Application63/288,474Us provisional application · filed 10 Dec 2021

Application63/303,941Us provisional application · filed 27 Jan 2022

Application63/374,832Us provisional application · filed 7 Sep 2022

PCT/US2022/081316Parent of this continuation · filed 9 Dec 2022

US 2024/0093186 A1Pre-grant publication · published 21 Mar 2024

US 12,270,029 B2This patent

From the US filing history. Foreign family members aren’t listed.