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US 2024/0200118 A1Published application
Compositions and methods for optimizing tropism of delivery systems for rna
AssigneeReNAgade Therapeutics Management Inc.
InventorsInna SHCHERBAKOVA, Grace CHEN, Abril Fleitas BEITANS, Brian GOODMAN, Ciaran LAWLOR, Kevin Yingxin YANG
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Abstract
The present disclosure details various compositions and/or methods of optimizing delivery systems the localized delivery of nucleic acid sequences, polypeptides or peptides for use in therapeutics and/or diagnostics.
Claims125
A method for determining the biodistribution of one or more pharmaceutical delivery vehicles upon administration to a subject, the method comprising:
- a) formulating the one or more pharmaceutical delivery vehicles to comprise a first engineered polynucleotide comprising:
- i) optionally a payload sequence region comprising a coding nucleic acid sequence or non-coding nucleic acid sequence;
- ii) a unique nucleotide identifier sequence;
- iii) at least one flanking sequence region located at one or more locations selected from upstream of the unique nucleotide identifier sequence and downstream of the unique nucleotide identifier sequence;
- and
- iv) optionally, at least one regulatory sequence region;
- b) administering the one or more pharmaceutical delivery vehicles comprising the first engineered polynucleotide to a subject;
- c) collecting at least one sample from the subject at one or more time points after administration of the one or more pharmaceutical delivery vehicles comprising the engineered polynucleotide;
- and
- d) analyzing the at least one sample to determine the amount of the one or more pharmaceutical delivery vehicles present in the at least one sample by measuring the amount of the unique nucleotide identifier sequence present in the sample;
- wherein, when more than one distinct pharmaceutical delivery vehicle is administered to the subject, each distinct pharmaceutical delivery vehicle comprises a distinct engineered polynucleotide comprising a different unique nucleotide identifier sequence.
8 more elements
An engineered polynucleotide comprising:
- a) a payload sequence region comprising a coding nucleic acid sequence or non-coding nucleic acid sequence;
- b) a unique nucleotide identifier sequence that is a barcode sequence;
- c) at least one flanking sequence region located at one or more locations selected from upstream of the barcode sequence and downstream of the barcode sequence;
- and
- d) optionally, at least one regulatory sequence region.
2 more elements
A method for determining the biodistribution of one or more pharmaceutical delivery vehicles upon administration to a subject, the method comprising:
- a) formulating the one or more pharmaceutical delivery vehicles to comprise a first engineered polynucleotide comprising:
- i) a unique nucleotide identifier sequence that is a barcode sequence;
- ii) at least one flanking sequence region located at one or more locations selected from upstream of the unique identifier sequence and downstream of the barcode sequence;
- and
- iii) optionally, at least one regulatory sequence region;
- and a second engineered polynucleotide comprising a payload sequence region comprising a coding nucleic acid sequence or a non-coding nucleic acid sequence;
- b) administering the one or more pharmaceutical delivery vehicles comprising the first and second engineered polynucleotides to a subject;
- c) collecting at least one sample from the subject at one or more time points after administration of the one or more pharmaceutical delivery vehicles comprising the engineered polynucleotide;
- and
- d) analyzing the at least one sample to determine the amount of the one or more pharmaceutical delivery vehicles present in the at least one sample by measuring the amount of unique identifier sequence(s) present in the sample;
- wherein, when more than one distinct pharmaceutical delivery vehicle is administered to the subject, each distinct pharmaceutical delivery vehicle comprises a distinct first engineered polynucleotide comprising a different unique identifier sequence.
8 more elements
Dependencies are inferred from the claim text.