Back to results
US 2026/0125690 A1Published application
Compositions and methods for treating alpha-1 antitrypsin deficiency
AssigneeIntellia Therapeutics, Inc.
InventorsJonathan Douglas Finn, Hon-Ren Huang, Anthony Forget, Xin Xie
Life of this patent
Estimating the term…
Abstract
Compositions and methods for expressing alpha 1 antitrypsin (AAT) in a host cell are provided. Also provided are compositions and methods for treating subjects having alpha 1 antitrypsin deficiency (AATD).
Claims21
-123. (canceled)
A method of expressing heterologous alpha-1 antitrypsin (AAT) in a subject, the method comprising administering to the subject:
- (a) a nucleic acid construct comprising a heterologous AAT protein coding sequence;
- (b) a Cas9 nuclease;
- and
- (c) a guide RNA (gRNA) comprising a sequence that targets the albumin locus at a genomic coordinate listed in Table 1,
- thereby expressing heterologous AAT from the albumin locus of the subject.
2 more elements
A method of introducing a nucleic acid encoding alpha-1 antitrypsin (AAT) to a cell or a population of cells, the method comprising delivering to the cell or population of cells:
- (a) a bidirectional nucleic acid construct comprising:
- (i) a first segment comprising a first coding sequence for an AAT polypeptide;
- and
- (ii) a second segment comprising a reverse complement of a second coding sequence for the AAT polypeptide;
- (b) a Cas9 nuclease;
- and
- (c) a gRNA that targets the albumin locus at a genomic coordinate listed in Table 1.
4 more elements
A bidirectional nucleic acid construct comprising:
- (a) a first segment comprising a first coding sequence for an alpha-1 antitrypsin (AAT) polypeptide;
- and
- (b) a second segment comprising a reverse complement of a second coding sequence for the AAT polypeptide,
- wherein the bidirectional nucleic acid construct does not comprise:
- (i) a promoter that drives the expression of the first coding sequence;
- (ii) a promotor that drives the expression of the second coding sequence;
- and (iii) a homology arm.
4 more elements
Dependencies are inferred from the claim text.