Compositions and methods for the treatment of neuromuscular disorders
AssigneeAstellas Gene Therapies, Inc.
InventorsCarlos FONCK, Clarice Weili CHEN, Dwaipayan SEN
Life of this patent
Estimating the term…
Abstract
Some embodiments relate to adeno-associated virus (AAV)-mediated delivery of nucleic acids for treating neuromuscular disorders, e.g., X-linked myotubular myopathy (XLMTM), in patients in need thereof. The AAV vectors of the disclosure may include, e.g., a transgene encoding a myotubularin protein operably linked to one or more transcription regulatory elements. In some embodiments, the one or more transcription regulatory elements are specifically active in muscle and/or liver tissue.
Claims91
A recombinant adeno-associated viral (AAV) vector comprising a transgene encoding myotubularin 1 (MTM1), wherein the transgene is operably linked to a promoter that is active in liver tissue.
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
A method of treating XLMTM in a human patient in need thereof, the method comprising administering to the patient therapeutically effective amounts of:
- (i) a non-viral composition comprising a nucleic acid encoding MTM1 operably linked to a promoter that is active (e.g., selectively active) in liver tissue, and (ii) an AAV vector comprising a transgene encoding MTM1 under the control of a promoter that is active (e.g., selectively active) in muscle tissue.
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
A kit comprising:
- (i) a non-viral composition comprising a nucleic acid encoding MTM1, and (ii) an AAV vector comprising a transgene encoding MTM1 under the control of a promoter that is active (e.g., selectively active) in muscle tissue, wherein the kit further comprises a package insert instructing a user to administer the non-viral composition and the AAV vector to a patient diagnosed as having XLMTM.
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
Dependencies are inferred from the claim text.