US2022025335A1PendingUtilityA1
Muscular dystrophy chimeric cells and method for treating muscular dystrophies
Est. expiryJun 11, 2035(~8.9 yrs left)· nominal 20-yr term from priority
Inventors:Maria Siemionow
A61P 21/00C12N 5/16A61P 25/14C12N 5/0663A61K 35/34C12N 2510/00A61K 45/06C12N 5/0658A61K 9/0019A61K 35/28
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Claims
Abstract
A Muscular Dystrophy Chimeric Cell generated by ex vivo fusion of a myoblast with a second myoblast, mesenchymal stem cell, or stromal cell is described as is the use of the same in the treatment of a muscular dystrophy.
Claims
exact text as granted — not AI-modifiedWhat is claimed is:
1 . An isolated Muscular Dystrophy Chimeric Cell (MDCC) population comprising cells produced by ex vivo fusion and expansion of
(a) a first myoblast; and (b) a second myoblast, mesenchymal stem cell, or stromal cell, wherein at least one of the first myoblast, second myoblast, mesenchymal stem cell, or stromal cell is from a healthy donor and the other is a dystrophin deficient cell from subject suffering from a muscular dystrophy.
2 . The MDCC of claim 1 , wherein the first myoblast and second myoblast are from different donors.
3 . The isolated MDCC of claim 1 , wherein the first myoblast, second myoblast, mesenchymal stem cell, or stromal cell is autologous or allogeneic.
4 . The isolated MDCC of claim 1 , wherein the healthy donor is the subject's father.
5 . The isolated MDCC of claim 1 , wherein the mesenchymal stem cells are derived from bone marrow or adipose tissue.
6 . The isolated MDCC of claim 1 , wherein said MDCC secretes one or more immunomodulatory cytokines and growth factors.
7 . The isolated MDCC of claim 6 , wherein the one or more immunomodulatory cytokines and growth factors comprise insulin-like growth factor 1, hepatocyte growth factor and myostatin.
8 . The isolated MDCC of claim 1 , wherein said an ex vivo fusion is between:
a) a dystrophin deficient human myoblast from a subject suffering from a muscular dystrophy and a human myoblast from a healthy donor; b) a dystrophin deficient human myoblast from a subject suffering from a muscular dystrophy and a human mesenchymal stem cell from a healthy donor; or c) a human myoblast from a healthy donor and a dystrophin deficient human mesenchymal stem cell from a subject suffering from a muscular dystrophy; or d) a dystrophin deficient human myoblast from a subject suffering from a muscular dystrophy and a human stromal cell from a healthy donor.
9 . A composition comprising the isolated MDCC of claim 1 and a pharmaceutically acceptable carrier.
10 . A method of treating a muscular dystrophy comprising administering to the subject in need of treatment an effective amount of the isolated MDCC of claim 1 thereby treating the subject's muscular dystrophy.
11 . The method of claim 10 , wherein the muscular dystrophy is Duchenne Muscular Dystrophy.
12 . The method of claim 10 , wherein said MDCC is administered by intravenous injection, intra-bone injection or intramuscular injection.Join the waitlist — get patent alerts
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