IP Library Granted Patent US 12,544,409
Granted Patent B2
US 12,544,409 · App. 17/528,822 · Granted Feb 10, 2026

Cardiosphere-derived cells and exosomes secreted by such cells in the treatment of muscular dystrophy

Inventors: Eduardo Marban (Santa Monica, CA); Mark Amin Aminzadeh (Los Angeles, CA)
Assignee: Cedars-Sinai Medical Center
A61K35/34A61K9/0019A61K31/713A61K38/1709A61P9/10A61P21/00C12N15/113C12N2310/141C12N2320/31C12N2320/32
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Quick Facts
Patent No.
US 12,544,409
App. No.
17/528,822
Granted
Feb 10, 2026
Kind
B2
Abstract

Described herein are compositions and techniques related to generation and therapeutic application of cardiosphere-derived cells (CDCs) and CDC-derived exosomes. These cells and their secreted vesicles contain a unique milieu of biological factors, including cytokines, growth factors, transcription factors, nucleic acids including non-coding nucleic acids such as microRNAs, that serve to initiate and promote many therapeutic effects. Exosomes and their “cargo” contents, such as microRNAs can favorably modulate apoptosis, inflammation and fibrosis in the injured heart. Thus, CDC-derived exosomes represent a novel “cellfree” therapeutic candidate for tissue repair.

Claims (24)

1 . A method of reducing oxidative stress and inflammation in the heart of a subject with muscular dystrophy, comprising:

selecting a subject having muscular dystrophy who is in need of treatment;

obtaining a composition comprising cardiosphere-derived cells; and

intravenously administering the composition comprising cardiosphere-derived cells to the subject;

wherein composition comprising cardiosphere-derived cells comprises a therapeutic amount of cardiosphere-derived cells; and

wherein administration of the composition comprising cardiosphere-derived cells to the subject reverses NFκB activation or decreases inflammatory cell number in the heart of the subject.

2 . The method of claim 1 , wherein a dose of the composition comprising cardiosphere-derived cells comprises 1×10 7 to 1×10 9 cardiosphere-derived cells.

3 . The method of claim 1 , wherein a single dose is administered multiple times to the subject.

4 . The method of claim 3 , wherein the single dose of the composition comprising cardiosphere-derived cells comprises 1×10 5 to 1×10 9 cardiosphere-derived cells.

5 . The method of claim 1 , wherein said administering of said composition to the subject results in one or more of decreased fibrosis, decreased inflammation, increased mitochondrial function, or increased cardiomyogenesis.

6 . The method of claim 5 , wherein the decreased fibrosis comprises a reduction in collagen accumulation.

7 . The method of claim 5 , wherein the decreased inflammation comprises an increase in cytoplasmic nuclear factor (erythroid-derived 2)-like 2 (Nrf2), reduction in fatty acid peroxidation end products, reduced numbers of inflammatory cells, and/or upregulated expression of antioxidants.

8 . The method of claim 7 , wherein the antioxidants comprise heme oxygenase-1 (HO-1), catalase, superoxide dismutase-2 (SOD-2), or glutamate-cystein ligase catalytic (GCLC) subunit.

9 . The method of claim 5 , wherein the increased mitochondrial function comprises increased mitochondrial ultrastructure or increased mitochondrial biogenesis.

10 . The method of claim 1 , wherein the muscular dystrophy is Duchenne muscular dystrophy.

11 . A method of reducing oxidative stress and inflammation in the heart of a subject with Duchenne muscular dystrophy, comprising:

selecting a subject with Duchenne muscular dystrophy in need of treatment; and

intravenously administering a composition comprising cardiosphere-derived cells to the subject, thereby treating the subject;

wherein composition comprises a therapeutic amount of cardiosphere-derived cells.

12 . The method of claim 11 , wherein administering the composition comprises providing one or more doses of about 1×10 5 to about 1×10 8 or more cardiosphere-derived cells to the subject.

13 . The method of claim 11 , wherein said administering of said composition to the subject results in one or more of decreased fibrosis, decreased inflammation, increased mitochondrial function, or increased cardiomyogenesis.

14 . The method of claim 13 , wherein the decreased fibrosis comprises a reduction in collagen accumulation.

15 . The method of claim 13 , wherein the decreased inflammation comprises an increase in cytoplasmic nuclear factor (erythroid-derived 2)-like 2 (Nrf2), reduction in fatty acid peroxidation end products, reduced numbers of inflammatory cells, or upregulated expression of antioxidants.

16 . The method of claim 15 , wherein the antioxidants comprise heme oxygenase-1 (HO-1), catalase, superoxide dismutase-2 (SOD-2), and/or glutamate-cystein ligase catalytic (GCLC) subunit.

Assignments (2)
CONFIRMATORY LICENSE Recorded Aug 29, 2025
From: CEDARS-SINAI MEDICAL CENTER
To: NATIONAL INSTITUTE OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 072771/0011 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 29, 2025
From: MARBAN, EDUARDO; AMINZADEH, MARK AMIN
To: CEDARS-SINAI MEDICAL CENTER
Reel/Frame 072771/0043 →
Continuity (3)
Continuation 15516658
Provisional Application 62059308 · Oct 3, 2014
Related Publication 20220072062A1 · Mar 10, 2022
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