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1. 发明申请

WO2021188899A1 CARDIOSPHERE-DERIVED CELL (CDC) THERAPY FOR THE TREATMENT OF VIRAL INFECTIONS 审中-公开
公开(公告)号：WO2021188899A1
公开(公告)日：2021-09-23
申请号：PCT/US2021/023165
申请日：2021-03-19
申请人： CEDARS-SINAI MEDICAL CENTER , CAPRICOR, INC.
发明人： MARBÁN, Eduardo , MARBÁN, Linda
IPC分类号： A61K35/34 , A61K35/545
摘要： Several embodiments of the methods and compositions disclosed herein relate to methods of treating viral infections, such as those caused by coronaviruses. In some embodiments, CDCs are administered to a patient, to treat the viral infection. In some embodiments, CDC-derived exosomes are administered to a patient. In some embodiments combinations of CDCs and CDC-derived exosomes are used. In still additional embodiments, combination therapies, such as CDCs or CDC-derived exosomes in combination with another therapeutic, such as an anti-inflammotry or other immune modulator are used to treat viral infections. In some embodiments, the viral infection is COVID-19, which is caused by SARS-CoV-2.

2. 发明申请

WO2018195210A1 METHODS AND COMPOSITIONS FOR TREATING SKELETAL MUSCULAR DYSTROPHY 审中-公开
公开(公告)号：WO2018195210A1
公开(公告)日：2018-10-25
申请号：PCT/US2018/028184
申请日：2018-04-18
申请人： CEDARS-SINAI MEDICAL CENTER , CAPRICOR, INC.
发明人： MARBÁN, Eduardo , AMINZADEH, Mark, A. , ROGERS, Russell , MOSELEY, Jennifer , RODRIGUEZ-BORLADO, Luis , KANAGAVELU, Saravana , SAKODA, Christopher, Steward
IPC分类号： A61K31/713 , A61K35/34 , C12N15/113
摘要： Some embodiments provide a method of treating skeletal muscular myopathy, e.g., Duchenne muscular dystrophy (DMD), with cardiosphere-derived cells (CDCs), wherein a therapeutically effective amount of CDCs is delivered to a targeted dystrophic skeletal muscle. Some embodiment enable delivery of a therapeutically effective amount of CDCs via intramuscular injection directly at a skeletal muscle or systemic administration, e.g., intravenous injection, in a single dose or multiple doses, to treat a targeted dystrophic skeletal muscle. Some embodiments provide a method for improving exercise capabilities in DMD patients. Additional embodiments relate to exosome mediated transfer of noncoding RNAs ameliorates Duchenne muscular dystrophy by restoring dystrophin in heart and skeletal muscle. Delivery of noncoding RNA species found in CDC-derived exosomes mimics the ability of CDCs and CDC-derived exosomes to increase dystrophin protein levels.

3. 发明申请

WO2013166281A1 BI-FUNCTIONAL COMPOSITIONS FOR TARGETING CELLS TO DISEASED TISSUES AND METHODS OF USING SAME 审中-公开
标题翻译：用于将细胞靶向疾病组织的双功能组合物及其使用方法
公开(公告)号：WO2013166281A1
公开(公告)日：2013-11-07
申请号：PCT/US2013/039255
申请日：2013-05-02
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , CHANG, Ke
IPC分类号： A61K33/00
CPC分类号： A61K49/1875 , A61K47/6923 , B82Y5/00
摘要： Disclosed herein are compositions and methods for the targeted delivery of therapeutic cells to a target tissue. In several embodiments, the therapeutic cells are captured by an antibody that is coupled to a magnetic particle, which is in turn coupled to an antibody directed against a specific marker expressed by a target tissue. In some embodiments, the therapeutic cells comprise the target tissue is damaged or diseased cardiac tissue. In several embodiments, in conjunction with an applied magnetic field, the methods, in combination with the compositions, yield enhanced delivery, of the therapeutic cells to the target tissue, thereby resulting in repair and/or regeneration of the target tissue. Also disclosed are methods for the non-invasive detection of immune responses to transplanted cells or organs.
摘要翻译：本文公开了将靶向组织靶向递送治疗细胞的组合物和方法。在几个实施方案中，治疗细胞被与偶联到磁性颗粒的抗体捕获，抗体依次与针对靶组织表达的特异性标记的抗体偶联。在一些实施方案中，治疗性细胞包含靶组织被损坏或患病的心脏组织。在几个实施方案中，结合施加的磁场，与组合物组合的方法产生治疗细胞到目标组织的增强的递送，从而导致靶组织的修复和/或再生。还公开了用于非侵入性检测对移植的细胞或器官的免疫应答的方法。

4. 发明申请

WO2019126068A1 ENGINEERED EXTRACELLULAR VESICLES FOR ENHANCED TISSUE DELIVERY 审中-公开
公开(公告)号：WO2019126068A1
公开(公告)日：2019-06-27
申请号：PCT/US2018/066072
申请日：2018-12-17
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , ANTES, Travis
IPC分类号： A61K9/51 , A61P35/00 , A61K45/00
摘要： Several embodiments relate to engineered extracellular vesicles (EVs) using the membrane cloaking platform technology described herein, the cloaking imparting to the EVs enhanced delivery to tissues of interest, such as damaged or dysfunctional tissue. Several embodiments relate to engineering exosomes derived from cardiosphere-derived cells (CDCs) using the membrane cloaking platform technology described herein to confer enhanced tissue homing specificities, thereby leading to repair and regeneration at sites of injury. Uses of engineered EV compositions to treat diseases are also provided for in several embodiments.

5. 发明申请

WO2021178514A1 CARDIOSPHERE-DERIVED CELLS, EXOSOMES DERIVED THEREFROM, AND METHODS OF USING SAME TO TREAT VOLUMETRIC MUSCLE LOSS 审中-公开
公开(公告)号：WO2021178514A1
公开(公告)日：2021-09-10
申请号：PCT/US2021/020640
申请日：2021-03-03
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , ROGERS, Russell
IPC分类号： A61K35/34 , A61P21/00 , C12N5/077
摘要： Provided herein are methods of treating a traumatic muscle injury in a subject in need thereof, by administering a therapeutically effective amount of cardiosphere-derived cells (CDCs) or CDC-derived exosomes to the subject. In certain embodiments, CDCs or CDC-derived exosomes are administered intravenously or intramuscularly. In certain embodiments, the CDC-derived exosomes are derived from immortalized CDCs or primary human CDCs.

6. 发明申请

WO2019152549A1 METHODS FOR THERAPEUTIC USE OF EXOSOMES AND Y-RNAS 审中-公开
公开(公告)号：WO2019152549A1
公开(公告)日：2019-08-08
申请号：PCT/US2019/015895
申请日：2019-01-30
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , CAMBIER, Linda , DE COUTO, Geoffrey
IPC分类号： A61K38/00 , A61P9/10 , A61P21/00
摘要： Some embodiments of the methods and compositions provided herein relate to treating a subject suffering from hypertension, a cardiac injury, or a metabolic disorder. Some embodiments include administering an exosome to a subject. Some embodiments include administering an oligonucleotide to the subject. In some embodiments, the oligonucleotide comprises a Y-RNA or Y-RNA fragment such as EV-YF1 or EV-YF1-U16. In some embodiments, the oligonucleotide or exosome also has a therapeutic effect on the subject.

7. 发明申请

WO2023278802A1 FORMULATIONS FOR ORAL DELIVERY OF NUCLEIC ACIDS 审中-公开
公开(公告)号：WO2023278802A1
公开(公告)日：2023-01-05
申请号：PCT/US2022/035870
申请日：2022-06-30
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , IBRAHIM, Ahmed, G.
IPC分类号： C07K1/00 , C12N15/85 , C12N15/113 , A61K31/712 , A61K48/00 , C12Q1/6876
摘要： Provided herein are formulations configured for the oral delivery of a nucleic acid, such as a non-coding RNA. The formulations provided for herein comprise a plurality of cationic lipids used to encapsulate the nucleic acid within a micelle and a mixture of casein proteins and chitosan polymers used to coat the lipids, which form a coating on the micelle. The coated micelle lends acid-resistance to the formulation such that oral administration is possible with enhanced bioavailability of the nucleic acid to conditions associated with inflammation or fibrosis, such as hypertrophic myocardiopathy, heart failure with preserved ejection fraction, muscle disorders, such as muscular dystrophy, scleroderma and/or viral infection.

8. 发明申请

WO2023278799A1 THERAPEUTIC NUCLEIC ACIDS AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：WO2023278799A1
公开(公告)日：2023-01-05
申请号：PCT/US2022/035866
申请日：2022-06-30
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , IBRAHIM, Ahmed, G.
IPC分类号： A61K31/712 , A61K48/00 , C12N15/113 , C12Q1/6876 , C07K1/00 , C12N15/85
摘要： An isolated nucleic acid that includes the nucleotide sequence: CGUCCGAUGGUAGUGGGUUAUCAG (SEQ ID NO: 12) is provided. In some embodiment, the nucleic acid includes at least one chemically-modified nucleotide. The nucleic acid and composition containing same find use in treating coniditions associated with inflammation and/or fibrosis, for example, without limitation, heart conditions, such as hypertrophic myocardiopathy, myocardial infarction, and heart failure with preserved ejection fraction; muscle disorders, such as muscular dystrophy; skin disorders, such as scleroderma; and inflammatory conditions, such as autoimmune conditions or inflammatory conditions associated with a viral infection.

9. 发明申请

WO2021237238A1 EXOSOME-DERIVED PIWI-INTERACTING RNA AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：WO2021237238A1
公开(公告)日：2021-11-25
申请号：PCT/US2021/070568
申请日：2021-05-17
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , CIULLO, Alessandra , IBRAHIM, Ahmed, G.
IPC分类号： C12N15/11
摘要： Provided herein are PIWI-interacting RNA (piRNA) derived from therapeutic exosomes, and methods of use thereof to treat a condition requiring tissue repair and/or regeneration. Conditions treated by the exosome-derived piRNAs and/or exosomes carrying the same include, in some embodiments, ischemic injury and/or tissue fibrosis. Also provided are therapeutic compositions comprising exosome-derived piRNA and a pharmaceutically acceptable excipient.

10. 发明申请

WO2014028493A2 EXOSOMES AND MICRO-RIBONUCLEIC ACIDS FOR TISSUE REGENERATION 审中-公开
标题翻译：用于组织再生的外用和微型抗氧化剂
公开(公告)号：WO2014028493A2
公开(公告)日：2014-02-20
申请号：PCT/US2013/054732
申请日：2013-08-13
申请人： CEDARS-SINAI MEDICAL CENTER
发明人： MARBÁN, Eduardo , CHENG, Ke , IBRAHIM, Ahmed
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , A61K9/0019 , A61K31/7105 , A61K35/34 , A61K47/42 , A61K47/46 , C12N5/0657 , C12N15/111 , C12N2310/141 , C12N2320/30 , C12N2502/1329 , C12N2502/28 , Y02A50/463 , A61K2300/00
摘要： Several embodiments relate to methods of repairing and/or regenerating damaged or diseased tissue comprising administering to the damaged or diseased tissues compositions comprising exosomes. In several embodiments, the exosomes comprise one or more microRNA that result in alterations in gene or protein expression, which in turn result in improved cell or tissue viability and/or function.
摘要翻译：一些实施方案涉及修复和/或再生受损或患病组织的方法，其包括对损伤或患病组织施用包含外来体的组合物。在几个实施方案中，外来体包含导致基因或蛋白质表达改变的一种或多种微小RNA，其又导致改善的细胞或组织活力和/或功能。

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