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1. 发明申请

WO2021207273A1 MANUFACTURING OF SYNTHETIC EXOSOMES FOR CNS AND NON-CNS DELIVERY OF THERAPEUTICS 审中-公开
公开(公告)号：WO2021207273A1
公开(公告)日：2021-10-14
申请号：PCT/US2021/026049
申请日：2021-04-06
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： CAMPAGNA, Jesus , JOHN, Varghese
IPC分类号： A61K45/06 , A61K9/00 , A61K9/127
摘要： This invention provides improved synthetic exosomes for delivery a one or more therapeutic agents to the central nervous system. In certain embodiments the synthetic exosome comprises a liposome formed from a lipid bilayer, where said lipid bilayer comprises: one or more phospholipids selected from the group consisting of phosphate lipids, phosphoglycerol lipids, phosphocholine lipids, and phosphoethanolamine lipids where the lipid carbon chain ranges from 3 to 24 carbon atoms; cholesterol, a cholesterol derivative (e.g., cholesterol hemicsuccinate), or a phytosterol; and a non-ionic surfactant; wherein the lipid bilayer does not contain an alcohol; and the liposome ranges in size from about 50 nm up to about 200 nm in diameter. Typically, the synthetic exosome is capable of crossing the blood brain barrier without substantially leaking said therapeutic moiety.

2. 发明申请

WO2018187240A1 DEFORMABLE NANO-SCALE VEHICLES (DNVS) FOR TRANS-BLOOD BRAIN BARRIER, TRANS-MUCOSAL, AND TRANSDERMAL DRUG DELIVERY 审中-公开
公开(公告)号：WO2018187240A1
公开(公告)日：2018-10-11
申请号：PCT/US2018/025749
申请日：2018-04-02
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： JOHN, Varghese , NISHIMURA, Ichiro , SUBBIAH, Naren , CAMPAGNA, Jesus , SPILMAN, Patricia, R. , ALAM, Mohammad, Parvez
IPC分类号： A61K47/24 , A61K47/28 , A61K47/48
摘要： In various embodiments deformable nano-scale vehicles (DNV) are provided that are useful for the delivery of therapeutic agents. In certain embodiments the DNVs are capable of transdermal delivery and can additionally cross the blood-brain barrier.

3. 发明申请

WO2017004436A2 A THERAPEUTIC SYSTEM COMPRISING PHARMACEUTICALS IN COMBINATION WITH METABOLIC ENHANCEMENT FOR NEURODEGENERATION 审中-公开
标题翻译：一种包含药物组合与代谢增强用于神经修复的治疗系统
公开(公告)号：WO2017004436A2
公开(公告)日：2017-01-05
申请号：PCT/US2016/040518
申请日：2016-06-30
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： JOHN, Varghese , BREDESEN, Dale E. , SPILMAN, Patricia R.
IPC分类号： A61K31/202 , A61K31/122 , A61K31/12 , A61K31/519 , A61K31/66 , A61K31/05 , A61K45/06
CPC分类号： A61K31/05 , A23L33/30 , A61K31/12 , A61K31/122 , A61K31/13 , A61K31/197 , A61K31/198 , A61K31/202 , A61K31/27 , A61K31/385 , A61K31/4045 , A61K31/445 , A61K31/473 , A61K31/519 , A61K31/522 , A61K31/55 , A61K31/593 , A61K31/7068 , A61K31/714 , A61K33/04 , A61K33/06 , A61K36/81 , A61K36/9066 , A61K45/06 , A61K2300/00

4. 发明申请

WO2016138533A2 SMALL MOLECULES THAT ENABLE CARTILAGE REJUVANATION 审中-公开
标题翻译：小分子，使启动软骨再生
公开(公告)号：WO2016138533A2
公开(公告)日：2016-09-01
申请号：PCT/US2016/020126
申请日：2016-02-29
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： EVSEENKO, Denis , VAN HANDEL, Benjamin, J. , JOHN, Varghese
IPC分类号： C07D417/04 , C07D277/38 , A61K31/426 , A61K31/427 , A61P19/00
CPC分类号： C07D417/04 , C07D277/42
摘要： There are provided, inter alia, methods and compounds for activating a proliferative program in competent adult chondrocytes.
摘要翻译：特别提供了在成人软骨细胞中激活增殖程序的方法和化合物。

5. 发明申请

WO2022047123A1 COMPOSITIONS AND METHODS FOR TREATING AMYLOID-RELATED CONDITIONS 审中-公开
公开(公告)号：WO2022047123A1
公开(公告)日：2022-03-03
申请号：PCT/US2021/047894
申请日：2021-08-27
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： CHANDRA, Sujyoti , JOHNSON-COHN, Samuel W. , JOHN, Varghese , JAGODZINSKA, Barbara , CAMPAGNA, Jesus
IPC分类号： C07C237/04 , A61K31/165 , A61P21/00 , A61P25/28 , A61P27/00 , A61K31/137
摘要： The present disclosure relates to compounds that are capable of upregulating sAPPα and/or stabilizing reticulon-3. The disclosure further relates to methods of treating neurodegenerative diseases and disorders (e.g., Alzheimer's disease).

6. 发明申请

WO2022055926A1 COMPOSITIONS AND METHODS FOR TREATING MUSCULAR DYSTROPHIES 审中-公开
公开(公告)号：WO2022055926A1
公开(公告)日：2022-03-17
申请号：PCT/US2021/049362
申请日：2021-09-08
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： CROSBIE, Rachelle, H. , SHU, Cynthia , JOHN, Varghese , CAMPAGNA, Jesus , JAGODZINSKA, Barbara , PARFENOVA, Liubov , MOKHONOVA, Ekaterina
IPC分类号： C07D405/12 , C07D403/12 , C07D413/12 , C07D215/16 , C07D401/12 , A61K31/47 , A61P21/00
摘要： The present disclosure relates to compounds that increase sarcospan expression. The disclosure further relates to methods of treating a disease related to diminution or dysfunction of a dystrophin-related complex in a subject in need thereof.

7. 发明申请

WO2022076507A1 COMPOSITIONS AND METHODS FOR TREATING NEURODEGENERATIVE DISEASES AND DISORDERS 审中-公开
公开(公告)号：WO2022076507A1
公开(公告)日：2022-04-14
申请号：PCT/US2021/053696
申请日：2021-10-06
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： CAMPAGNA, Jesus , JOHN, Varghese , JUNG, Michael, E.
IPC分类号： C07C229/08 , C07C219/00 , C07C225/00 , C07D211/00 , A61K31/16 , A61K31/19 , A61K31/40 , A61K31/4458 , A61P25/28 , A61K31/12 , A61K31/221 , C07C229/26 , C07C229/28 , C07C229/36 , C07C237/06
摘要： The present disclosure relates to compounds that are capable of inhibiting PRMT8 and/or upregulating SirTl. The disclosure further relates to methods of treating neurodegenerative diseases and disorders (e.g., Alzheimer's disease).

8. 发明申请

WO2018048953A1 ALLOSTERIC CORTICOTROPIN-RELEASING FACTOR RECEPTOR 1 (CRFR1) ANTAGONISTS THAT DECREASE P-TAU AND IMPROVE COGNITION 审中-公开
标题翻译：抑制P-TAU和改善认知的变应原促细胞分裂素因子受体1（CRFR1）拮抗剂
公开(公告)号：WO2018048953A1
公开(公告)日：2018-03-15
申请号：PCT/US2017/050347
申请日：2017-09-06
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： ALAM, Mohammad Parvez , JOHN, Varghese , JOGODZINSKA, Barabara
IPC分类号： C07D409/04 , A61K31/5377 , A61K31/519
摘要： In various embodiments novel allosteric antagonists of the CRFR1 receptor are provided. It discovered that allosteric CRFR1 receptor antagonists are effective to modulate p-Tau levels in Alzheimer's disease (AD) models.
摘要翻译：在不同的实施方案中，提供了CRFR1受体的新变构拮抗剂。它发现变构CRFR1受体拮抗剂可有效调节阿尔茨海默病（AD）模型中的p-Tau水平。

9. 发明申请

WO2018027193A1 GP130 MODULATORS 审中-公开
标题翻译： GP130调制器
公开(公告)号：WO2018027193A1
公开(公告)日：2018-02-08
申请号：PCT/US2017/045626
申请日：2017-08-04
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA , VADIVEL, Kanagasabai
发明人： VADIVEL, Kanagasabai , ALAM, Mohammad, Parvez , SPILMAN, Patricia , JOHN, Varghese
IPC分类号： A61K31/426 , A61K31/41 , A61P25/00
摘要： There are provided, inter alia, compounds and methods useful for the treatment of neurological conditions, such as neurological disorders and neurodegenerative diseases, including Alzheimer's Disease.
摘要翻译：尤其提供了用于治疗神经病症如神经病症和神经退行性疾病（包括阿尔茨海默氏病）的化合物和方法。

10. 发明申请

WO2017124000A1 3D-EXOQUANT METHOD FOR THE ANALYSIS OF SURFACE MOLECULES AND QUANTIFICATION OF TISSUE-SPECIFIC EXOSOMES IN BIOLOGICAL FLUIDS 审中-公开
标题翻译：用于分析生物流体中表面分子和组织特异性外生体的3D-EXOQUANT方法
公开(公告)号：WO2017124000A1
公开(公告)日：2017-07-20
申请号：PCT/US2017/013510
申请日：2017-01-13
申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
发明人： JOHN, Varghese , CAMPAGNA, Jesus , BILOUSOVA, Tina , SPILMAN, Patricia , HEINZELMAN, Peter , HATAMI, Asa , ALAM, Mohammad Parvez
IPC分类号： G01N33/536 , G01N33/543 , G01N33/68 , G01N33/574
CPC分类号： G01N33/567 , C07D207/46 , G01N33/542 , G01N33/54326 , G01N33/54353 , G01N33/57484 , G01N2800/2821 , G01N2800/2835 , G01N2800/285
摘要： In various embodiments methods are provided for identifying and/or quantifying one or more antigens of interest (biomarkers) on the surface of cell- or tissue-specific exosomes. In an illustrative embodiments the methods comprise: i) incubating a population of exosomes with one or more tissue-specific antibodies that bind an antigen specific to a tissue or cell type of interest that produces exosomes, where the tissue specific antibodies are attached to acceptor bead or magnetic beads so the antibodies bind exosomes displaying the antigen; ii) obtaining a purified population of exosomes bound by the tissue specific antibodies with and/or without photo-cleavable linker based technology; iii) incubating a test subset of the isolated tissue-specific exosomes with acceptor beads attached to test antibodies that bind an antigen of interest thereby binding exosomes that display the antigen of interest and a control subset with negative control acceptor beads; v) incubating the test subset of isolated exosomes and the control subset of exosomes with a donor-bearing antibody that binds an exosome specific antigen; and vi) detecting a signal produced upon illumination of the control subset and/or the test; and vii) detecting the antigen(s) of interest.
摘要翻译：在各种实施方式中，提供了用于鉴定和/或定量细胞或组织特异性外泌体表面上的一种或多种感兴趣抗原（生物标志物）的方法。在说明性实施方案中，所述方法包括：i）将外泌体群体与一种或多种组织特异性抗体一起孵育，所述组织特异性抗体结合对产生外来体的感兴趣的组织或细胞类型特异性的抗原，其中组织特异性抗体附着于受体珠或磁珠，使得抗体结合展示抗原的外来体; ii）使用和/或不使用基于光切割接头的技术获得由组织特异性抗体结合的外泌体的纯化群体; iii）将分离的组织特异性外泌体的测试子集与附着测试抗体的受体珠孵育，所述测试抗体结合感兴趣抗原，从而结合显示感兴趣抗原的外来体和具有阴性对照受体珠的对照子集; v）将分离的外泌体的测试子集和外泌体的对照子集与结合外泌体特异性抗原的携带有供体的抗体一起孵育; 和vi）检测在照射控制子集和/或测试时产生的信号; 和vii）检测感兴趣的抗原。

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