Zika virus infection during pregnancy is associated with severe birth defects including microcephaly in the new born. The lack of specific treatment calls for the development of a safe and effective vaccine for use in pregnant women. We recently tested the efficacy of a Virus Like Particle (VLP) vaccine for Zika virus in mice and found that Capsid-preMembrane-Env (CprME) VLPs generated a better neutralizing antibody response than preMembrane-Env (prME) VLPs. The superiority of CprME VLPs suggested that inclusion of capsid in the vaccine may enhance the immune response. However, production of CprME VLPs requires co-expression of NS2B-3 protease, which creates a major hurdle for generation of stable cell lines. To overcome this limitation, we generated a bicistronic vector that expresses CprME and NS2B-3 using an IRES sequence. This bicistronic expression cassette, in a lentiviral vector, was used to create a stable cell line that constitutively secretes CprME VLPs. The expression of NS2B-3, presence of capsid in the secreted VLPs, efficiency of VLP release, and stability of the cell line was extensively tested. Antigen sparing studies in mice using prME and CprME VLPs, both derived from stable cell lines, confirmed the superiority of CprME VLPs in generation of neutralizing antibody response. Capsid specific antibodies were detected in CprME VLP immunized mice providing mechanistic insights into the superiority of these VLPs. Challenge of CprME VLP immunized mice with Zika PRVABC59 showed complete protection against day 3 viremia further validating the efficacy of the vaccine. Our study is the first to generate a stable cell line secreting Zika CprME VLPs via natural NS2B-3 cleavage, demonstrate incorporation of capsid in CprME VLPs and complete protection in challenge studies. This is a major advancement for the Zika vaccine platform that is safe for use in pregnant women and readily scalable for use in developing countries.

2017-01-01·Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology

Enhancement of Anti-Leukemia Immunity by Leukemia–Derived Exosomes Via Downregulation of TGF-β1 Expression

Article

作者: Hu, Weiwei ; Wan, Jiangbo ; Hao, Siguo ; Huang, Fang

Background/Aims: Minimal residual leukemia cells (MRLs) are difficult to eradicate through traditional treatment and therefore remain to be a major threat to the long-term survival of leukemia patients. Tumor-derived exosomes (TEXs), which carry tumor associated antigens (TAA), may be a potential cell-free tumor vaccine for the specific eradication of MRLs. However, TEXs are intended to be less immunogenic due to exosomal TGF-β1. To further optimize the efficacy of TEX-based vaccines, we investigated whether exosomes from TGF-β1 silenced leukemia cells (LEXTGF-β1si) had an increased potential to induce a specific antitumor effect compared with non-modified exosomes. Methods: Exosomal TGF-β1 was downregulated via lentiviral shRNA silencing of TGF-β1 in leukemia cells. The characteristics of LEXTGF-β1si were determined via electron microscopy, western blot analysis, and flow cytometry. The antitumor effect of LEXTGF-β1si was evaluated by detecting the properties of LEXTGF-β1si-pulsed dendritic cells (DCs), CD4+ T-cell proliferation, Th1 cytokine secretion, specific CTL activity, and NK cell function. Moreover, to verify the superiority of LEXTGF-β1si immunization, LEXTGF-β1si was subcutaneously injected into DBA/2 mice: either followed by tumor challenge or tumor bearing. Results: The lentiviral shRNA silencing of TGF-β1 in parental leukemia cells successfully downregulated the TGF-β1 level in leukemia cell derived exosomes (LEX). LEXTGF-β1si was uptaken by DCs and was more potent in promoting DC function by upregulating the surface expression of costimulatory factors and MHC class II molecules, while inducing the secretion of IL-12p70 and TNF-α. Furthermore, immunization with LEXTGF-β1si facilitated CD4+ T-cell proliferation and Th1 cytokine secretion, and stimulated stronger specific cytotoxic lymphocyte (CTL) response and nature killer (NK) cell cytotoxicity more efficiently compared to non-modified LEX. In mice models, immunization with LEXTGF-β1si resulted in a more potent capability to inhibit tumor growth and to prolong survival, suggesting that LEXTGF-β1si was more effective in both protective and therapeutic antitumor tests than non-modified LEX. Conclusions: These data suggested that down-regulation of exosomal TGF-β1 effectively induced potent anti-tumor immunity. Our strategy of optimizing exosome vaccine may have promising potential for leukemia immunotherapy.

2013-02-01·Inflammation2区 · 医学

Immature Dendritic Cell-Derived Exosomes: a Promise Subcellular Vaccine for Autoimmunity

2区 · 医学

Review

作者: Yi Li ; Weifan Yin ; Huan Yang ; Bo Xiao ; Song Ouyang

Exosomes, 60-90-nm-sized vesicles, are produced by a large number of cell types, including tumor cells, neurons, astrocytes, hemocytes, intestinal epithelial cells, and so on. Dendritic cell (DC), the most potent professional antigen-presenting cell in the immune system, produces exosomes in the course of maturation. Mature DCs produce exosomes with the ability to elicit potent immunoactivation, resulting in tumor eradication and bacterial or virus elimination. Given the notion that exosomes are stable and easy to be modified artificially, autologous mature DC-derived exosomes have been vaccinated into patients with malignant diseases. In clinical trials utilizing exosomes as therapeutic approaches, researchers observed considerable curative effect with little side effect. However, immature or suppressive DC-derived exosomes harbor anti-inflammatory properties distinct from mature DC-derived exosomes. In murine models of autoimmune disease and transplantation, immature DC-derived exosomes reduced T cell-dependent immunoactivation, relieved clinical manifestation of autoimmune disease, and prolonged survival time of transplantation. Although the exact mechanism of how immature DC-derived exosomes function in vivo is still unclear, and there are no clinical trials regarding application of exosome vaccine into patients with autoimmune disease, we will analyze the promise of immature DC-derived exosomes as a subcellular vaccine in autoimmunity in this review.

项与 Exosome VLP vaccine (Capricor) 相关的新闻（医药）

2023-08-02

·GlobeNewswire-Health Care

Capricor Therapeutics to Present Second Quarter 2023 Financial Results and Recent Corporate Update on August 7

Company to Host Conference Call, August 7, 2023, at 4:30 p.m. ETSAN DIEGO, Aug. 02, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, will release its financial results for the second quarter ended June 30, 2023, after the market close on Monday, August 7, 2023. Management will then host a webcast and conference call at 4:30 p.m. ET on August 7. Title:Capricor Therapeutics Second Quarter 2023 Financial Results and Recent Corporate Update Conference Call and WebcastDate:Monday, August 7, 2023Time:4:30 p.m. ETConference Call Details:Toll-Free: 1-888-886-7786International: 1-416-764-8658 Conference ID: 02236204Participants can use guest dial-in numbers above and be answered by an operator or click the Call me™ link for instant telephone access to the event.Webcast:Webcast Link - Click Here A replay of the webcast will be available following the conclusion of the live broadcast and will be accessible on the Company’s website. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Further, Capricor has entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Capricor’s focus is on developing exosomes capable of delivering nucleic acids as well as proteins to treat or prevent a variety of diseases. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams and revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission on March 17, 2023 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023, as filed with the Securities and Exchange Commission on May 12, 2023. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Media Contact:Raquel ConaKCSA Strategic Communicationsrcona@kcsa.com 212.896.1204 Capricor Investor Contact:Joyce AllaireLifeSci Advisors, LLCjallaire@lifesciadvisors.com 617.435.6602 Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com 858.727.1755

财报细胞疗法

2023-07-24

·BioSpace

Capricor Therapeutics Announces Appointment of Philip J. Gotwals, Ph.D. to its Board of Directors

SAN DIEGO, July 24, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced the appointment of Philip J. Gotwals, Ph.D., to its Board of Directors, effective immediately. Dr. Gotwals, most recently the Global Head, Vice President of Business Development and Licensing at Novartis Institutes for Biomedical Research, brings his strategic vision for research, innovation for drug development and valuable industry experience to the Company. "It is a pleasure to welcome Philip to our Board of Directors," said Linda Marbán, Ph.D., Capricor’s chief executive officer. "Philip has a strong track record for leading research and development programs, from drug discovery through commercialization. His scientific acumen and ability to strategically drive forward business development for research will be invaluable as we continue efforts to advance our cell and exosome technologies. Philip is a tremendous addition to our board, and we look forward to working with him.” Dr. Gotwals added, "I am delighted to join Capricor as a member of the board of directors. The positive results shown to date suggest that CAP-1002 is well positioned, subject to regulatory approval, to become an anchor therapy for patients with Duchenne muscular dystrophy. Further, the StealthX™ exosome platform is progressing rapidly and holds the potential for a broad range of new therapeutic applications in the fields of vaccinology as well as targeted oligonucleotide, protein and small molecule therapeutics. I look forward to working with the Capricor team to advance the company’s mission.” Dr. Gotwals has experience in drug development, research, corporate strategy and business development with a career spanning nearly 30 years in the biotechnology industry. Dr. Gotwals most recently served as the Global Head, Vice President of Business Development and Licensing at Novartis Institutes for Biomedical Research (NIBR) where he oversaw business development efforts for all disease areas and technology platforms. Under Dr. Gotwals’ leadership, NIBR business development and licensing executed over 50 major strategic transactions which included licensing deals, collaborations, acquisitions and new company creations. These transactions led to significant corporate evolution and growth. During his 13 years at Novartis, Dr. Gotwals was instrumental in building the company’s immuno-oncology strategic research area and spearheading the collaboration with the University of Pennsylvania to develop chimeric antigen receptor (CAR) T-cell therapies. Prior to Novartis, he was Vice President of Program Management at Altus Pharmaceuticals, where he was responsible for all product development project management activities. Prior to Altus, he was Senior Director of Program and Alliance Management at Biogen, where he oversaw leadership of internal and allied early product development teams in the autoimmune, neurology and oncology therapeutic areas. Dr. Gotwals has a B.A. in Biology from Amherst College, holds a Ph.D. in Genetics from the University of California at Berkeley, completed postdoctoral research at the Massachusetts Institute of Technology, business training at Harvard Business School and has published extensively in the area of integrin biology. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Further, Capricor has entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Capricor’s focus is on developing exosomes capable of delivering nucleic acids as well as proteins to treat or prevent a variety of diseases. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams and revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission on March 17, 2023 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023, as filed with the Securities and Exchange Commission on May 12, 2023. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Media Contact: Raquel Cona KCSA Strategic Communications rcona@kcsa.com 212.896.1204 Capricor Investor Contact: Joyce Allaire LifeSci Advisors, LLC jallaire@lifesciadvisors.com 617.435.6602 Capricor Company Contact: AJ Bergmann, Chief Financial Officer abergmann@capricor.com 858.727.1755

高管变更细胞疗法

2023-07-13

·BioSpace

Capricor Therapeutics Announces Appointment of Paul Auwaerter, M.D. to its Board of Directors

-John Hopkins School of Medicine Clinical Director Joins Capricor’s Board of Directors with over 30 Years of Internal Medicine and Infectious Disease Experience- SAN DIEGO, July 13, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced the appointment of Paul Auwaerter, M.D. to its Board of Directors, effective immediately. Dr. Auwaerter brings over three decades of experience in internal medicine and infectious disease to Capricor. “I am extremely pleased to welcome Dr. Auwaerter to our Board of Directors,” said Linda Marbán, Ph.D., Capricor’s CEO. “His extensive background across medicine for over 30 years will bring an added level of drug development and medical experience to our team as we continue to advance our pipeline of therapeutic product candidates. Over the course of his career, Dr. Auwaerter has worked with some of the field’s most innovative leaders in the life sciences industry, and his experience and forward-thinking vision will make him a tremendous asset to our team.” “The opportunity to join Capricor at this crucial stage of development for the Company is compelling. The Company is an innovative leader with its late-stage CAP-1002 cell therapy program for Duchenne muscular dystrophy as well as its exosomes platform that has demonstrated such promising potential in early development,” added Dr. Auwaerter. “I look forward to working alongside the executive team and my fellow Board members to help Capricor continue to advance their promising therapeutic pipeline.” Dr. Auwaerter holds multiple positions at Johns Hopkins University School of Medicine as the Clinical Director of the Division of Infectious Diseases, Director of the Sherrilyn and Ken Fisher Center for Environmental Infectious Diseases, and Chief Medical Officer for the Point of Care-Information Technology Center (POC-IT) for Healthcare Solutions. Additionally, Dr. Auwaerter is an attending physician for medicine and pediatrics at Johns Hopkins Hospital and Clinics. Prior to Capricor, he contributed to over one hundred published pieces of original research and is the Sherrilyn and Ken Fisher Professor of Medicine in the Divisions of Infectious Diseases and General Internal Medicine at Johns Hopkins University School of Medicine, where he has been since 1988. Dr. Auwaerter received an A.B. in Biology and M.D. in Medicine from Columbia University and also obtained an M.B.A. at the Johns Hopkins University School of Professional Studies in Business and Education. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Further, Capricor has entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Capricor’s focus is on developing exosomes capable of delivering nucleic acids as well as proteins to treat or prevent a variety of diseases. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams and revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission on March 17, 2023 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023, as filed with the Securities and Exchange Commission on May 12, 2023. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Media Contact: Raquel Cona KCSA Strategic Communications rcona@kcsa.com 212.896.1204 Capricor Investor Contact: Joyce Allaire LifeSci Advisors, LLC jallaire@lifesciadvisors.com 617.435.6602 Capricor Company Contact: AJ Bergmann, Chief Financial Officer abergmann@capricor.com 858.727.1755

高管变更细胞疗法

100 项与 Exosome VLP vaccine (Capricor) 相关的药物交易

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