Reduced Intensity Haploidentical Peripheral Blood Stem Cell Transplantation With Post-transplant Cyclophosphamide and Sirolimus/Mycophenolate Mofetil/RGI-2001 Based GVHD Prevention: a Pilot Study

This research study is studying the RGI-2001 for preventing Graft-vs-Host Disease (GVHD) in people with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), chronic myelomonocytic leukemic (CMML), chemosensitive hodgkin lymphoma (HL), or Non-Hodgkin lymphoma (NHL).who will have a blood stem cell transplantation.
GVHD is a condition in which cells from the donor's tissue attack the organs.
RGI-2001 is an investigational treatment

开始日期2020-08-14

申办/合作机构

REGiMMUNE Corp.

NCT04014790

/ Completed临床2期

An Open Label Phase 2, Study to Evaluate the Safety and Efficacy of RGI-2001 for the Prevention of Acute Graft-vs-Host Disease Compared to Contemporary Controls in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation

This Phase II open label study will evaluate the safety and efficacy of repeat doses of RGI-2001 in combination with standard of care treatment for the prevention of acute graft-vs-host-disease (aGvHD) in subjects following Allogeneic Hematopoietic Stem Cell Transplantation (alloHSCT). These subjects will be compared to contemporary controls.

开始日期2019-11-25

申办/合作机构

REGiMMUNE Corp.

JPRN-UMIN000009607

/ Completed临床2期IIT

The double-blind, randomised controlled phase II study of the adjuvant therapy using alpha-galactosylceramide pulsed antigen presenting cells for patients with head and neck mucosal melanoma followed by standard therapy. - Chiba NKT therapy for HNSCC

开始日期2013-04-01

申办/合作机构-

100 项与 KRN-7000 liposomal 相关的临床结果

登录后查看更多信息

100 项与 KRN-7000 liposomal 相关的转化医学

登录后查看更多信息

100 项与 KRN-7000 liposomal 相关的专利（医药）

登录后查看更多信息

503

项与 KRN-7000 liposomal 相关的文献（医药）

2025-02-03·JOURNAL OF EXPERIMENTAL MEDICINE

Identification of α-galactosylceramide as an endogenous mammalian antigen for iNKT cells

Article

作者: Yamasaki, Sho ; Kumanogoh, Atsushi ; Izumi, Yoshihiro ; Compostella, Federica ; Tomiyasu, Noriyuki ; Ishikawa, Eri ; Kasai, Hayato ; Takahashi, Masatomo ; Ishida, Hideharu ; Bamba, Takeshi ; Imamura, Akihiro ; Hosono, Yuki ; Kida, Hiroshi

Invariant natural killer T (iNKT) cells are unconventional T cells recognizing lipid antigens in a CD1d-restricted manner. Among these lipid antigens, α-galactosylceramide (α-GalCer), which was originally identified in marine sponges, is the most potent antigen. Although the presence of α-anomeric hexosylceramide and microbiota-derived branched α-GalCer is reported, antigenic α-GalCer has not been identified in mammals. Here, we developed a high-resolution separation and detection system, supercritical fluid chromatography tandem mass spectrometry (SFC/MS/MS), that can discriminate hexosylceramide diastereomers (α-GalCer, α-GlcCer, β-GalCer, or β-GlcCer). The B16 melanoma tumor cell line does not activate iNKT cells; however, ectopic expression of CD1d was sufficient to activate iNKT cells without adding antigens. B16 melanoma was unlikely to generate iNKT cell antigens; instead, antigen activity was detected in cell culture serum. Activity-based purification and SFC/MS/MS identified dihydrosphingosine-based saturated α-GalCer as an antigenic component in serum, bile, and lymphoid tissues. These results show the first evidence for the presence of potent antigenic α-GalCer in mammals.

2024-12-01·Molecular Therapy-Nucleic Acids

The role of mRNA-galsomes and LNPs in enhancing HIV-specific T cell responses across various lymphoid organs

Article

作者: Verbeke, Rein ; Roover, Sabine den ; Cosyns, Joëlle ; Meulewater, Sofie ; Aerts, Joeri L ; Laeremans, Thessa ; Vanbellingen, Sarah ; D'haese, Sigrid ; Aernout, Ilke ; Meert, Jessy ; Lentacker, Ine ; den Roover, Sabine

mRNA nanoparticles have been investigated in the context of prophylactic vaccination against HIV, but their effectivity has not been widely investigated in therapeutic vaccination. It has been suggested that a profound CD8⁺ T cell response within lymphoid tissues, a primary site for viral reservoirs, is crucial for achieving optimal viral control, potentially correlating with protection. This study aimed to evaluate the effectiveness of mRNA lipid nanoparticles (LNPs), including a modified variant containing α-galactosylceramide as an adjuvant, termed galsomes. C57BL/6 mice were immunized intramuscularly with nucleoside-modified mRNA encoding ovalbumin (li80tOVA), revealing that mRNA-galsomes induced slightly higher proliferation levels of li80tOVA-specific CD8⁺ T cells in lymphoid tissues across various anatomical sites compared with mRNA-LNPs. In addition, immunization with nucleoside-modified HIV-1 Gag mRNA elicited a notable Gag-specific immune response in both formulations even at low mRNA doses. Remarkably, mRNA-galsomes induced lower polyfunctional responses in CD4⁺ T cells, but similar polyfunctional responses in CD8⁺ T cells in the spleen compared with mRNA-LNPs. Importantly, the Gag-specific CD8⁺ T cells demonstrated cytolytic capacity against target cells in both the spleen and lymphoid tissues, including gut-associated lymph nodes. These findings underscore the potential of both mRNA-galsomes and mRNA-LNPs as tools for therapeutic vaccination against HIV.

2024-10-10·JOURNAL OF MEDICINAL CHEMISTRY

Fully Synthetic TF-Based Self-Adjuvanting Vaccine Simultaneously Triggers iNKT Cells and Mincle and Protects Mice against Tumor Development

Article

作者: Yang, Deying ; Li, Tongtong ; Ming, Wenbo ; Luo, Xiang ; Li, Xiaohui ; Liao, Guochao ; Li, Jinmei ; Liao, Pan ; Liu, Zhongqiu ; Liu, Zichun

The Thomsen-Friedenreich (TF) antigen has proven to be a promising target for developing novel therapeutic cancer vaccines. Here, a new strategy that TF antigen covalently coupled with KRN7000 and vizantin was developed. The resulting three-component vaccine KRN7000-TF-vizantin simultaneously triggers invariant natural killer T (iNKT) cells and macrophage-inducible C-type lectin (Mincle) signaling pathways, eliciting much stronger TF-specific immune responses than glycoprotein vaccine TF-KLH/alum and the corresponding two-component conjugate vaccines TF-KRN7000 and TF-vizantin. The analysis of IgG isotypes and the secretion of cytokines revealed that KRN7000-TF-vizantin induced Th1/Th2 mixed immune responses, where Th1 was dominant. In vivo experiments demonstrated that KRN7000-TF-vizantin increased the survival rate and survival time of tumor-challenged mice, and surviving mice rejected further tumor attacks without any additional treatment. This work demonstrates that covalently coupled KRN7000 and vizantin could serve as a promising TF-based vaccine carrier for antitumor immune therapy, and KRN7000-TF-vizantin features great potential to be a vaccine candidate.

项与 KRN-7000 liposomal 相关的新闻（医药）

2024-10-18

·regimmune.com

REGiMMUNE and Kiji Therapeutics Announce Intention to merge

The intended merger will create a pan-global Treg specialist to use multiple modalities to target Tregs for a number of indications. Taipei, TAIWAN, and Paris, FRANCE, October 18, 2024 – REGiMMUNE, the regulatory T cell targeting drugs for immunotherapy and Kiji Therapeutics, the specialist in Induced Pluripotent Stem Cells-Mesenchymal Stem Cells (IPSC-MSC) engineered cell therapies for [...] TAIPEI, June 4, 2024 – REGiMMUNE Limited is pleased to announce that our company has recently been featured in a report by “China Times”. The article highlights our development progress of RGI-2001 and our future IPO plans. We are grateful for the attention and support from “China Times”. You can read the full article here: [...] TAIPEI, May 6, 2024 – REGiMMUNE Limited, a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies for immune disorders and cancer, is pleased to announce the appointment of Dr. Steve Yang as its Chief Operating Officer (COO), effective on May 6th 2024. In this role, Dr. Yang will lead the company’s operational strategies, driving product [...] TAIPEI, April 11, 2024 – REGiMMUNE Limited, a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies for immune disorders and cancer, today announces the poster presentation at the American Association of Cancer Research (AACR) Annual Meeting. The poster presentation is to highlight RGI-2001 phase 2b result, including the latest correlative analyses of changes in regulatory [...] TAIPEI, February 22, 2024 – REGiMMUNE Limited, a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies for immune disorders and cancer, today announced the positive results of their phase 2b clinical trial for the prevention of acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic cell transplantation (alloHCT) at the 2024 Tandem Meeting of ASTCT and CIBMTR. [...] TAIPEI, December 28, 2023 – REGiMMUNE Limited, a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies for immune disorders and cancer, today announced that the Phase 2b clinical data from 48 patients receiving RGI-2001 for the prevention of acute graft-versus-host disease (aGVHD) will be presented at the 2024 Tandem Meeting in San Antonio, Texas on [...] Taipei, July 6, 2023 – REGiMMUNE Limited (REGiMMUNE), a biotech company focused on creating innovative immunotherapies for immune disorders and cancer, today appointed Hua Nan Securities as the lead underwriter for its upcoming initial public offering (IPO) in Taiwan. The IPO is anticipated to take place in 2025, signifying an important milestone in REGiMMUNE's expansion [...] Taipei, March 29, 2023 – REGiMMUNE Limited (REGiMMUNE), a biotech company focused on creating innovative immunotherapies for immune disorders and cancer, and San Fu Biotech (SFB), a subsidiary of San Fu Chemical Co., Ltd. (4755.TW) have entered a licensing agreement to develop and commercialize RGI-2001 for the prophylaxis of acute Graft-versus-host disease (aGvHD) in major [...] RGI-2001 was well tolerated and associated with a meaningful reduction in the incidence of severe acute GVHD TAIPEI, December 12, 2022 – REGiMMUNE Limited, a clinical-stage biopharmaceutical company developing innovative immunotherapies for immune disorders and cancer, today announced the positive results of their Phase 2b clinical trial for the prevention of acute graft-versus-host disease (aGvHD) [...] TAIPEI, November 3, 2022 – REGiMMUNE Limited, a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies for immune disorders and cancer, today announced that the Phase 2b clinical data from 49 patients receiving RGI-2001 for the prevention of acute graft-versus-host disease (aGVHD) will be presented at the 2022 American Society of Hematology (ASH) Annual Meeting [...] Taipei, 16 May 2022 – REGiMMUNE Limited, a biopharmaceutical company focused on creating innovative immunotherapies for rare diseases and cancer, today announced Charlie Hsu, a seasoned financial executive, join the company's management team as Chief Financial Officer (CFO). Charlie brings more than 25 years of business experience in finance management and equity investment in both [...] REGiMMUNE Corporation has presented clinical data on RGI-2001 Intravenous Infusion for the prevention of acute GVHD in the annual meeting of the American Society of Hematology on December 5 2020. Repeat weekly doses of RGI-2001 100 μg/kg IV infusion (30min) in allogeneic bone marrow transplant is safe when given with standard GvHD prophylaxis [...] Tokyo, Japan – April 7, 2017 – REGiMMUNE Corporation announced the closing of a Series E financing on April 7th. The company raised 6 million US in this new round, which was led by SMBC Venture Capital Co., Ltd., the VC arm of Sumitomo Mitsui Banking Corporation(SMBC) and Japan Asia Investment Corporation (JAIC). Additional investors [...] We are proud to announce that we have moved our business to a new location. Our new address is as follows. 35-3 Nihonbashi Hakozaki-cho BRICK GATE 5F Chuou-ku Tokyo 103-0015 Japan.Our Telephone numbers remain the same. Tokyo Japan — July 1, 2016 — REGiMMMUNE today announced that its Board of Directors has appointed Kenzo Kosuda as Chief Executive Officer effective July 1, 2016. Yasuyuki Ishii will assume the role of Executive Chairman on July 1, 2016, and will continue to serve as the Chairman of REGiMMUNE’s Board. "This is an appropriate [...]

临床2期高管变更免疫疗法引进/卖出

2023-04-01

·Business Wire

REGiMMUNE Limited Licenses the Rights to Develop and Commercialize RGI-2001 To San Fu Biotech in Major Asian Countries

TAIPEI, Taiwan--( BUSINESS WIRE )--REGiMMUNE Limited (REGiMMUNE), a biotech company focused on creating innovative immunotherapies for immune disorders and cancer, and San Fu Biotech (SFB), a subsidiary of San Fu Chemical Co., Ltd. (4755.TW) have entered a licensing agreement to develop and commercialize RGI-2001 for the prophylaxis of acute Graft-versus-host disease (aGvHD) in major Asian countries. RGI-2001, with the novel mechanism to improve existing treatments, is a potentially first-in-class small molecule drug candidate targeted for preventing aGvHD, a life-threatening complication resulting from allogeneic hematopoietic stem cell transplantation (HSCT). GvHD is a systemic disorder that occurs when the graft's immune cells recognize the host as foreign and attack the recipient’s body cells or organs, and leads to skin rash, liver problems, abdominal pain or cramps, diarrhea, and increased risk for infections. Patients undergoing allogeneic HSCT have a high-risk of developing aGVHD associated with significant morbidity and mortality. Despite the use of prophylactic immunosuppressive therapy, clinically significant aGvHD develops in 20%-40% of HLA-matched related and unrelated allogeneic HSCT and severe cases contribute to non-relapse mortality. Under the terms of agreement, REGiMMUNE and SFB will collaborate closely to accelerate the development of RGI-2001 in major Asian countries and will conduct clinical trials to study the efficacy of the drug in aGvHD. Together, both parties intend to submit an Investigational New Drug Application to the Taiwan Food and Drug Administration to initiate clinical trials in Taiwan for the prophylaxis of aGvHD before the end of 2023. SFB will pay to REGiMMUNE development milestones and royalties on net profit upon successful commercialization of RGI-2001. SFB also retains the first rights of negotiation to develop and commercialize RGI-2001 for new indications in the authorized territories. RGI-2001 of REGiMMUNE, awarded Orphan Drug Designation by the US FDA in GvHD in 2012, has completed the phase II clinical trial targeting at the prophylaxis of aGvHD in the US. The efficacy and safety results were positive. Furthermore, RGI-2001 was selected for oral presentation at the American Society of Hematology (ASH) annual meeting in December 2022 and planned to submit phase III IND to US FDA in Q4 of 2023. Kenzo Kosuda , Chief Executive Officer and President, REGiMMUNE Limited, said : “ This agreement underscores our focus and commitment to improve the incumbent therapies of aGvHD prevention and treatment. The fast-growing patients with GvHD in Asia create a high unmet need for more effective treatments. ” “We are pleased to collaborate with SFB and San Fu Chemical Group which has a long history in Taiwan. The Group’s abundant biomedical resources, expertise and innovative strategy make San Fu a strong commercial partner for REGiMMUNE in Asia. Together, we look forward to expediting the development of RGI-2001 in this important market.” Simon H.H. Wu, SFB & San Fu Chemical Group Chairman, emphasized: “We are excited to add RGI-2001 to our growing portfolio of innovative therapeutic agents. This enhances our autoimmune pipeline and aligns with our long-term research and development strategy. RGI-2001 has demonstrated promising results in phase II studies in the US and we look forward to working together with REGiMMUNE to deliver treatments that will benefit the lives of patients.” About RGI-2001 RGI-2001 is a liposomal formulation of an alpha-galactosylceramide (alpha-GalCer) analog. Alpha-GalCer is a ligand for CD1d expressed on antigen presenting cells and invariant natural killer T cells (iNKT). Liposomal alpha-GalCer promotes tolerogenic immune cascades, resulting in the activation and expansion of regulatory T cells (Tregs). RGI-2001 is REGiMMUNE’s lead drug candidate currently in Phase 3 planning for the prophylaxis of acute graft-versus-host disease (aGVHD). About REGiMMUNE Limited REGiMMUNE is a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies by harnessing the power of regulatory T cells (Tregs). REGiMMUNE is creating a pipeline of novel product candidates that either enhance Treg activities for immune diseases or suppress Treg activities for cancer. About San Fu Biotech San Fu Biotech, a wholly-owned subsidiary of San Fu Chemical, develops first-in-class new drugs to address unmet medical needs, such as cancer tumors, autoimmune disease and ophthalmology, for benefiting patients around the world.

临床结果临床2期引进/卖出免疫疗法临床3期

2022-12-22

·Science Daily

Cellular messengers improve cancer therapy

Nano-sized membrane bubbles known as extracellular vesicles activate the immune system in mice and seem to render their tumors sensitive to a type of immunotherapy drug called a checkpoint inhibitor. Nano-sized membrane bubbles known as extracellular vesicles activate the immune system in mice and seem to render their tumours sensitive to a type of immunotherapy drug called a checkpoint inhibitor. This is according to a new study published in Cancer Immunology Research by researchers at Karolinska Institutet in Sweden. Treatments for various forms of cancer have improved considerably over recent years thanks to a type of drug called a checkpoint inhibitor, which helps the immune system's T cells to attack the cancer cells. However, even though some patients respond extremely well to treatment, a large proportion only see temporary improvement, if any. Scientists are devoting considerable energy to understanding why this is so and to combining checkpoint inhibitors with other therapies in order to increase the cancer survival rate. A new cancer therapy Researchers from Karolinska Institutet how show that a form of round nanoparticles called exosomes or extracellular vesicles are a promising path to follow. "Is seems that the vesicles make the tumour immunologically active so that the checkpoint therapy can gain purchase and start to work," says the study's last author Susanne Gabrielsson, professor at the Department of Medicine (Solna), Karolinska Institutet. "These results give support to the further development of extracellular vesicles as a new cancer therapy." Extracellular vesicles are sometimes referred to as the body's messengers. They are membrane-bound nano-scale bubbles that cells can send to each other to exchange information. Vesicles from tumour cells, for instance, can switch off the immune system so that the cancer can spread, while vesicles from immune cells can activate an immune response. Can activate immune cells In earlier studies, the KI researchers have shown that a certain type of extracellular vesicle from immune cells can activate immune T cells and reduce tumour growth in mice. In the present study, they examined how these vesicles function in a mouse model of a skin cancer that is resistant to checkpoint inhibitor therapy. The extracellular vesicles used in the study were isolated from the mice's own immune cells (dendritic cells). An in this case cancer-specific protein called ovalbumin and a molecule called alpha-Galactosylceramide were then added, both of which stimulate T cells and natural killer cells. When the vesicles were introduced into mice therapeutically to treat their tumours or prophylactically before their tumours had started to develop, they activated their immune systems to produce a strong T-cell response to the cancer protein. The same effect was not achieved if the animals were only given checkpoint inhibitors, and was most pronounced in animals that received a combination of vesicles and checkpoint therapy. However, the researchers observed no effect on survival when the animals received the vesicle-checkpoint combination therapy compared with vesicles alone, and believe that the duration of the experiment was possibly too short for the activated immune system to affect the tumours. When the treatments were given prophylactically to mice, which gives a longer duration of action, the mice that received the combination treatment showed greater survival than those that only received vesicles. Tested on human patients Other researchers tried giving extracellular vesicles from immune cells to human patients as a cancer therapy already back in 2005. In these studies, the vesicles have proved safe but only minimally effective. Professor Gabrielsson believes that this was because the experiments were conducted too early, before scientists knew what molecules the vesicles should contain to be effective, something to which her team has devoted considerable effort. They are also trying to simplify the manufacture of the extracellular vesicles. "Our aim is to be able to use cell lines instead of having to take the patients' own cells," she says. "This will mean that the vesicles can be prepared in advance and frozen until needed. We also believe that variants of the treatment could be used for other forms of cancer and other diseases." The study was supported by grants from the Swedish Research Council, the Swedish Cancer Society, the Cancer Research Funds of Radiumhemmet, Region Stockholm and the Swedish Heart-Lung Foundation and with KID funding from Karolinska Institutet. Susanne Gabrielsson holds a patent for immunotherapeutic exosomes from B cells and is on the scientific advisory committee at Anjarium Biosciences.

免疫疗法临床结果

100 项与 KRN-7000 liposomal 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB12232

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
移植物抗宿主病	临床2期	日本	REGiMMUNE Corp.	2022-01-30
血液肿瘤	临床2期	日本	REGiMMUNE Corp.	2022-01-30
急性移植物抗宿主病	临床2期	美国	REGiMMUNE Corp.	2019-11-25
慢性丙型肝炎	临床2期	荷兰	Kyowa Kirin Co., Ltd.	2003-08-01
急性淋巴细胞白血病	临床1期	美国	REGiMMUNE Corp.	2020-08-14
急性髓性白血病	临床1期	美国	REGiMMUNE Corp.	2020-08-14
慢性粒单核细胞白血病	临床1期	美国	REGiMMUNE Corp.	2020-08-14
霍奇金淋巴瘤	临床1期	美国	REGiMMUNE Corp.	2020-08-14
骨髓增生异常综合征	临床1期	美国	REGiMMUNE Corp.	2020-08-14
骨髓增生性疾病	临床1期	美国	REGiMMUNE Corp.	2020-08-14

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04014790 (RGI-2001-003, CTgov)	临床2期	急性移植物抗宿主病	49	Standard of Care+RGI-2001	繭積齋鬱繭憲糧鹽蓋網(廠網觸糧膚鑰範築夢製) = 範糧淵膚壓憲構鹹鏇艱廠衊艱壓廠願簾壓淵遞 (窪壓選壓衊醖醖膚顧鏇, 顧鬱窪顧壓醖餘選襯壓 ~ 製鏇積鹹積衊範顧願積) 更多	-	2024-04-17
RGI-2001-003 (EBMT2023)	临床2期	急性移植物抗宿主病	49	RGI-2001 IV infusion	鹹餘範鬱醖觸鏇憲淵糧(簾築顧齋願鹽範鏇窪鏇) = 築製廠獵廠膚鑰鹹壓憲繭觸築鬱選選淵膚網築 (構簾鹹網遞齋艱餘壓鏇, 10.2 ~ 34.3)	-	2023-04-23
RGI-2001-003 (Tandem Meetings ASTCT and CIBMTR2023)	临床2期	急性移植物抗宿主病	49	RGI-2001	淵淵願簾構鏇憲繭膚餘(蓋糧繭鏇衊齋淵壓鹹繭) = 6% 蓋壓襯襯廠製醖襯窪鹹 (繭顧鏇淵範膚衊鹽觸淵 ) 更多	积极	2023-02-01
NCT04014790 (RGI-2001-003, ASH 12022 \| ASH 22022) 人工标引	临床2期	急性移植物抗宿主病	53	RGI-2001 100 ug/kg	蓋糧壓窪鬱憲鑰鏇齋選(鬱淵膚淵膚鑰窪獵鹹構) = 簾鏇繭願艱觸餘齋鏇積簾構餘簾觸憲繭願積衊 (簾餘繭簾壓簾鑰壓壓網, 10.2 ~ 34.3) 更多	积极	2022-11-15