Delving into the Latest Updates on HBG gene modified autologous CD34+ hematopoietic stem cells(Guangzhou Ruifeng) with Synapse

Overview

Basic Info

Drug Type

Gene therapy, Hematopoietic stem cell therapy

Synonyms

Target

HBG1

Action

stimulants

Mechanism

HBG gene stimulators

Therapeutic Areas

Hemic and Lymphatic Diseases

Active Indication

Transfusion-dependent Beta Thalassemia

Inactive Indication-

Originator Organization

Guangzhou Ruifeng Biological Technology Co., Ltd.

Active Organization

Guangzhou Ruifeng Biological Technology Co., Ltd.

Inactive Organization-

License Organization-

Drug Highest PhasePhase 1

First Approval Date-

Regulation-

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In this descriptive study, we used metagenomics to analyze the relationship between the morphological aspects of chicken feces and its respective bacterial compositions. The microbiota composition was determined by sequencing the V4 region of the 16S rRNA genes collected from fresh broiler feces at 19 d old. In total, 48 samples were collected and divided into 8 groups of 6 samples each. The morphological changes studied were feed passage (FP) and reddish mucus (RM). Each was classified into 3 levels of intensity: 1 (slight), 2 (moderate), or 3 (intense). Thus, the 8 groups studied were feed passage (FP-1; FP-2; FP-3), reddish mucus (RM-1; RM-2; RM-3), normal ileal feces (NIF), and cecal discharge (CD). The alpha diversity (Shannon's index) revealed that the CD group showed greater diversity, and was significantly different from FP-2, FP-3, and RM-1. The beta diversity showed that the CD group samples were more homogeneous than the ileal feces groups. The relative abundance analysis revealed that Firmicutes and Proteobacteria were the most abundant phyla in the ileal feces groups. In CD, Firmicutes and Bacteroidetes were the most abundant. The relative abundance at the genus level revealed 136 different bacterial genera. In the ileal feces groups, the two most abundant genera were Lactobacillus and Escherichia/Shigella, except in the FP-1 and RM-2 groups, which had the opposite order. Unlike the others, the CD group had a higher abundance of Bacteroides and Faecalibacterium. When comparing the NIF group with the others, significant changes were found in the fecal microbiota, with nine genera for the FP groups, 19 for the RM groups, and 61 when compared to CD. The results of the present study suggest that evaluation of fecal morphology is a fundamental task that makes it possible to act quickly and assertively, as the morphological aspects of the feces may be related to the composition and structure of fecal microbiota.

01 Jan 2002·Biological and Pharmaceutical BulletinQ4 · MEDICINE

Round Shape Enlargement of the Yeast Spheroplast of Saccharomyces cerevisiae by HM-1 Toxin.

Q4 · MEDICINE

Article

Author: Komiyama, Tadazumi ; Kimura, Tetsuya ; Furuichi, Yasuhiro

The effects of HM-1 killer toxin (HM-1) on yeast spheroplasts of Saccharomyces cerevisiae were examined under osmotically stabilized conditions. Prolonged incubation of spheroplasts in nutrient-rich media resulted in an increase in volume, accompanied by aberrant morphological changes. By contrast, spheroplasts were enlarged, maintaining a round shape, when incubated in HM-1 media. The required 50% effective dose of HM-1 was as low as 2.2 x 10(-8) M, and this effect by HM-1 was specific to yeast sensitive to RM-1. Some parts of the enlarged spheroplasts were stable, but the round shape was deformed as HM-1 was removed from the medium. In both the control and HM-1-treated spheroplasts, the total protein and DNA content were increased by approximately three and four times in response to their incubations, respectively. Cytochemical analysis by 4'6-diamidino-2-phenylindol (DAPI) staining showed multiple nuclei. Consistently, actin patches of cells were evenly distributed in both the control and HM-1-treated spheroplasts. A similar enlargement of spheroplasts was observed with lipophilic antifungal compounds, aculeacin A and papulacandin B, but the effects were distinct from those of HM-1 because the spheroplasts resulted in lysis after a long incubation. The molecular mechanism(s) behind this unique observation remains to be studied, but it is clear that HM-1 is an excellent tool for studying yeast cell biology.

04 Jul 2001·JNCI Journal of the National Cancer InstituteQ1 · MEDICINE

Inhibition of Murine Prostate Tumor Growth and Activation of Immunoregulatory Cells With Recombinant Canarypox Viruses

Q1 · MEDICINE

Article

Author: Julie Ritchey ; Timothy L. Ratliff ; Inder Sehgal ; Mutsushi Kawakita ; Thomas S. Griffith ; Weicheng Zhao ; Jun Tian ; Timothy C. Thompson ; James Tartaglia

BACKGROUNDImmunization with modified tumor cells carrying recombinant immunomodulatory genes is being explored as cancer immunotherapy. In this study, we examine whether canarypox ALVAC viruses carrying immunostimulatory cytokine genes (granulocyte-macrophage colony-stimulating factor, interleukin 2, interleukin 12, and tumor necrosis factor-alpha) can induce antitumor immunity (to rechallenge) in the RM-1 model of a highly aggressive, weakly immunogenic murine prostate cancer.METHODSFor antitumor activity studies, RM-1 murine prostate cancer cells were infected with the parental ALVAC virus or one or two recombinant ALVAC-cytokine viruses and then injected into male C57BL/6 mice. For rechallenge studies, other mice were first given an injection subcutaneously with irradiated (nonproliferating) recombinant ALVAC-infected RM-1 cells and then (10 days later) with untreated RM-1 cells. For the determination of which immune cells were required for antitumor activity, mice were immunodepleted of CD4, CD8, or natural killer (NK) NK1.1 cells with the corresponding monoclonal antibodies and were then given an injection of ALVAC-cytokine-infected RM-1 cells. For all experiments, tumor outgrowth and animal survival were monitored.RESULTSAfter subcutaneous injection into mice, RM-1 cells infected with one (except ALVAC-interleukin 2) or two ALVAC-cytokine recombinants had statistically significantly greater antitumor activity than RM-1 cells infected with parental ALVAC (P<.001 for all; two-sided test). The antitumor activity of RM-1 cells infected with any two ALVAC-cytokine recombinants was greater than, but not statistically significantly different from, that of RM-1 cells infected with any one ALVAC-cytokine recombinant. NK1.1 cells were necessary for antitumor activity, but tumor-specific CD4(+) regulatory T cells were also induced that inhibited CD8(+) RM-1-specific cytotoxic T cells, resulting in the lack of immunity to a rechallenge by RM-1 cells.DISCUSSIONCanarypox viruses can transfer immunostimulatory cytokine genes into RM-1 prostate cancer cells. When such cells were injected into mice, the cytokines induced an antitumor response against this highly aggressive, weakly immunogenic tumor. This response, however, did not protect the mouse against a rechallenge with RM-1 cells because suppressor CD4(+) T cells were induced that inhibited tumor-specific CD8(+) cytotoxic T cells.

100 Deals associated with HBG gene modified autologous CD34+ hematopoietic stem cells(Guangzhou Ruifeng)

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Transfusion-dependent Beta Thalassemia	Phase 1	China	Guangzhou Ruifeng Biological Technology Co., Ltd.	15 Dec 2022

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


RM-001 (ASGCT2025)	Phase 1	Transfusion-dependent Thalassemia	19	RM-001	(raghnpwhav) = rkevjwtpgy gthtbhyblr (dazsofssir ) View more	Positive	13 May 2025
ChiCTR2100052858 \| ChiCTR2300069244 \| ChiCTR2100053406 (ASH2024)	Not Applicable	Transfusion-dependent Thalassemia	-	RM-001	zqxgjwneod(hccqcnjgmj) = No RM-001-related serious adverse event report. All of adverse events have been resolved. There were no deaths, discontinuations, or malignancies. tqzuknuylb (nwehdojevu )	-	09 Dec 2024
ChiCTR2100052858 \| ChiCTR2300069244 \| ChiCTR2100053406 (EHA2024) Manual	Phase 1	Beta-Thalassemia	16	RM-001	(ytprurjrrn) = xjhgavitvr votsjgbgwa (gawbpjfcvh ) Met View more	Positive	14 May 2024
ASH2023	Not Applicable	Transfusion-dependent Beta Thalassemia	7	RM-001	(tluncdrnmt) = qbaybsgkhj ciqenerkdx (itumnvvkfo ) View more	-	11 Dec 2023
EHA2022	Not Applicable	Transfusion-dependent Thalassemia	2	RM-001	(zbfosrpqgz) = oogvskusvx uoiaucoade (muaukmuquc )	Positive	12 May 2022