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Overview

Basic Info

Drug Type

ASO

Synonyms

ENA oligonucleotides, duchenne muscular dystrophy therapeutics - Daiichi Sankyo/Orphan Disease Treatment Institute

+ [1]

Target

dystrophin

Mechanism

DMD gene modulators

Therapeutic Areas

Nervous System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]

Active Indication

Muscular Dystrophy, Duchenne

Inactive Indication-

Originator Organization

Daiichi Sankyo Co., Ltd.

Active Organization

Daiichi Sankyo Co., Ltd.

Inactive Organization

Orphan Disease Treatment Institute Co., Ltd.

Drug Highest PhasePhase 2

First Approval Date-

Regulation-

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Structure/Sequence

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Sequence Code 32858997

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Research on the occurrence and seasonal monitoring of pharmaceutically active compounds (PhACs) in estuarine and coastal waters has intensified recently. However, few studies have been conducted with PhACs flowing into the marine waters of South America (such as Brazil). Against this backdrop, the aims of this study were: (i) evaluate, for the first time, the seasonal occurrence throughout a year and the potential ecological risks of ten selected PhACs in marine bathing waters from Santos Bay, São Paulo, Brazil (a tropical low-wave energy semi-closed bay); and (ii) develop a list of high-priority PhACs for the monitoring based on "occurrence, persistence, bioaccumulation, and toxicity" criteria (OPBT). Four water sampling campaigns were carried out throughout the four seasons of the year. The results showed that: (i) ten PhACs (namely, caffeine/CAF (87.20-567.23 ng/L); carbamazepine/CAR [below the limit of quantification ( ACE > DIC and CAR to algae, crustaceans, and fishes; (iv) Finally, regarding the OPBT ranking, the DIC was the highest-priority PhAC in Santos Bay, followed by: ORP > LOS > CIT > CAR > FUR > ATE > CAF > ACE > ENA.

01 Dec 2024·Pathology

Macaque liver substrate for evaluating dense fine speckled-like patterns on HEp2010 cells

Article

Author: Lee, Adrian Y.S. ; Silvestrini, Roger A. ; Anantharajah, Anthea ; Campbell, David ; Lee, Adrian Y S ; Silvestrini, Roger A ; Culican, Suzanne ; Lin, Ming Wei

Antinuclear antibody (ANA) detection by indirect immunofluorescence (IIF) is instrumental in the evaluation of systemic autoimmune diseases (SADs). The dense fine speckled (DFS) ANA staining predominantly associates with anti-DFS70, an autoantibody that is thought to exclude the presence of SAD. However, the DFS pattern may mask the presence of other ANA patterns that may be clinically relevant. Our laboratory uses the HEp2010 substrate which contains both HEp2 and liver substates. The aim of this study was to determine whether negative liver nucleus immunofluorescence could exclude the presence of antibodies to extractable nuclear antigens (ENA) in sera with DFS-like patterns. One hundred consecutive sera samples suspicious for DFS pattern, along with 15 sera of control patterns (positive metaphase bars) were included in the study. Each sample was examined separately on HEp2010 (Euroimmun) and liver by two independent readers. Anti-DFS70 was assessed by line and chemiluminescent immunoassays. DFS-like sera were more likely to be liver nucleus-negative compared with control sera. Of the liver-negative sera, 61/64 (95.3%) were deemed anti-ENA negative. Using the liver substrate in the evaluation of anti-ENA had a sensitivity of 90.0% and a negative predictive value of 95.4%. In our cohort, concurrent evaluation of sera with the liver substrate helped rule out the presence of other anti-ENA. This technique may be a safeguard for DFS-like ANA patterns that may mask underlying anti-ENA on IIF.

01 Nov 2024·Toxicology

Unexpected antagonism of deoxynivalenol and enniatins in intestinal toxicity through the Ras/PI3K/AKT signaling pathway

Article

Author: Sun, Xiulan ; Tang, Lili ; Tang, Luyao ; Ye, Yongli ; Sheng, Lina ; Wang, Jia-Sheng ; Ning, Xiao ; Sun, Jiadi

Deoxynivalenol (DON) is a kind of widespread traditional Fusarium mycotoxins in the environment, and its intestinal toxicity has received considerable attention. Recently, the emerging Fusarium mycotoxin enniatins (ENNs) have also been shown to frequently coexist with DON in animal feed and food with large consumption. However, the mechanism of intestinal damage caused by the two mycotoxins co-exposure remains unclear. In this study, Caco-2 cell line was used to investigate the combined toxicity and potential mechanisms of four representative ENNs (ENA, ENA₁, ENB, and ENB₁) and DON. The results showed that almost all mixed groups showed antagonistic effects, particularly ENB at 1/4 IC₅₀ (CI = 6.488). Co-incubation of ENNs mitigated the levels of signaling molecule levels disrupted by DON, including reactive oxygen species (ROS), calcium mobilization (Ca²⁺), adenosine triphosphate (ATP). The differentially expressed genes (DEGs) between the mixed and ENB groups were significantly enriched in the Ras/PI3K/Akt signaling pathway, including 28 up-regulated genes and 40 down-regulated genes. Quantitative real-time PCR further confirmed the lower expression of apoptotic gene in the mixed group, thereby reducing the cytotoxic effects caused by DON exposure. This study emphasizes that co-exposure of ENNs and DON reduces cytotoxicity by regulating the Ras/PI3K/Akt signaling pathway. Our results provide the first comprehensive evidence about the antagonistic toxicity of ENNs and DON on Caco-2 cells, and new insights into mechanisms investigated by transcriptomics.

100 Deals associated with Renadirsen

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Muscular Dystrophies	Preclinical	JP	Orphan Disease Treatment Institute Co., Ltd.	28 Jan 2016
Muscular Dystrophy, Duchenne	Preclinical	JP	Orphan Disease Treatment Institute Co., Ltd.	01 Oct 2015

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02667483 (CTgov)	Phase 1/2	Muscular Dystrophy, Duchenne	8	DS-5141b (All: DS-5141b 2.0 mg/kg)	zjcdvopzxa(ruvjcubgnw) = zehttevyrk szlqjbhhlz (cynovkhixc, gpdzwjaepa - wpzooomfmq) View more	-	07 Mar 2024
				DS-5141b (All: DS-5141b 6.0 mg/kg)	zjcdvopzxa(ruvjcubgnw) = tzfdotztgy szlqjbhhlz (cynovkhixc, hzipiipxvk - wzxabtpkej) View more
ASH2023	Phase 1/2	-	51	ASTX727 + VEN + IVO	(qfvvgxzouf) = moozjfydua fmxlywxsoc (ekekpyojdm ) View more	-	11 Dec 2023
				ASTX727 + VEN + ENA	(qfvvgxzouf) = uqffluqfac fmxlywxsoc (ekekpyojdm ) View more