- |||||||||| Nypta (tideglusib) / ASD Therap, miglustat / Generic mfg.
Journal: Naringenin and SMER28 target lysosomal reformation and rescue SPG11 and SPG15 Hereditary Spastic Paraplegia phenotypes. (Pubmed Central) - Jul 28, 2025 Their effects were compared with those of SMER28 and of miglustat, the latter tested in a phase II clinical trial in SPG11 patients, in both SPG15 and SPG11 patient's derived cells and in the corresponding Drosophila models...Both compounds induced lysosomal tubulation, downstream of mTOR, promoting lysosomal reformation. Our work indicates that lysosomal reformation is a good strategy for HSPs with impaired lysosomal function and identifies naringenin as new modulator of this process, offering further hand to planning phase II clinical trials in SPG11-SPG15 patients.
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Trial completion date, Trial primary completion date: REACH CDM X: Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (clinicaltrials.gov) - May 27, 2025 P2/3, N=76, Recruiting, This suggests that tideglusib, either alone or in combination with bone graft materials, could serve as a promising alternative for the repair of bone defects. Trial completion date: Mar 2025 --> Dec 2026 | Trial primary completion date: Mar 2025 --> Dec 2026
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Journal: Bioactive deproteinized bovine bone mineral based on self-assembled albumin nanoparticles promoted bone regeneration via activation of Wnt/?-catenin pathway. (Pubmed Central) - Apr 27, 2025 In this study, we synthesised bovine serum albumin nanoparticles (BNP) loaded with Tideglusib (TD), TD and BNP were bound together by self-assembly, and mixed with deproteinized bovine bone mineral (DBBM) to form a bone substitute material (TD-BNP@DBBM) that had low cytotoxicity, promoted cell proliferation and migration, induced cell differentiation, and regulated osteogenesis...Furthermore, this result suggested a link between the Wnt/?-catenin pathway and the osteogenic effect, providing a basis for subsequent investigations into the mechanism of bone regeneration induced by osteogenic biomaterials. TD-BNP@DBBM might be a promising new approach for treating bone defects.
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Enrollment open, Trial completion date, Trial primary completion date: Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy (clinicaltrials.gov) - Apr 3, 2025 P2, N=120, Recruiting, Together these studies document tideglusib as a major modulator of chronic ethanol consumption-evoked brain gene expression signatures, and identify possible new targets for therapeutic modulation of AUD. Not yet recruiting --> Recruiting | Trial completion date: Jul 2026 --> Jul 2027 | Trial primary completion date: Feb 2026 --> Feb 2027
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Review, Journal: GSK-3: An "Ace" Among Kinases. (Pubmed Central) - Nov 13, 2024 Significant findings of miRNA regulation by GSK-3 exemplify the underpinnings of kinase-mediated transcriptional regulation in cancers. The review provides evidence on the role of GSK-3 as a possible master regulator of proteins and noncoding RNA, thereby implicating the fate of a cell.
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Journal: The Effect of Tideglusib Application on Type 1 and Type 3 Collagen Expressions by Human Dental-Pulp Derived Stem Cells: A Preliminary Study. (Pubmed Central) - Sep 30, 2024 Immature crystallites formed in dentin treated with undoped NPs will account for a high remineralizing activity. The fact that Wnt signaling pathway activation obtained by Tideglusib application on DPSCs confirmed by the finding in the increase of Axin-2 at short and long-term evaluation periods which is resulted in the increase in the type 1 collagen expression at 24 h and decrease at 1 week together with the decrease in type 3 collagen expression at 1 week warrants further studies to evaluate the effect of Tideglusib on extracellular matrix expression.
- |||||||||| Nypta (tideglusib) / ASD Therap, Amnolake (tamibarotene) / Syros
Journal: Cocktail Cell-Reprogrammed Hydrogel Microspheres Achieving Scarless Hair Follicle Regeneration. (Pubmed Central) - Jan 16, 2024 In vitro and in vivo studies show that AHFS can regulate fibroblast fate, induce fibroblast-to-DPC reprogramming by activating the PI3K/AKT pathway, finally promoting wound healing and in situ HF regeneration while inhibiting scar formation in a two-pronged translational approach. In conclusion, AHFS provides a new and effective strategy for functional repair of skin wounds.
- |||||||||| Nypta (tideglusib) / ASD Therap
Journal: Dentin remineralization using a stimuli-responsive engineered small molecule GSK3 antagonists-functionalized adhesive. (Pubmed Central) - Dec 20, 2023 These findings suggest that Tideglusib and CHIR99021 can be applied clinically in pulp regeneration to improve strategies for vital pulp regeneration and to promote dentine repair. Doping hydrophilic polymeric NPs with tideglusib, infiltrated in etched dentin represents a reproducible technique to create reparative dentin at the resin-dentin interface, by inducing therapeutic bioactivity.
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Journal: Tideglusib-incorporated nanofibrous scaffolds potently induce odontogenic differentiation. (Pubmed Central) - Jul 24, 2023 In contrast to current pulp capping material driving dentin repair, the sophisticated, polymeric scaffold systems with soluble and insoluble spatiotemporal cues described here can direct stem cell differentiation and dentin regeneration. Hence, bioactive small molecule-incorporated nanofibrous scaffold suggests an innovative clinical tool for dentin tissue engineering.
- |||||||||| Nypta (tideglusib) / ASD Therap
Journal: Therapeutic Targeting of the GSK3?-CUGBP1 Pathway in Myotonic Dystrophy. (Pubmed Central) - Jul 18, 2023 We have previously described the benefits of the correction of the GSK3?-CUGBP1 pathway in DM1 mice (HSA model) expressing 250 CUG repeats using the GSK3 inhibitor tideglusib (TG)...Using a mouse model with dysregulated CUGBP1, which mimics alterations in DM1, we showed that the dysregulated CUGBP1 contributes to the toxicity of expanded CUG repeats by changing gene expression and causing CNS abnormalities. These data show the critical role of the GSK3?-CUGBP1 pathway in DM1 muscle and in CNS pathologies, suggesting the benefits of GSK3 inhibitors in patients with different forms of DM1.
- |||||||||| Nypta (tideglusib) / ASD Therap
Trial completion date, Trial primary completion date: TIDALS: Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - Jun 2, 2023 P2, N=98, Not yet recruiting, degraders as potential therapeutic agents. Trial completion date: Mar 2024 --> Mar 2026 | Trial primary completion date: Dec 2023 --> Dec 2025
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Enrollment status, Enrollment change, Trial completion date, Trial primary completion date: REACH CDM X: Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (clinicaltrials.gov) - May 24, 2023 P2/3, N=76, Recruiting, Trial completion date: Mar 2024 --> Mar 2026 | Trial primary completion date: Dec 2023 --> Dec 2025 Enrolling by invitation --> Recruiting | N=56 --> 76 | Trial completion date: Mar 2023 --> Mar 2025 | Trial primary completion date: Mar 2023 --> Mar 2025
- |||||||||| Review, Journal: The myotonic dystrophy type 1 drug development pipeline: 2022 edition. (Pubmed Central) - Mar 2, 2023
Three interventional first-in-human clinical trials got underway with distinct drug classes, namely AOC 1001 and DYNE-101 nucleic acid-based therapies, and the small molecule pitolisant, which joins the race toward market authorization with other repurposed drugs, including tideglusib, metformin, or mexiletine, already in clinical evaluation. Furthermore, newly disclosed promising preclinical data for several additional nucleic-acid therapeutic candidates and a CRISPR-based approach, as well as the advent into the pipeline of novel therapeutic programs, increase the plausibility of success in the demanding task of providing valid treatments to patients with DM1.
- |||||||||| Nypta (tideglusib) / ASD Therap
Enrollment closed, Trial primary completion date: REACH CDM: Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy (clinicaltrials.gov) - Feb 24, 2023 P2/3, N=56, Active, not recruiting, Furthermore, newly disclosed promising preclinical data for several additional nucleic-acid therapeutic candidates and a CRISPR-based approach, as well as the advent into the pipeline of novel therapeutic programs, increase the plausibility of success in the demanding task of providing valid treatments to patients with DM1. Recruiting --> Active, not recruiting | Trial primary completion date: Jan 2023 --> Apr 2023
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