TUG1 ASO / NANO MRNA, Nagoya University - LARVOL DELTA

R-loops: Biological Functions, Regulatory Mechanisms, and Therapeutic Implications in Brain Diseases-A Review. (PubMed, Mol Cell Probes) - "R-loops are central to brain disease pathogenesis, offering promising therapeutic targets. Future research should prioritize precision R-loop modulators, non-invasive biomarkers, and combinatorial strategies."

Journal • Review • Brain Cancer • CNS Disorders • Glioma • Huntington's Disease • Movement Disorders • Neuroblastoma • Oncology • Solid Tumor • FUS • METTL3 • TARDBP • TGFB1 • ZPR1

TUG1-mediated replication stress regulation as a potential vulnerability in bladder cancer (AACR 2026) - "Functional assays using a TUG1-targeting antisense oligonucleotide (TUG1-ASO) demonstrated efficient depletion of endogenous TUG1, reduced cell viability, and downregulation of cell-cycle-related transcripts. Loss of TUG1 also induced replication stress and DNA damage markers, including pRPA32 and γH2AX, and sensitized BCa cells to RS-inducing conditions.Taken together, these findings indicate that BCa cells depend on TUG1 to maintain proliferation under high RS pressure. Therapeutic depletion of TUG1 disrupts this adaptive mechanism and impairs tumor cell growth, supporting TUG1 as a potential therapeutic target in BCa."

Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • ERCC2 • RB1 • TP53 • TUG1

Investigator-initiated phase I trial of an oligonucleotide therapeutic targeting long noncoding RNA TUG1 for recurrent glioblastoma (AACR 2026) - "We are conducting a multicenter Phase I trial to determine the safety and maximum tolerated dose (MTD) of TUG1ASO. This trial enrolled patients aged 18 to 75 years who relapsed after receiving standard postoperative treatment, chemoradiotherapy with temozolomide. So far, this trial had enrolled 4 cases at Level 1, 3 cases at Level 2, 6 cases at Level 3, and 3 cases at Level 4. Adverse events leading to delayed administration included 3 cases of leukopenia, 1 case of neutropenia, 6 cases of hypertriglyceridemia, 1 case of increased blood cholesterol, 1 case of hyperuricemia, 1 case of pharyngitis, and 2 cases of cerebral infarction. One case of an allergic reaction led to discontinuation."

First-in-human • New P1 trial • P1 data • Brain Cancer • Glioblastoma • Oncology • Solid Tumor • TUG1

Synergistic impact of long non-coding RNA TUG1 depletion in overcoming temozolomide chemoresistance in glioblastoma (AACR 2024) - "Depleting TUG1 by using antisense oligonucleotide (TUG1-ASO) sensitized U251MG-R cells to TMZ. The combination of antiTUG1-DDS with TMZ displayed the most effective suppression of tumor growth compared to either TMZ or antiTUG1-DDS alone. Targeted therapy aimed at TUG1 may be a promising approach for overcoming TMZ resistance in GBM."

Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • TUG1

Investigator-initiated phase I trial of an oligonucleotide therapeutic targeting long noncoding RNA TUG 1 for recurrent glioblastoma. (PubMed, BMC Cancer) - "TUG1ASO is the first oligonucleotide therapeutic with drug delivery system targeting TUG1, expected to show an efficacy in rGB patients. In this first-in-human study, safety, tolerability and MTD of this new targeted therapy will be confirmed to find the recommended dose for the further clinical trial. This study may contribute to develop a new treatment option for rGB patients."

Clinical protocol • Journal • New P1 trial • P1 data • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • TUG1

INITIATION OF INVESTIGATOR-INITIATED CLINICAL TRIAL OF NANO MRNA-POST-FIELD PLUS CONVERSION NUCLEIC ACID DRUG 'TUG1 ASO' [Google translation] (Yahoo Finance) - "NANO MRNA..TURNED POSITIVE IN THE SECOND ROUND. At 12:30 p.m. on the 15th, the company announced that it had completed a 30-day investigation by the Pharmaceuticals and Medical Devices Machinery (PMDA) regarding the clinical trial protocol notification for the investigator-initiated clinical trial of the nucleic acid drug 'TUG1ASO' for the treatment of recurrent glioblastoma....As a result, investigator-initiated clinical trials are scheduled to begin at Nagoya University Hospital, Kyoto University Hospital, and National Cancer Center Hospital."

Clinical protocol • New trial • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

NANO MRNA---A paper related to the pipeline ’TUG1 ASO’ being developed as a treatment for glioblastoma was published in a British scientific journal [Google translation] (Yahoo Finance) - "On the 23rd, NANO MRNA...published a paper related to 'TUG1 ASO', which is progressing research and development toward the start of a doctor-initiated clinical trial in 2023, in the online version of the British scientific journal 'Nature Communications'. Announced that Nagoya University and Hamamatsu University School of Medicine announced that...The paper clarifies at the molecular level how the long-chain non-translated RNA 'TUG1' works and why cancer cells die when its action is suppressed. It clarifies the usefulness of 'TUG1 ASO' that suppresses the action...The company will continue to prepare for the start of clinical trials....In addition, regarding this mechanism, we plan to distribute an explanation video on the website on Friday, August 25th."

New trial • Preclinical • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor