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BRIEFING ON RNA TRIALS – JANUARY 2019
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BRIEFING ON RNA TRIALS – JANUARY 2019

BioPharmAnalyses is proud to announce the launch 

of the first issue of its 

“BRIEFING ON RNA TRIALS”

This 86-page document provides you an accurate overview

to explore the latest trials in the RNA therapeutics area.

   « BRIEFING ON RNA TRIALS » gives you a breakdown of trials:

• by disease
• by product
• by  target
• by company
• by trial location
• by licensing agreement

RNA THERAPEUTICS TRIALS BY NUMBERS

86 pages detailing 101 clinical trials conducted in 44 countries

42 companies and academic institutions developing 76 RNA products
(antisense oligonucleotides, RNAi/siRNA, microRNA, mRNA…) 

55 targets and 57 diseases in a wide range of therapeutic areas
(cancers, cardiovascular and metabolic diseases, infectious diseases,
genetic diseases, ophthalmological diseases….)

 The trials presented in this study have been registered and updated

on Clinicaltrials.gov from 01/01/2017 to 12/31/2018.

EXAMPLE OF BREAKDOWN BY DISEASE

LIVER CANCER (Target: CEBPA gene)

First-in-Human Safety and Tolerability Study of MTL-CEBPA in Patients With Advanced Liver Cancer 

ID :  NCT02716012 – MNA-3521-011 – 2015-003051-21 EudraCT Number) – 20332 (Other Identifier: UK NIHR CRN) – OUTREACH
Mina Therapeutics (UK) 
Product: MTL-CEBPA (Small activating RNA (saRNA) – short double stranded RNA designed to activate CCAAT enhancer binding protein alpha (CEBPA) gene)
Phase: I
Status: Recruiting 

Start/Planned completion: MAR 2016/DEC 2019 
Estimated Enrollment: 51
Sites: 9 (SG, TW, UK)
Updated: September 10, 2018
Contact: clinicaltrials@minatx.com
The study is in two parts: dose escalation followed by a dose expansion; both parts of the study will recruit advanced hepatocellular carcinoma patients with cirrhosis. All participants will be refractory to or ineligible for loco-regional therapy including surgery, radiofrequency tumour ablation, transarterial chemoembolisation or sorafenib.
Related Informations/Publications
DEC 2018 – MiNA Therapeutics announced enrolment of the first patients treated with MTL-CEBPA in combination with Sorafenib in OUTREACH
Link: Press Release
 Oncol Rep. 2018 Dec 13. doi: 10.3892/or.2018.6930.
MicroRNA‑486‑5p functions as a tumor suppressor of proliferation and cancer stem‑like cell properties by targeting Sirt1 in liver cancer. Yan X et al. College of Life Science and Bioengineering, Beijing University of Technology, Beijing, CN.
– Results/Comments : MicroRNAs are small non‑coding RNAs that target the 3’untranslated region of mRNAs. Their dysregulation has been implicated in several types of cancer including liver cancer, but it still remains unknown if they play a role in targeting liver cancer stem‑like cells (CSCs). These researchers compared the miRNA profiles between liver cancer samples and adjacent non‑tumor tissues using The Cancer Genome Atlas (TCGA) datasets. The results of their study indicated that the miR‑486‑Sirt1 axis was involved in suppressing CSC traits and tumor progression.
Link: Abstract 
Oncol Rep . 2018 Dec 12. doi: 10.3892/or.2018.6928.
Inhibition of PIKfyve using YM201636 suppresses the growth of liver cancer via the induction of autophagy.  Hou JZ et al. Institute for Innovative Drug Design and Evaluation, School of Pharmacy, Henan University, Kaifeng, Henan, CN.
– Results/Comments : This study incidates that phosphatidylinositol‑3‑phosphate 5‑kinase (PIKfyve) may be a potential therapeutic target for the treatment of liver cancer. Link: Abstract
OCT 2018 – MiNA Therapeutics announces Sosei will not exercise acquisition option. Link: Press Release  
SEP 2018 – MiNA Therapeutics provided  an update from the ongoing Phase I study of  MTL-CEBPA in advanced liver cancer patients at International Liver Cancer Association Conference. Link: Press Release

« BRIEFING ON RNA TRIALS » 
(Updated DEC 31 2018)
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