Small activating RNA from concept to phase 2 clinical trials

Title : Small activating RNA from concept to phase 2 clinical trials

Abstract:

Small activating RNAs (saRNA) are double stranded 21 nucleotide RNA that either target promoters or enhance genes leading to mRNA upregulation. saRNAs can be delivered with liposomes into the systemic circulation or subcutaneously by conjugation with aptamers or GalNAC. MTL-CEBPA is an investigative drug that resulted from the conjugation of saRNA CEBPA with NOV 340 lipsomes that targets tumour associated macrophages in order to alter favourably the tumour microenvironment. The relevance of the technology is that it can deliver transcription factors to the bone marrow CD34+ cells and that the effect lasts at least 6 weeks in vivo. Clearly small activating RNS can be applied to iPSCs and other aspects of regenerative medicine. MTL-CEBPA has been administered safely in over 100 patients with advanced cancer and improved clinical outcome in a sub-set of patients when co-administered with TKI or check point inhibitor.

Biography:

Nagy is a founder and Head of R&D of MiNA Therapeutics. For over three decades Nagy has been at the forefront of clinical research and clinical practice in cancer. He pioneered the first clinical trial in the use of adenovirus and plasmid for the treatment of liver cancer, as well as the use of plasmid gene therapy in hydrodynamic gene delivery. Currently, he is driving the development of an saRNA drug (a new class of medicines) which is currently being trialed in patients with liver cancer in eight UK centres, and sites in Singapore and Taiwan (OUTREACH study, ClinicalTrials.gov ID NCT02716012) and in a second trial in patients with solid tumours (TIMEPOINT study, ClinicalTrials.gov ID NCT 04105335) in the UK, USA, Europe, Singapore and Taiwan.