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. 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
Audience Take Away:
- It will improve their knowledge in activating gene which could be very useful for cell differentiation and function especially in the context of pleuripotent stem cells.
- It would be highly relevant in the field of stem cell and regeneration. In particular it will help them to avoid the high risk of lentivirus.
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. Nagy is a founder and was the Head of R&D of MiNA Therapeutics. Whilst at MiNA he was 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. He has published widely in gene therapy, stem cell therapy, oligonucleotides, endoscopy and surgery. Currently he is the CEO of Apterna Limited, a company focussed on novel oligonucleotide deliver and Dawn Therapeutics specialising in gene therapy. Nagy is Lead Clinician and Head of the Department of HPB Surgery at Imperial College London.