Main Conference Day 2 - CET (Central European Time, GMT+01:00)
The most studied and well-described mode of action of miRNAs is their binding to the 3ʹ UTRs of target messenger RNAs in the cytoplasm, thereby downregulating gene expression through post-transcriptional gene silencing (PTGS). However, recent research has demonstrated that miRNAs can also bind non-coding RNAs at gene promoters in the nucleus, either activating or repressing their transcription. Especially the miRNA-induced gene transcription, named RNA activation, can provide a new way of gene therapy, where the genome of patient stays intact and only small RNA is delivered to treat the disease. At RNatives, we are harnessing this power of small nuclear RNAs to develope novel RNA medicine. In our lead program, we have worked with transcriptional induction of Vascular Endothelial Growth Factor A (VEGF-A) gene for the treatment of cardiovascular diseases. In vivo studies in mouse model of peripheral artery disease showed great therapeutic effect after treatment with single injection. This program is now moving towards clinical studies. In addition, we explore multiple other indications, such as neurological diseases and oncology, to create novel RNA activation -based therapies.
- Mikko Turunen, PhD - Founder and CSO, RNatives Inc.
This presentation will discuss how peptides unlock a completely new possibility for dissolving neurotoxic protein aggregates, which are a hallmark to various neurodegenerative disorders. The development of PRI-002 from discovery to phase II in Alzheimer’s disease will be presented.
- Philipp Buerling - CEO, Priavoid GmbH