Next generation
gene therapy company
RNatives is a platform company utilizing the novel biology of nuclear microRNAs to modulate gene expression of desired target genes.
Our current focus is to upregulate endogenous VEGFA-gene. By upregulating the cell´s own VEGFA by small RNA, diseases such as myocardial ischemia and peripheral artery disease can be treated. VEGFA upregulation in the affected tissue leads to neovascularization and recovery from the disease condition.
RNatives propriety platform technology is based on years of academic research published by Turunen et al.
Our laboratory
Our new molecular biology laboratory and BSL-2 clean room streamline the discovery and the development of RNA activation drugs for novel indications.
Pipeline
Our long track record with RNA activation of VEGFA for the treatment of cardiovascular disease drive the development of our lead programs. We have also discovered novel nuclear microRNAs for RNA activation of therapeutic genes for the treatment of neurological disease.
Mikko Turunen explains the background to his invention:
“Back in mid-2000s I was excited of findings from professor Kevin Morris who showed that endogenous gene can be transcriptionally silenced by chromatin targeted small RNAs (Science 2004). Importantly, soon after, professor Long-Cheng Li showed that cell´s own genes can also be activated by small RNAs targeted to gene promoter (PNAS 2006).
I immediately realized the potential for gene therapy applications and cloned small hairpin RNAs targeted to the promoter of VEGFA, to a viral vector. Hence, we were able to be the first to show that these promoter targeted small RNAs are very efficient also in vivo in animal models, first in the treatment of hindlimb ischemia (Circ.Res 2009) and later in the treatment of myocardial infarction (PLOS ONE 2014).
These data, and our other experiments we performed during the years, led us to the conclusion that our hairpin-RNAs have mimicked naturally existing microRNAs and that microRNAs must have a role in the nuclear regulation of gene expression. In fact, we have recently shown that mature microRNAs are very abundant in the nucleus of cell and they take part in the regulation of hypoxic response (Sci.Rep. 2019). Our competitive edge as a company is to utilize this novel microRNA biology to the treatment of various disease.”