Targeting cardiac fibrosis with next generation RNA therapeutics

Introduction

The aim of FIBREX is developing a novel, highly innovative and close to clinical readiness noncoding RNA (ncRNA)-based antisense oligonucleotide therapeutic for the treatment of heart failure (HF) derived from cardiac fibrosis. HF is the most common cause of morbidity and mortality with a pressing social and economic burden.

Background

HF remains a deadly disease with no curative treatment, underlining the need for innovative therapeutic strategies. The revolutionary technology we are proposing acts by reversing cardiac fibrosis, the main driver of HF development and progression. The excess fibrotic tissue leads to continuous stiffening and impairment of the heart muscle.

Research Findings

In our prior work, ncRNA Meg3 was identified as the most promising target, expressed mainly by cardiac fibroblasts and by consistent transcriptional dysregulation in HF models. The critical role for Meg3 in cardiac fibrogenesis was validated both in vitro and in vivo, and a lead compound targeting Meg3 showed promising efficacy in relevant models, human tissues, and excellent exploratory safety data.

Project Proposal

We propose a development project for an antiMEG3 inhibitor to reach close to clinical readiness by completing non-clinical pharmacodynamic and safety studies. This work will be carried out by an experienced team headed by Prof. Thum, with a track record of discovering and licensing various ncRNA inhibitors programs, and creating a clinical stage spin-off company Cardior, which has reached phase 2 development and Series B funding.

Future Outlook

We are confident that our technology, with the requested support, will be further advanced into an innovative novel drug candidate. Driven by the substantial value increase, the project will be transitioned into a spin-off drug development or a licensing deal with the industry.

Conclusion

The development of the unique, RNA-based antiMEG3 therapeutic approach for HF derived from excessive fibrosis offers a new opportunity to revolutionize medical practice, reduce healthcare costs, and improve patients' lives.