Our target

The interactions between microRNA (miRNA) and messenger RNA (mRNA) is essential to orchestrate protein expression

Genetic information encoded in DNA is transcribed to RNA, which is then translated to protein. The interactions between microRNA (miRNA) and messenger RNA (mRNA) constitute a transient set of information that cells use to elaborately orchestrate protein expression. Dysregulation of this interaction can induce disease conditions.


The miRNA features that control several targets are appropriate for multifactorial diseases like Alzheimer’s disease. And the miRNAs don’t affect the genome thus it’s not a permanent genetic effect. Also, it has minimal risk of side effects through the miRNA’s transient nature. Furthermore, this transient RNA therapy shows less immune reaction and toxicity in comparison with gene-based therapy and protein-based therapy.


We, at BIORCHESTRA, develop RNA-based therapeutics that can remedy such dysregulation. RNA-based therapeutics can be categorized by the mechanism of action. Gene delivery, noncoding RNAs, RNA editing, and Antisense oligonucleotides (ASOs) bind to nucleic acids; aptamers target proteins; mRNAs encode proteins. We design a strategy for drug delivery of a selected RNA modality using our proprietary drug delivery system (DDS).

Our R&D Strategy

An Integrated Approach to RNA Therapeutics

BIORCHESTRA uses fully integrated platforms for drug development. Our RNA modalities are loaded onto a proprietary delivery platform. The drug product is then complemented with a companion diagnostic; the biomarker-driven approach increases the clinical success rate.

Our goal is to change the therapeutic paradigm from treating the symptoms to providing fundamental cure for neurodegenerative diseases.

Novel Target Discovery
BIORCHESTRA is dedicated to developing innovative disease-modifying therapies with clinically validated novel targets
Drug Delivery system
We use a proprietary Drug Delivery System(BDDS™) to efficiently deliver our drugs to target organs, including CNS and muscle
RNA Therapeutics
miRNA, ASO, siRNA, mRNA and other relevant RNA modalities are optimized for delivery with BDDS™
Clinically validated biomarkers are used to develop diagnostics and monitoring tools
Clinical Approaches
Biomarker-driven clinical trials