RNA molecules can perform various functions and regulate the expression of specific proteins through transcriptional and translational control. This allows for the modulation and targeting of aberrant gene expression associated with diseases.
The discovery of RNA candidates offers several key benefits:
Various pharmacological modalities may be used to achieve RNA interference. mRNA, siRNA, ASO and aptamers are examples of RNA-based therapies being developed, offering potential applications in gene therapy, mRNA modulation, protein inhibition, preventive vaccination, and more.
RNA interference can be utilized in various treatment modalities. mRNA therapy, RNA interference, and RNA aptamers are examples of diverse RNA-based therapies being developed, offering potential applications in gene therapy, protein inhibition, preventive vaccination, and more.
BIORCHESTRA discovered miR-485-3p, the world’s first microRNA linked to degenerative brain diseases. MicroRNAs are short single-stranded RNA molecules involved in the regulation of gene expression. Over 2,500 microRNAs are present in the human genome, with well-preserved sequences across various species, indicative of their importance in evolution. MiR-485-3p, in particular, was found to be elevated in patients with Alzheimer’s Disease, Parkinson’s Disease, and Amyotrophic Lateral Sclerosis.
miR-485-3p levels in healthy subjects vs. AD patients1
At BIORCHESTRA, we have developed a range of therapeutic strategies using various RNA interference modalities and chemistries, to selectively modulate specific RNAs for the purpose of functional regulation and disease treatment.
Antisense RNA binds to specific mRNA molecules through complementary sequences, efficiently inhibiting translation and thereby suppressing or regulating the expression of specific proteins.
RNAi involves the use of double-stranded RNA molecules that bind to specific mRNA molecules, inducing their degradation. This allows for the inhibition or modulation of specific gene expression.
Ribozymes are RNA molecules that can recognize and catalytically cleave or modulate specific RNA regions. This enables the control or elimination of specific RNA functions.
Short synthetic RNA or DNA molecules are used to interact with specific mRNA or microRNA, thereby inhibiting or modulating gene expression.
The CRISPR-Cas9 system is a revolutionary tool in RNA targeting technology. It is used to specifically target and modify RNA sequences and finds extensive application in gene editing and modification.
RNA targeting technology holds a great potential for understanding the causes of diseases and regulating the function of specific RNA molecules for disease treatment and, potentially, prevention.
We believe that, with RNA therapeutics, we can surpass existing limitations of current pharmacological treatments of neurodegenerative diseases.
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