SCIENCE
Our Science
At BIORCHESTRA, we are investing at the forefront of next-generation genetic science and non-lipid nanoparticle delivery technologies, to deliver innovative RNA therapies and help millions of patients around the world.
Our diversified, symbiotic research activities include microRNA target and biomarker discovery, inert nanoparticle delivery chemistry, cell and tissue targeting capabilities, cargo-encapsulation strategies, and further research into the diseases that we aim to treat.
All for a common purpose: to bring first or best-in-class medicines to patients with high unmet medical needs in neurodegenerative and rare diseases, worldwide.
SCIENCE
Our Discovery
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:
Potential for personalized therapies
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.
Opportunity to use different RNA interference modalities
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.
Target Discovery
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
SCIENCE
Our Target
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
Antisense RNA binds to specific mRNA molecules through complementary sequences, efficiently inhibiting translation and thereby suppressing or regulating the expression of specific proteins.
RNA interference (RNAi)
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.
RNA ribozymes
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.
Oligonucleotide-based therapies
Short synthetic RNA or DNA molecules are used to interact with specific mRNA or microRNA, thereby inhibiting or modulating gene expression.
CRISPR-Cas9
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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