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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:

Targeted Mechanisms

RNA can specifically target and modulate specific genes through complementary sequence binding. This offers the advantage of precisely targeting and controlling genes that are the root cause of diseases.

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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PUBLICATIONS

Our Publications

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The microRNA-485-3p concentration in salivary exosome-enriched extracellular vesicles is related to amyloid β deposition in the brain of patients with Alzheimer’s disease

July 13, 2023
Objectives Alzheimer’s disease (AD) is an irreversible neurodegenerative disease characterized by progressive long-term memory loss…
AD

The role of microRNA-485 in neurodegenerative disease

July 7, 2022
Neurodegenerative diseases (NDDs) are age-related disorders characterized by progressive neurodegeneration and neuronal cell loss in…
AD Biomarker

Targeting MicroRNA-485-3p Blocks Alzheimer’s Disease Progression

December 4, 2021
1. BIORCHESTRA Co., Ltd., 17, Techno 4-ro, Yuseong-gu, Daejeon 34013, Korea 2. Department of Infection…
AD

Innate Immunity and Cell Death in Alzheimer’s Disease

October 20, 2021
The innate immune system plays key roles in controlling Alzheimer's disease (AD), while secreting cytokines…

WHAT’S NEW

News & Press releases

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IR Event

Polymer Chemistry&Formulation in Drung Delivery Summit, May 21-23 2024 I Boston, MA

February 21, 2024
Please join us for an enlightening session on Brain Target Delivery of RNA Therapeutics using…
IR Event

RNA Leaders Europe Congress, March 13-14 2024 I Basel, Switzerland

February 21, 2024
BIORCHESTRA is excited to announce that David Oxley, our Chief Business Officer (CBO), will be…
Press Release

BIORCHESTRA Wins Grand Prize in 2023 Merck Advance Biotech Grant Program

July 17, 2023
Recognition of outstanding in-vivo outcomes from a lead RNA program in CNS Underpinned by proprietary…
AD

The microRNA-485-3p concentration in salivary exosome-enriched extracellular vesicles is related to amyloid β deposition in the brain of patients with Alzheimer’s disease

July 13, 2023
Objectives Alzheimer’s disease (AD) is an irreversible neurodegenerative disease characterized by progressive long-term memory loss…