South Korean bio-venture BIORCHESTRA Co. Ltd., is an RNA therapeutics firm focused on neurodegeneration. BIORCHESTRA discovered a disease-associated microRNA that was found to be significantly up-regulated in samples from Alzheimer’s disease patients. It then conducted gain-of-function studies and discovered the over-expressed miRNA may be an underlying cause of the disease, deciding to develop an anti-miRNA antisense oligonucleotide combined with its proprietary platform technology to allow high brain cell-selective delivery.
BIORCHESTRA CEO Dr. Branden and CMO Dr. O’Dea will attend the Clinical Trials on Alzheimer’s Disease conference (CTAD) to introduce a pathological miRNA ASO therapeutic effect. BIORCHESTRA discovered the pathological miRNA, a “molecular switch by epigenetic changes” in a cell. The pathological miRNA is over-expressed in human brain and CSF sample, and it induces Alzheimer’s disease pathology such as amyloid-beta (Aβ) 1-42, tau, neuro-inflammation, and synaptic plasticity impairment. This gain of function study suggests that pathological miRNA is a useful biomarker as well as a causative factor of the inflammatory pathophysiology in Alzheimer’s disease(AD). BIORCHESTRA studies with BMD-001 (pathological miRNA antisense oligonucleotide (ASO)) have demonstrated significant reductions in neuroinflammation and Aβ and tau pathology, as well as enhancement in neurorestoration and cognitive abilities in Alzheimer’s animal models, along with a significant reduction in neuroinflammation.
“BMD-001 is a therapeutic candidate for AD pathology and cognitive decline, establishing a new paradigm change in the AD field. For the clinical development of BMD-001, we have also developed a nanoparticle formulation to deliver ASO drugs in sufficient amounts to be therapeutically effective.” Dr. Branden said.