BIORCHESTRA will present data on the therapeutic efficacy of their proprietary ASO against pathological miRNA, a key modulator of neuroinflammation and neurodegeneration, at the CTAD Meeting in Boston
BOSTON,Nov. 5,2021 --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 Diseaseconference(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.
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