BIORCHESTRA, a South Korean bio-venture pioneering the development of RNA therapeutics, announced on November 5th that the company will present the research data at the 14th annual Clinical Trials on Alzheimer's Disease (CTAD) conference, being held in Boston on November 9-12, 2021.
The company will present data on the therapeutic efficacy of Antisense Oligonucleotides (ASO) against pathological miRNA, a key modulator of neuroinflammation and neurodegeneration, at the CTAD.
BIORCHESTRA is an RNA therapeutics firm focused on neurodegeneration. The company discovered a pathologic microRNA that was found to be significantly up-regulated in Alzheimer's disease (AD), conducted gain-of-function studies, and found that the over-expressed miRNA may be an underlying cause of the disease. The company finally decided to develop an anti-miRNA ASO combined with its novel nanoparticle platform technology to allow high brain cell-selective delivery.
The over-expressed pathological miRNA ("molecular switch by epigenetic changes") induces AD pathology such as beta-amyloid, tau, neuro-inflammation, and synaptic plasticity impairment. The gain-of-function study suggests that pathological miRNA is a useful biomarker as well as a causative factor of the inflammatory pathophysiology in AD.
Pre-clinical studies with BMD-001 (ASO against pathological miRNA) have demonstrated significant reductions in neuroinflammation and beta-amyloid and tau pathology, as well as enhanced restoration of cognitive deficits in AD animal models.
"BMD-001 is a therapeutic candidate for AD, 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." said the company.
In September 2021, BIORCHESTRA appointed "Dr. Louis St. L. O'Dea", a pharma veteran, as its first CMO. He will play a key role in overall strategic direction, planning, execution and interpretation of clinical research for neurodegenerative diseases. He will attend the CTAD to introduce the pathological miRNA ASO therapeutic effects.
In June 2021, BIORCHESTRA was selected as the awardee in the NYC QuickFire Challenge in Neuroscience for its achievements by Johnson & Johnson Inovation.
Meanwhile, the company has a plan to start a clinical trial in the US next year.
