BIORCHESTRA

BIORCHESTRA is a biotherapeutics company focused on developing ribonucleic acid (RNA)-based therapeutics for the treatment of neurodegenerative diseases such as Alzheimer's disease, Amyotrophic Lateral Sclerosis (ALS), and Parkinson's disease. The company's lead program, BMD-001, is an RNA-based therapeutic targeting specific microRNAs associated with neuroinflammation, mitochondrial dysfunction, neurodegeneration, and amyloid protein deposition in these diseases.

BIORCHESTRA has developed a proprietary platform called Brain-targeting Ribonucleic Acid Interference (RNAi) Nanomedicine (BTRiNTM) for intravenous (IV) administration of RNA therapeutics. This platform combines targeted cell delivery capabilities and proprietary RNA chemistries to enable efficient delivery of RNA drugs across the blood-brain barrier and into target cells within the brain.

The company has successfully demonstrated broad biodistribution and significant disease target knockdown in the brain across multiple animal models, including non-human primates, using a single IV formulation. BMD-001 has shown promising results in suppressing specific microRNAs, normalizing homeostatic processes, reducing amyloid and tau protein deposition, reducing neuroinflammation, and improving cognitive function in these models.

In July 2023, BIORCHESTRA received the grand prize award in the Merck Advance biotech Grant Program, recognizing the outstanding in-vivo outcomes achieved with its BTRiNTM platform and BMD-001 program.