BIORCHESTRA

About BIORCHESTRA

BIORCHESTRA is a biotherapeutics company established in 2016 with headquarters in Daejeon, South Korea, and Cambridge, Massachusetts. The company was founded by Dr. Branden Ryu, who also serves as its Chairman and CEO. Dr. Ryu's background includes a Ph.D. from the University of Tokyo's Graduate School of Medicine and research experience at Harvard Medical School, providing a strong scientific foundation for the company's focus on RNA-based therapeutics.

The company concentrates on developing treatments for neurodegenerative and rare diseases affecting the Central Nervous System (CNS), such as Alzheimer's, Parkinson's, and Amyotrophic Lateral Sclerosis (ALS). BIORCHESTRA's core technology is its Brain Targeting RNAi Nanomedicine (BTRiN™) platform, formerly known as the Biorchestra Drug Delivery System (BDDS™). This platform is designed to overcome the significant challenge of the blood-brain barrier, enabling the intravenous delivery of various RNA-based drugs (including ASO, siRNA, and mRNA) directly to the brain. The technology works by encapsulating therapeutic agents in a proprietary non-lipid polymer that targets an amino acid receptor found on neurons, astrocytes, and microglia.

The lead drug candidate, BMD-001, is an antisense oligonucleotide (ASO) therapy that targets a specific microRNA (miRNA-485-3p). This microRNA is implicated as a key regulator of neuroinflammation and the pathological accumulation of amyloid-beta and tau proteins, which are hallmarks of Alzheimer's disease. Preclinical studies in rodent and non-human primate models have shown that BMD-001 can suppress the target microRNA, reduce these pathological proteins, decrease neuroinflammation, and improve cognitive function.

BIORCHESTRA's business model is centered on internal drug development, strategic partnerships, and licensing its delivery platform. The company has secured significant financing to advance its pipeline, including a $45 million Series C round in February 2022, bringing its total funding raised to nearly $60 million. This capital is being used to fund R&D, complete a GMP manufacturing facility, and advance BMD-001 towards an Investigational New Drug (IND) filing. The company has entered into major collaborations, including a research agreement with SK Biopharmaceuticals to develop epilepsy treatments and a licensing deal worth up to $861 million with an unnamed U.S. biopharmaceutical company to develop nucleic acid therapies for neurological disorders.