- BIORCHESTRA announces at the RNA Leaders USA Conference in Boston that BMD-001 reduces amyloid-β and tau in a non-human primate animal model of Alzheimer's disease

BOSTON, Nov. 8, 2022 /PRNewswire/ -- Dr. Branden Ryu, CEO and Founder of BIORCHESTRA Ltd. (S. Korea), was a featured speaker at a plenary session of the RNA Leaders Congress USA in Boston, MA, on October 18, 2022. Dr. Ryu focused on new efficacy data for his lead compound, BMD-001, derived from studies in non-human primates that demonstrated clearance of the pathological proteins amyloid-β and tau.

RNA Leaders USA is focused on the scientific, clinical, and commercial development of RNA therapeutics and vaccines. The meeting brings together top executives to provide updates on the entire RNA landscape (RNAi, antisense technologies, small molecule targeting, mRNA, and more). In addition to Dr. Branden Ryu, other senior managers in attendance represented Ionis, Alnylam, Sanofi, Bayer, Eli Lilly, Astra Zeneca, Roche, and several emerging biotech companies. The meeting focused in particular on the challenge of delivering RNA-based therapies to specific organs, in particular the brain.

In his speech, Dr. Ryu presented data generated in collaboration with the National Primate Research Center of the Korea Bioscience and Biotechnology Research Institute. Using a primate Alzheimer's disease induction model, BIORCHESTRA was able to demonstrate the involvement of the brain target (microRNA-485-3p) by intravenous administration of BMD-001, and subsequent removal of amyloid-β and tau proteins . Thus, it was confirmed again, this time using a primate model of Alzheimer's disease, that BIORCHESTRA's drug delivery system (BDDS™) penetrates the blood-brain barrier and reaches the relevant cell types to enhance changes neurodegenerative. The data further demonstrated the wide distribution of BMD-001 in the cortex, medullary regions, and medulla.

In the conference presentation, Dr. Ryu stated, "The encouraging results of this experiment are that intravenous injection of our BDDS™ can deliver drugs systemically to the CNS: to the frontal lobe, thalamus, hypothalamus, the striatum and hippocampus, as well as in the medulla. He further added: "This experiment confirmed previously established biodistribution data in the non-human primate, but, in addition, the efficacy of BMD-001 established in smaller animal disease models is now confirmed with an efficacy readout in the non-human primate." This gives us confidence that our brain delivery platform is scalable to the human context," he concluded.

[about BIORCHESTRA]

BIORCHESTRA is a South Korea-based biotechnology company focused on treating neurodegenerative disorders such as Alzheimer's disease and amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. BIORCHESTRA was founded in 2016 by Dr. Branden Ryu from his doctoral and postdoctoral work in this field.

The most advanced drugs in BIORCHESTRA's pipeline focus on neurodegenerative diseases and target a key regulator of neuroinflammation and neurodegeneration, microRNA-485-3p. Other programs focus on CNS oncology and genetic brain diseases, such as Huntington's disease.

BIORCHESTRA remains privately funded and has recently opened an R&D center and GMP manufacturing facility in its hometown of Daejeon, South Korea. In addition to Daejeon, South Korea, BIORCHESTRA also has offices at One Kendall, Cambridge, MA, USA.

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