• Presentation:"Successfully Achieving Intravenous (IV)-formulated RNA Delivery to the Brain, Evidence of Efficacy" scheduled for June 08, 2023, 12:00 p.m. ET, Room 104A
• Pioneering IV-formulated inert drug delivery platform targeting neurodegenerative diseases
• Lead program (BMD-001) advancing to IND
• IV-formulated ASO and siRNA encapsulation strategies with compelling in-vivo outcomes
• Drug delivery platform expanded to previously inaccessible neurological diseases, including brain cancers

CAMBRIDGE, Mass. and DAEJEON, South Korea, June 1, 2023 /PRNewswire/ -- BIORCHESTRA, a biotherapeutics company focused on the treatment of rare and degenerative diseases within the Central Nervous System (CNS), today announced that Dr. Branden Ryu, Founder, Chairman, and Chief Executive Officer, has accepted an invitation to present the company's discovery program, brain-specific gene targeted siRNA-BTRiN and non human primate (NHP) study outcomes from its lead program – BMD-001 – at the 2023 Biotechnology Innovation Organization International Convention. The conference, the largest in the industry, is scheduled for June 5 - 8, 2023, in Boston.   

In addition to the formal presentation by Dr. Ryu, company's senior executives will be meeting current global partners and responding to a growing list of leading pharmaceutical companies from the US, Europe, and Japan interested in the company's proprietary, IV-formulated drug delivery platform, following BIORCHESTRA's blockbuster deal news in March.

Leveraging an IV-formulated, proprietary brain-targeting Ribonucleic Acid Interference (RNAi) Nanomedicine (BTRiN^TM) platform that combines select tissue and target-cell delivery capabilities together with proprietary RNA chemistries, BIORCHESTRA has advanced BMD-001 and brain-specific gene targeted siRNA programs against significant milestones in its degenerative diseases franchise.

A sampling of the key results to be presented include:

• ASO BTRiN^TM (BMD-001): Evidence of successful intravenous delivery in NHP and disease amelioration in Alzheimer's acute NHP model; and
• siRNA BTRiN programs: Target gene knockdown efficiency in sub-brain regions following intravenous injection

These promising data are consistent with earlier NHP and rodent study findings that noted broad biodistribution in the brain, coupled with noteworthy neurodegenerative disease target knockdown across multiple measures, driven by a single IV formulation.

Ahead of engagements with the FDA regarding an Investigational New Drug (IND) pathway, the company expects to complete IND enabling work in 2024.

BIORCHESTRA's Founder, Chairman, and Chief Executive Officer, Dr. Branden Ryu, commented, "We are honored to speak at BIO23 to share our latest siRNA findings, which further demonstrates that our IV formulation achieves consistent, sustained broad brain biodistribution, in addition to significant disease target knockdown across multiple measures.  Today's results further strengthen BMD-001 within our neurodegenerative diseases franchise targeting Alzheimer's disease, Amyotrophic Lateral Sclerosis, and Parkinson's disease."

Company President & Chief Medical Officer Dr. Louis St.L. O'Dea commented, "We are making steady progress advancing our IV-formulated proprietary nanomedicine platform, that uniquely targets upstream epigenetic hallmarks associated with downstream neurodegenerative disease pathologies, including amyloid β plaques, neurofibrillary tangles, the sustained activation of brain-resident macrophages, and other immune cells that exacerbate both amyloid and tau pathology over time."  Dr. O'Dea summarized his satisfaction with these results, concluding, "I am extremelyconfident with these compelling pre-clinical results around the therapeutic potential of an RNAi medicine approach, targeting some of the most vexing brain diseases and disorders of our time, while current treatments offer minimal results."

About Brain-Targeting Ribonucleic Acid Interference (RNAi) Nanomedicine (BTRiN^TM)

BTRiN^TM targets an amino acid uptake receptor in the blood-brain barrier shared by neurons, astrocytes, and microglia (optional: thus ensuring both transfer across the BBB and entry into the target cells).  In addition to significant functional outcomes in a non-human primate study of Alzheimer's disease, BTRiN^TM has been leveraged with siRNA to validate the effective knockdown of a target implicated in Amyotrophic Lateral Sclerosis and Parkinson's disease.  Ongoing company discovery and development research will leverage BTRiN^TM for other underserved diseases, such as Glioblastoma multiforme, Lewy body dementia, Spinocerebellar ataxia type three, Frontotemporal dementia, Progressive supranuclear palsy. For more information, visit: www.biorchestra.com/science/our-scientific-approach

About BMD-001

Under development for efficient and effective IV administration targeting specific micro-RNA associated with neuroinflammation, mitochondrial dysfunction, neurodegeneration, and amyloid protein deposition in Alzheimer's disease, Amyotrophic Lateral Sclerosis, and Parkinson's disease.  BMD-001 has successfully demonstrated the suppression of specific microRNA across rodent and primate models, normalizing homeostatic processes, reducing amyloid and tau protein deposition, reducing neuroinflammation, and improving cognitive function. 

About BIORCHESTRA

BIORCHESTRA is a leading biotherapeutics company focused on the treatment of rare and degenerative diseases within the Central Nervous System by leveraging its proprietary Brain Targeting RNAi Nanomedicine (BTRiN^TM) platform.  These technologies combine targeted cell delivery capabilities and proprietary RNA chemistries to develop first and best-in-class therapeutics.  The company has extensive research and development and GMP manufacturing capabilities in Daejeon, South Korea, and a U.S. headquarters in Cambridge, Massachusetts, U.S.A.  For more information, visit: www.biorchestra.com and follow us on Twitter @biorchestra and LinkedIn.

About BIO International Convention 

BIO International Convention is the largest gathering worldwide for the biotech industry, bringing together influential leaders in the field to foster networking and partnering opportunities, and share insights on major trends shaping the industry. The event showcases keynote speeches and sessions from policymakers, scientists, CEOs, and celebrities, delivering inspiration and knowledge to attendees. It also features the BIO Business Forum, a One-on-One Partnering platform, where participants can connect and collaborate. Hundreds of sessions covering biotech trends, policy issues, and technological innovations are available to attendees, and the BIO Exhibition, the largest of its kind, exhibits the latest advancements and services within the biotechnology industry.  For more information about the convention, visit https://www.bio.org/events/bio-international-convention .

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