BIORCHESTRA 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.
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 (BTRiNTM) 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:
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 (BTRiNTM) BTRiNTM 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, BTRiNTM 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 BTRiNTM 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 (BTRiNTM) 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 . Safe Harbor Statement This press release contains forward-looking statements. In addition, from time to time, our representatives or we may make forward-looking statements orally or in writing. We base these forward-looking statements on our expectations and projections about future events, which we derive from the information currently available to us. Such forward-looking statements relate to future events or our future performance, including our financial performance and projections, revenue and earnings growth, and business prospects and opportunities. You can identify forward-looking statements by those that are not historical in nature, particularly those that use terminology such as “may,” “should,” “expects,” “anticipates,” “contemplates,” “estimates,” “believes,” “plans,” “projected,” “predicts,” “potential,” or “hopes” or the negative of these or similar terms. In evaluating these forward-looking statements, you should consider various factors, including our ability to change the company’s direction, keep pace with new technology and evolving market needs, and the competitive environment of our business. These and other factors may cause our results to differ materially from any forward-looking statement. Forward-looking statements are only predictions. The forward-looking events discussed in this press release and other statements made from time to time by our representatives or us may not occur, and actual events and results may differ materially and are subject to risks, uncertainties, and assumptions about us. We are not obligated to publicly update or revise any forward-looking statement, whether due to uncertainties and assumptions, the forward-looking events discussed in this press release, and other statements made from time to time by our representatives or us might not occur. Investor Contact: Skyline Corporate Communications Group, LLC
SOURCE BIORCHESTRA Co., Ltd. |
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