CAMBRIDGE, Mass. and DAEJEON, South Korea, March 8, 2023 /PRNewswire/ — BIORCHESTRA, a biotherapeutics company focused on the treatment of rare and degenerative diseases within the Central Nervous System (CNS) by leveraging its proprietary Brain-Targeting RNAi Nanomedicine (BTRiNTM) technology to combine targeted cell delivery capabilities and proprietary RNA interference (RNAi) chemistries, to develop best-in-category RNAi therapeutics, today announced that it will be participating in two upcoming therapeutic RNA-focused events:
1. TIDES Asia Kyoto, Japan (March 7-9)
Presentation – March 08, 1:00 p.m. (GMT+9)
2. RNA Leaders Europe Basel, Switzerland (March 15-16)
Presentation – March 15, 12:00 p.m. (GMT+1)
BIORCHESTRA Founder, Chairman, and Chief Executive Officer Dr. Branden Ryu has accepted an invitation to speak at the two conferences to discuss the successful development of BIORCHESTRA’s proprietary brain-targeting RNAi nanomedicines (BTRiNTM) for Central Nervous System-related diseases. Dr. Ryu will share insights on company-sponsored successful in-vivo proof of concept data which has been achieved across multiple animal models from its lead program, BMD-001, and give an update on the company’s expanding neurological diseases franchise targeting Alzheimer’s disease (AD), Amyotrophic Lateral Sclerosis (ALS), and Parkinson’s disease (PD), and other pipeline programs.
While RNAi and RNA therapeutics have recently made significant advances and entered clinical practice, the ability to target and reach specific organs and diseases is limited. Although progress has been made in addressing a limited number of liver-modulated diseases, the major organ systems of the brain, kidney, and muscle remain largely unaddressed by this drug class. BIORCHESTRA has developed a nanomedicine-based approach to deliver its proprietary RNAi chemistries to the brain, and wider CNS regions, such as the spinal cord, to address the growing global issues of dementia and neurodegeneration. Using the combined expertise in targeted brain delivery and RNAi chemistry which are merged in its BTRiNTM platform, BIORCHESTRA has demonstrated successful proof of concept in models up to primates and demonstrated functional improvement in animal models of Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and Parkinson’s disease (PD).
RNAi (RNA interference) is a cellular process of gene silencing occurring in nature and emerging as the frontier in drug development. A new class of RNA medicines is now possible by leveraging this natural biological process of RNAi occurring in the cellular microenvironment. BIORCHESTRA’s proprietary therapeutic platform is uniquely designed to cross the blood-brain barrier and deliver therapeutic RNA cargo to act on intracellular targets – genetic precursors – that encode for disease-causing or disease pathway proteins common across AD, ALS, and PD.
Lead drug program developed to target a specific micro-RNA overexpressed in neurological diseases. An intravenous (IV) formulation of BTRiNTM encapsulated RNAi has successfully demonstrated the suppression of specific microRNA across rodent and primate models, normalizing homeostatic processes, reducing amyloid and tau protein deposition, and reducing amyloid and tau protein deposition and reducing neuroinflammation, and improvements in cognitive function.
About Brain-Targeting RNAi Nanomedicine (BTRiNTM) Platform
Formulated for IV administration, BTRiNTM uniquely targets an amino acid receptor in the blood-brain barrier shared by neurons, astrocytes, and microglia. In addition to significant functional outcomes in a non-human primate study of AD, BTRiNTM has been leveraged to validate the knockdown of similar microRNA in ALS and PD models. Ongoing company discovery and development research promise to extend BMD-001 to other underserved Central Nervous System diseases, including Glioblastoma multiforme, Lewy body dementia (LD), Spinocerebellar ataxia type three, Frontotemporal dementia, Progressive supranuclear palsy.
BIORCHESTRA is a biotherapeutics company focused on the treatment of rare and degenerative diseases within the Central Nervous System (CNS) by leveraging its proprietary Brain-Targeting RNAi Nanomedicine (BTRiNTM) technology to combine targeted cell delivery capabilities and proprietary RNA interference (RNAi) chemistries, to develop best-in-category RNAi therapeutics. The company’s lead Alheimer’s disease program (BMD-001) is under development for efficient and effective IV administration to treat targets associated with neuroinflammation, mitochondrial dysfunction, neurodegeneration, and amyloid protein deposition. Consistent across multiple animal models, BMD-001 has demonstrated concomitant disruption of the epigenetic pathways related to neurological diseases, including Alzheimer’s disease (AD), Amyotrophic Lateral Sclerosis (ALS), Lewy body dementia (LD), and Parkinson’s disease (PD). The company has extensive research and development, GMP manufacturing capabilities in Daejeon, South Korea, and a U.S. operational headquarters in Cambridge, Massachusetts, U.S.A. For more information, visit: www.biorchestra.com and follow us on Twitter @biorchestra and LinkedIn.
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