Recognition of outstanding in-vivo outcomes from a lead RNA program in CNS Underpinned by proprietary IV-formulated, Brain-targeting Ribonucleic Acid Interference (RNAi) Nanomedicine (BTRiNTM) platform BIORCHESTRA to receive in-kind products and services from Merck Group
CAMBRIDGE, Mass. and DAEJEON, South Korea, July 17, 2023 /PRNewswire/ — BIORCHESTRA, a biotherapeutics company focused on the treatment of rare and degenerative diseases within the Central Nervous System (CNS), today announced receipt of the grand prize award in the 2023 Merck Advance Biotech Grant Program.
Since 2014, Merck’s Advance Biotech Grant program has recognized innovative biotechnology companies worldwide with this award, by further supporting efforts to improve patient outcomes for diseases such as cancer, brain tumors, osteoarthritis, and cardiovascular disorders.
BIORCHESTRA’s Founder, Chairman, and Chief Executive Officer, Dr. Branden Ryu, commented, “We are deeply honored to receive Merck’s prestigious grand prize award for 2023 in recognition of the significant in-vivo outcomes achieved from our proprietary IV-formulated, Brain-targeting Ribonucleic Acid Interference (RNAi) Nanomedicine (BTRiNTM) platform. BRTiNTM combines industry-leading and proprietary, IV-formulated RNA chemistries with biological barrier cross and cell targetted delivery of RNA medicine focused on Alzheimer’s, Amyotrophic Lateral Sclerosis, and Parkinson’s disease.”
Key results from our lead ASO and siRNA programs (BMD-001) in 2023 include:
Evidence of successful intravenous cell-targeted delivery in non-human primates (NHP) and disease amelioration in an Alzheimer’s model; and 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.
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
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.
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.
Merck, a leading science and technology company, operates across life science, healthcare and electronics. More than 64,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2022, Merck generated sales of € 22.2 billion in 66 countries.
Scientific exploration and responsible entrepreneurship have been key to Merck’s technological and scientific advances. This is how Merck has thrived since its founding in 1668. The founding family remains the majority owner of the publicly listed company. Merck holds global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the business sectors of Merck operate as MilliporeSigma in life science, EMD Serono in healthcare, and EMD Electronics in electronics.
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