BIORCHESTRA Announces Significant Progress in Studies of BMD-001 Drug Program

BIORCHESTRA, a biotherapeutics company focused on rare and degenerative diseases within the Central Nervous System (CNS), today announced significant progress in its lead drug program, BMD-001.  Leveraging a proprietary, IV-formulated brain-targeting RNAi Nanomedicine (BTRiN^TM) platform that combines targeted cell delivery capabilities and proprietary RNA chemistries, BMD-001 has achieved significant milestones, including:

• Broad biodistribution across the brain, demonstrating disease target knockdown and related functional outcomes; and
• Downstream engagement of epigenetic disease hallmarks, including amyloid β plaques and neurofibrillary tangles.

BIORCHESTRA’s Scientific Founder, Chairman, and Chief Executive Officer, Dr. Branden Ryu, commented, “We are encouraged by these NHP findings, revealing that our IV formulation achieves consistent, sustained broad brain biodistribution and remarkable target knockdown across multiple measures.  Today’s results underpin the company’s accelerated development of BMD-001 within our neurodegenerative disease franchise focused on Alzheimer’s disease, Amyotrophic Lateral Sclerosis, and Parkinson’s disease.”

President & Chief Medical Officer, Dr. Louis St. L. O’Dea, commented, “Beyond achieving broad biodistribution in the brain, demonstrating significant target knockdown and related functional outcomes, our IV-formulated nanomedicine platform uniquely targets upstream epigenetic hallmarks associated with core downstream neurodegenerative disease pathologies. These include amyloid β plaques, neurofibrillary tangles, sustained activation of brain-resident macrophages and other immune cells that exacerbate amyloid and tau pathology.”

Dr. O’Dea added, “These results are notable given the challenges that have been encountered in providing meaningful therapeutic solutions for the rising number of patients, and their families, affected by neurodegenerative diseases.” Dr. O’Dea concluded, “Based on my experience in RNA therapeutics at Moderna and Akcea/Ionis, I see these results as very promising ahead of early meetings with international regulatory agencies to discuss and gain agreement on our development strategies.”