• June 3-6, 2024
  • San Diego Convention Center, California

Sessions

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Biorchestra

Monday, June 03, 2024
CP
CNS/Neurological
Biorchestra specializes in innovative IV-formulated and SC-formulated RNA therapeutics for neurodegenerative and other disorders. Our RNA medicines platform combines multiple modalities, chemistry innovations, and deep insights into human genetics to deliver scientific breakthroughs that treat rare and prevalent disorders. Our toolkit includes proprietary targeting ligands with an IV-formulated, non-lipid polymer and broad RNA cargo chemistry validation (ASO, siRNA, mRNA), providing premier capabilities for designing and delivering durable and potent drug candidates that optimally address disease biology through systemic administration. Our diverse pipeline includes late pre-clinical programs in Alzheimer's, ALS, and Parkinson's disease and earlier preclinical programs in Retina and Obesity.
Biorchestra
Company Website: http://biorchestra.com
Lead Product in Development: Biorchestra introduces a groundbreaking ASO therapy, BMD-001s, utilizing our innovative BDDS technology for IV delivery, offering unparalleled targeting within the CNS. This therapy aims at astrocytes, microglia, and neurons with exceptional precision, focusing on miR-485-3p. This microRNA is crucial for maintaining mitochondrial function and axonal health by regulating PGC1a and STMN2, whose imbalance plays a significant role in ALS pathology, leading to neurodegeneration and cognitive decline. BMD-001s represents a significant advancement, showcasing the potential to completely restore neuromuscular junction (NMJ) connectivity in ALS models, an essential step in combating the disease's rapid progression. In SOD1*G93A mice, a key model for ALS research, we observed a 100% restoration rate of fully functional NMJs at an optimal dosage, indicating a breakthrough in reversing neurodegeneration. Our research demonstrates remarkable improvements in neuromuscular function, as shown by better CMAP latencies and amplitudes, which are crucial measures of muscular health. Additionally, BMD-001s significantly lower NfL levels in the CSF, a promising indicator of neurodegeneration, underscoring its potential not just to stall but to reverse ALS progression. This development highlights our profound dedication to profoundly altering the course of patient outcomes.
Number Of Unlicensed Products (For Which You Are Seeking Partners): 8

Company HQ City

Cambridge

Company HQ State

Massachusetts

Company HQ Country

United States

CEO/Top Company Official

Dr. Branden Ryu

Development Phase of Primary Product

Pre-Clinical
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