Branden Ryu
Chief Executive Officer Biorchestra
Seminars
Wednesday 9th December 2026
BDDS™ (CNS Platform) & the Therapeutic Effect of BMD-002 for Genetic Epilepsy
2:15 pm
- BDDS™ is a polymer-based nanoparticle that displays multivalent L-Phe ligands on its surface. These ligands engage the LAT1–CD98hc heterodimer to promote endocytic uptake. In vitro and in vivo evidence strongly suggests LAT1-mediated ASO delivery to the CNS
- BMD-002 is an anti-miR ASO, or antagomir, formulated with BDDS™ that restores GABRA1 protein in the hippocampus of animal models of epilepsy following intravenous administration
- GABRA1 encodes the α1 subunit of the inhibitory GABA-A receptor. Loss-of-function variants result in haploinsufficiency characterized by developmental delay, drug-resistant seizures, hypotonia, and limited communication. Reduced α1 protein is also a hallmark of acquired drug-resistant epilepsy
- Therapeutic effects were observed for up to 12 weeks following a single intravenous dose of BMD-002 in two well-characterized chronic epilepsy models
