Yacoub Habib
Chief Executive Officer Ophidion, Inc.
Seminars
Thursday 20th August 2026
Silencing Defective Genes in the Brain: Treating Neurodegenerative Diseases by Leveraging a Novel Brain-Centric Shuttle
12:00 pm
- Discussing a novel “Trojan horse” technology that enables the direct delivery of oligos to the brain via intravenous or subcutaneous administration
- Therapeutic efficacy in Huntington’s disease demonstrated at a dose significantly lower than the standard intrathecal ASO dose (less than 1,000 the dose used intrathecally) Over 95% halting of disease progression in chronic preclinical studies
- In a collaboration with a large pharmaceutical company, a single IV dose of an ASO complexed to our Trojan horse was able to achieve equivalent protein reduction as an ICV dose. Confirmed safety and consistent brain delivery in repeat-dose non-human primate studies
- This non-invasive delivery approach offers a significant clinical advantage over invasive modalities or other BBB shuttles and could be a powerful enabler for any siRNA/ASO or peptide/protein programs targeting CNS indications
