29 Mar, 2019 Pre-Clinical Data on Delivery of RNAi Therapeutics to the CNS and Eye
We presented new pre-clinical data on our efforts in CNS and ocular delivery of novel siRNA conjugates in rats and non-human primates at the sixth annual Cold Spring Harbor Conference on RNA and Oligonucleotide Therapeutics, held March 27 to 30 in Cold Spring Harbor, NY.
In rat and non-human primate (NHP) pre-clinical studies, a single intrathecal injection of an siRNA conjugate led to widespread distribution and uptake in the CNS, resulting in robust and durable silencing of the target gene transcript throughout key anatomical areas of the brain and spinal cord.
In a separate rat pre-clinical study comparing siRNA conjugates to antisense oligonucleotides (ASOs), a single intrathecal injection of an siRNA conjugate targeting SOD1 showed superior silencing in all regions of the brain and spinal cord as compared to a single intrathecal injection of an ASO targeting SOD1, at the same dose.
With regard to ocular delivery, a single intravitreal injection of an siRNA conjugate targeting TTR in rats and NHPs led to potent and durable silencing in the eye.
RNAi therapeutics directed to disease-causing, CNS- or ocular-expressed genes represent a potential opportunity to address devastating diseases with enormous burden and unmet need. We are excited to pursue this opportunity.