Invited Presentation: Single-Walled Carbon Nanotubes for Delivery of siRNA: Producing a Pharmaceutically Stable and Effective Drug Product

siRNA has the potential to be a selective targeted cancer therapy but currently lacks an effective delivery mechanism. Ensysce has been exploring the use of single-walled carbon nanotubes (SWCNT) as delivery vehicles for siRNA and has extensively examined the safety, and in vitro and in vivo efficacy of this platform. We have determined that siRNA/SWCNT complexes are well tolerated, and can be administered on a multi-weekly intravenous (i.v.) dosing schedule over extended periods of time.  Our work has explored formulation and processing to provide a drug product with a good pharmacokinetic half-life allowing distribution of the SWCNT to desired tissues in animals and that provides suitable pharmaceutic properties for use in a clinical setting.  Complexes with various siRNA payloads have been evaluated in animal models with human tumor xenografts.  Antitumor activity has been demonstrated with siRNA/SWCNT complexes targeting one or two growth driving proteins. Preclinical development has begun and a product has been selected to move through IND enabling toxicology.  SWCNT offer a novel approach to safely and effectively deliver oligonucleotides, and by manipulating formulation of the complex we can alter the siRNA biodistribution to tissues of interest with resulting target inhibition for disease control.