Arrowhead presentation at OTS Conference | ARWR Message Board Posts

Arrowhead Pharmaceuticals, Inc.

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Msg  4749 of 10176  at  10/5/2015 8:45:37 PM  by


Arrowhead presentation at OTS Conference

Have they previously disclosed APO(a) as a target?  SubQ? 

Development of RNAi-Based Therapeutics using DPC Technology

Bruce D. Given, MD

Arrowhead Research Corporation

We have developed a RNA interference (RNAi) trigger delivery platform called Dynamic Polyconjugates (DPC). Key features of the DPC platform include a reversibly masked endosomolytic polymer whose activity is revealed in response to environmental cues or the action of endosomal proteases. DPCs are also targetable, allowing ligand-dependent binding to the cell or tissue type of interest.

The modular design of DPCs allows them to be tailored to specific disease indications. A hepatocyte-targeted DPC is currently in clinical studies for the treatment of chronic hepatitis B and alpha-1 antitrypsin deficiency-related liver disease. This DPC contains an endosomolytic peptide reversibly masked with pH-labile moieties containing the N-acetyl galactosamine ligand specific for the hepatocyte-specific asialoglycoprotein receptor. Co- injection with this DPC and cholesterol-conjugated RNAi triggers allows efficient delivery of the RNAi triggers to the cytoplasm where they elicit high-level knockdown of target genes. A DPC designed for extra-hepatic tissues has also been developed. This DPC contains an endosomolytic polymer masked with moieties that are released upon exposure to proteases in the endosome. Attachment of a ligand that binds to αV-containing integrins on tumor cells allows uptake and target gene knockdown in mouse renal cell carcinoma models. A subcutaneous delivery system is also being developed that enables high-level and sustained knockdown of the liver-expressed protein apo(a). The apo(a) protein determines circulating Lp(a) concentration, which has a validated genetic association with residual cardiac risk following full LDL suppression. These examples illustrate the flexibility of Arrowhead’s proprietary delivery platforms and their utility in RNAi-based therapeutics.

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4750 Re: Arrowhead presentation at OTS Conference wsummar 7 10/5/2015 9:10:50 PM

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