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Arrowhead Research White Paper - RNAi

I just read this whitepaper from a research firm in Southern California.  Looks like some promising stuff is in the pipeline for us chronically infected patients.  Any thoughts?


Arrowhead Research Corporation and Axolabs GmbH Enter into Strategic Alliance and Master Services Agreement for RNAi Therapeutics

PASADENA, Calif. — March 5, 2012 — Arrowhead Research Corporation, a nanomedicine company with development programs in RNAi therapeutics and obesity, and Axolabs GmbH, a custom research organization offering preclinical solutions and consultancy in the field of oligonucleotide therapeutics, today announced that they have entered into a Strategic Alliance and Master Services Agreement.
Under the terms of the agreement, Axolabs, founded by the principals of former Roche Center of Excellence for RNAi Therapeutics in Kulmbach, Germany, will provide Arrowhead and its partners first-in-class oligonucleotide optimization, synthesis, and analytics. These services include bioinformatics for siRNA design; lead identification, optimization and characterization; as well as siRNA synthesis and CMC-related activities.
"With multiple candidates in the clinic addressing a wide range of indications, data demonstrating the power of RNAi as a therapeutic modality are rapidly accumulating," said Dr. Christopher Anzalone, Arrowhead's CEO. "Axolabs' scientists are pioneers in the field. Having access to their expertise in preclinical development complements Arrowhead's capabilities well and provides us with yet another tool for preclinical and clinical collaborations in the field. Now, with the company's already established intellectual property position and array of proprietary delivery technologies including Dynamic PolyConjugates™ and RONDEL™, Arrowhead is uniquely positioned to build its own pipeline of RNAi therapeutics and provide partners entry to this promising area in a rapid and cost effective manner."
"This agreement continues the execution of Axolabs' strategy to become the preeminent custom research organization in the oligonucleotide therapeutics field," added Dr. Roland Kreutzer, Managing Director of Axolabs GmbH. "We know the colleagues at Arrowhead very well. We are happy to support them in establishing a leading role in the area of RNAi therapeutics and excited to be chosen as their preclinical service provider."

21 Responses
Avatar universal
Here's the proper press release:

Arrowhead Releases White Paper on Hepatitis B Virus and Potential RNAi Treatment

PASADENA, Calif. — March 7, 2012 — Arrowhead Research Corporation (NASDAQ: ARWR), a nanomedicine company with development programs in RNA therapeutics and obesity, announced today that it has released a white paper describing the health problem posed by the hepatitis B virus (HBV), the substantial unmet need for chronic HBV infected patients, and how Arrowhead’s Dynamic Polyconjugate (DPC) enabled RNAi therapeutic in development could potentially address deficiencies of current treatment options.
“Hepatitis B is a global health problem without effective treatment for a vast number of patients with chronic disease.  The World Health Organization estimates that 360 million people, or 5% of the world’s population, suffer from chronic hepatitis B,” said Christopher Anzalone, Ph.D., President and CEO of Arrowhead.  “Advances in hepatitis C treatment have drawn considerable attention over the last year, and we see HBV as a similarly high value target.  Extensive data from our development programs across multiple in vitro and animal models suggest that we can leverage RNAi and our DPCs to produce a powerful and highly specific candidate to fight HBV.”
HBV infection occurs primarily in hepatocytes and long-term infection causes hepatic inflammation that leads to acute and chronic hepatic dysfunction including acute hepatic failure, cirrhosis, and hepatocellular carcinoma.  The DPC delivery platform is being employed for the HBV product candidate, which is supported by multiple studies including demonstration in non-human primates of safe and effective delivery of siRNA to hepatocytes and promising data generated through mouse models of HBV using a DPC formulation.
“The development of a DPC-enabled HBV candidate began under Roche and we are very pleased with the progress that our scientists have made,” said David Lewis, Ph.D., Vice President of Biology and the site head at Arrowhead’s research and development facility in Madison, WI.  “We are excited to provide more detail on the DPC platform and the HBV program during upcoming scientific conferences and through future publication in peer-reviewed journals.”
Avatar universal
siRNi is a very promising technology with a very wide range of applications including cancer. However, it has many hurdles to overcome. especially delivery and safety issues. I would say we have to wait for a long time before we will see a drug - unless someone makes a break-through.

I think Gish wrote a paper about this (Gish RG, Satishchandran C, Young M, Pachuk C. RNA interference and its potential applications to chronic HBV treatment: results of a Phase I safety and tolerability study. Antivir Ther. 2011;16(4):547-54).)  Gish is also involved with Nuron Biotech.
"This unique technology provides Nuron Biotech with the capability to       develop a highly efficient vaccine for the treatment and prevention of       HBV and HCV by inducing both cellular (T cell) and humoral (B cell,       antibody) immune responses to clear disease," said Robert G. Gish, M.D.,       Chief of Clinical Hepatology and Professor of Clinical Medicine,       University of California, San Diego. Dr. Gish is also a member of the       Scientific Advisory Board for Nuron Biotech. "HBV and HCV are       life-altering and potentially fatal diseases, and this technology may       offer a break-through for a new generation of novel therapies and       preventative vaccines for patients around the world."  
Avatar universal
Maybe I can ask him about these exciting technologies on my next office visit with him.  He did mention that there were a couple of things that he had coming up in his "bag of tricks"
Avatar universal
Yes, please do. You can ask him:
1. The clinical trial testing stopping of Tenofovir
2. The more potent form of Tenofovir
3. Gilead's therapeutic vaccine

Avatar universal
When you mention "Gileads therapeutic vaccine" did you mean its TollLike7 receptor agonist drug, that is currently in development or do they indeed also have a therapeutic vaccine in development?
The TL agonist drug was tested in Woodchucks as presented in Orlando last year and showed very impressive Vl reductions of 3 to 4 logs.

I would like to mention that an other TL7 agonist is already approved for skin conditions. Its name is Imiquimod and the brand name. in most countries is "Aldara cream". It is also absorbed from the skin into the systemic circulation and might well have similar or the same effects on HBV supression by stimulation of the Tcell system by reactivating dormant or exhausted Tcells, the standard scenario in chronic HBV.
Avatar universal
i have read this RNAi, however understand nothing ..! .. Stephencastle .. you said it may take long before we will see the drug ..

is this technology can kill HBsAg or HBVDNA or boost the immune system or just recover the damage on liver caused by hbv .. thank for your effort in this forum to approach to scientific discription into simple words for normal reader ..
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