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Arrowhead - Press Releases and SEC Filings	Chart:	Clinical Trials:	Goals for 2016:	C. Anzalone on CNBC:	What is RNAi ?

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About Arrowhead Arrowhead Pharmaceuticals is a biopharmaceutical company developing targeted RNAi therapeutics.
Targeting Innovation
Arrowhead Pharmaceuticals develops novel drugs to treat intractable diseases by silencing the genes that cause them. Using the broadest portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. Arrowhead’s most advanced drug candidate in clinical development is ARC-520, which is designed to treat chronic hepatitis B infection by inhibiting the production of all HBV gene products. The goal is to reverse the immune suppression that prevents the body from controlling the virus and clearing the disease. Arrowhead’s second clinical candidate is ARC-AAT, a treatment for a rare liver disease associated with a genetic disorder that causes alpha-1 antitrypsin deficiency.

Lead Products
ARC-520 is an RNAi-based therapeutic designed to treat chronic hepatitis B virus (HBV) infection. It is the first clinical-stage drug candidate from Arrowhead’s Dynamic Polyconjugate® delivery platform. It is designed to treat chronic HBV infection by reducing the expression and release of new viral particles and key viral proteins with the goal of achieving a functional cure for HBV.

ARC-AAT is a novel unlocked nucleobase analog (UNA)-containing RNAi-based therapeutic for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease that can severely damage the liver and lungs of affected individuals. The goal of treatment with ARC-AAT is to reduce the production of the mutant Z-AAT protein to prevent and potentially reverse accumulation-related liver injury and fibrosis.

The companys pre-clinical stage drug candidates include ARC-521, an RNAi-based therapeutic for the treatment of chronic hepatitis B virus; ARC-F12, an RNAi-based therapeutic to treat hereditary angioedema and thromboembolic diseases; ARC-HIF2, an RNAi-based therapeutic to treat renal cell carcinoma; and ARC-LPA, an RNAi-based therapeutic for the treatment of cardiovascular diseases. It also holds patents related to Adipotide for the treatment of obesity and related metabolic disorders. The company has research collaboration and license agreement with Shire AG to develop and commercialize targeted peptide-drug conjugates.

Platform Delivery Technology
The Dynamic Polyconjugate (DPC®) platform is an RNAi delivery system that has been demonstrated to preferentially deliver to hepatocytes, induce efficient endosomal escape, promote high levels of gene knockdown in multiple animal models, and appears to be well tolerated using a variety of RNAi trigger molecules. It is a modular system that can be optimized on a target-by-target basis and may be targeted in the future to address multiple organ systems and cell types.

Pipeline Development Strategy
Arrowhead’s internal drug pipeline is intended to drive value directly through the clinical development of novel therapeutics and to provide proof of concept for our platform technologies. In addition to our two lead product candidates, ARC-520 and ARC-AAT, we intend to nominate additional clinical candidates that utilize the DPC delivery system. Our core areas of focus for expanding our internal pipeline of RNAi therapeutics are: (1) develop intravenous (IV) administered liver-targeted candidates; (2) develop subcutaneously administered liver-targeted candidates; and (3) explore extra-hepatic targets, including oncology.

To learn more about Arrowhead visit the company's website at: http://arrowheadpharma.com/

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Articles related to Arrowhead's sister technology: CRISPR

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Think2Succeed
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Sentiment: Strong buy !

Posts: 182
ARWR shares held: 20,000

Registered: Nov 24, 2015 2:03:46 GMT -5

Articles related to Arrowhead's sister technology: CRISPR Nov 30, 2015 2:46:44 GMT -5

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Post by Think2Succeed on Nov 30, 2015 2:46:44 GMT -5

Arrowhead's DPC is designed to silence genes (or to turned them on/off) however CRISPSR is the technology of changing, replacing and deleting genes in any living organism (in terms of medicine this should be riskier than gene silencing not to mention the controversial moral aspects of it). Anyway, does anyone know if ARWR's DPC technology is capable or "upgradable" to match CRISPR technology ? Of course there is also the patent issues...but from a science point of view is interesting to know.

Below you have a few interesting articles and a nice video explaining CRISPR and its potential:

Article 1 : How Controversial Gene Editing Could Lead To Groundbreaking Cures

Article 2: Future of human gene editing to be decided at landmark summit

Article 3 + excellent video: Unproven medical technique could save countless lives, billions of dollars

T2S

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Last Edit: Nov 30, 2015 2:47:24 GMT -5 by Think2Succeed

ARWR New Member

Posts: 22

Registered: Mar 7, 2016 16:47:58 GMT -5

Articles related to Arrowhead's sister technology: CRISPR Sept 6, 2016 17:30:13 GMT -5 Think2Succeed likes this

Quote

Post by buffettlike on Sept 6, 2016 17:30:13 GMT -5

I think Synthetic Biologist Feng Zhang of the Broad Institute is in deep $hit(EDIT):

www.ipwatchdog.com/2016/06/05/crispr-clash-dna-technology/id=69650/

*I was totally wrong on my assertion, humbly speaking. Updated 10-11-2018 @ 12:39pm

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Last Edit: Oct 11, 2018 11:39:48 GMT -5 by buffettlike

investorsd ARWR New Member Posts: 1 Registered: Aug 15, 2018 14:35:42 GMT -5	Articles related to Arrowhead's sister technology: CRISPR Aug 15, 2018 17:50:08 GMT -5 Quote Select Post Deselect Post Link to Post Member Give Gift Back to Top Post by investorsd on Aug 15, 2018 17:50:08 GMT -5 With all due respect, you're mixing apples with oranges.
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