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A researcher into gene silencing as a technology for improving the lives of those suffering from incurable diseases. Benitec Biopharma is my primary investment in this technology as ddRNAi offers one-time treatments for a broad range of these diseases.
  • DdRNAi Is In The Eye Of The Beholder 12 comments
    Mar 20, 2014 2:19 PM | about stocks: BNIKF


    Benitec's DNA Directed RNA Interference (ddRNAi) literally is or has been in the eyes of many beholders; albeit that these beholders are limited to mice and large animals at this point in time. This research, where ddRNAi constructs have been injected into the eyes of lab animals, has been conducted in order to advance treatments for a range of eye disease. The application of ddRNAi to human beholders will come when Genable Technologies (a ddRNAi licensee) applies this technology to the treatment of Retinitis Pigmentosa (NASDAQ:RP). The company was forecasting the start of human trials by the end of 2013 but this date has slipped. The application of ddRNAi to Age-Related Macular Degeneration (NYSE:AMD) will be fast-tracked by Benitec itself once a second tranche of capital raising has been approved (subject to shareholder acceptance) in April of this year.


    When RNA Interference (RNAi) was considered as a viable means of treating human diseases, many in the field thought that its application to ocular diseases would be the first to show success. This was in part due to the conserved nature of the eyes and ocular diseases. Trials using synthetic siRNA (a competitive technology to ddRNAi) were commenced in humans but ultimately proved to be unsuccessful. To some extent these trials exposed some of the then limitations of siRNA technology. One of these limitations was the that the treatments only had a short-term effect and this is a limitation that can be removed with the use of ddRNAi as ddRNAi can produce a continual supply of therapeutic short hairpin RNA (shRNA) molecules.

    For this reason, the application of shRNA to treat ocular diseases has been advanced by a number of research teams for a range of afflictions such as RP, AMD, and Retinal Neovascularisation.


    Genable Technologies is using gene therapy to treat RP. RP is a group of inherited eye disease for which there is no cure. It is estimated to affect 1 in every 3,500 to 4,000 people in the US and Europe.

    Genable's first drug candidate, GT038, is targeting rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP). GT038 has been designed to use a suppression and replacement technique where the faulty gene is silenced using ddRNAi and a normal copy of the gene is supplied via an Adeno-Associated Virus (NYSE:AAV). It has been engineered so that the normal gene avoids the suppression provided be the ddRNAi construct.

    This form of the disease affects approximately 30,000 patients in the US and Europe. The company has received Orphan Disease status from both the European and US regulatory authorities. It estimates the market to be worth about $500M pa.

    The company is yet to publish a new date for the start of its clinical trial but I would speculate that it will be later this year.


    AMD is the leading cause of blindness and sight loss in Americans over the age of 65. With an aging population this affliction is set to increase. About 1.75 million US residents have the disease and this is forecast to grow to 3 million by 2020.

    The disease has two forms, Wet and Dry, depending on whether the degeneration is caused by invasive blood vessels or not. Dry is the more common form and wet the more serious.

    There is no cure but monthly injections can delay its progress.

    When Benitec acquired Tacere it not only acquired a treatment for Hepatitis C but also the design for a treatment for Wet AMD. While this program is only at the start of its pre-clinical development, it is set to build on the previous work done with the siRNA trials as the first version of the treatment, TT-211, targets the expression of vascular endothelial growth factor A (VEGF-A ) which is the same target as those previous studies.

    TT-211 is being designed as a single injection that will provide years of protection against the disease and this approach is supported by independent research.

    The company estimates this market to be worth $2B in the US alone.

    TT-211 also represents the next generation of ddRNAi technology. The shRNA is modelled into a microRNA (miRNA) backbone (ShMir) and ShMir will be the next development in ddRNAi. TT-211 will have cell specific promoters and its design is being optimised to deliver ShMir to all the layers of the retina from one intravitreal injection.

    The follow on drug, TT-231, is already being designed to treat both Dry and Wet AMD. This ShMir will incorporate three shRNA's targeting as yet unnamed proteins.

    Approval of the company's second tranche of capital raising is essential if this next generation of technology is to advance.


    Benitec's ddRNAi technology is being used by many research teams around the world to find cures for ocular diseases including RP and AMD. The first in human trial is likely to be when Genable treats its first patient, probably later this year. Benitec's own program is at an early stage but it has the promise of a treatment for the yet incurable AMD.

    The estimated market for RP and AMD combined is about $2.5B in the US alone.

    Disclosure: I am long BNIKF.

    Additional disclosure: The views expressed here do not represent investment advice.

    Stocks: BNIKF
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Comments (12)
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  • pga2003
    , contributor
    Comments (70) | Send Message
    Great article again! Keep it up!
    20 Mar 2014, 05:44 PM Reply Like
  • MrPeeps
    , contributor
    Comments (12) | Send Message
    Voting yes for the next tranche. Long BNIKF!
    20 Mar 2014, 06:50 PM Reply Like
  • lemmema2
    , contributor
    Comments (24) | Send Message
    Nice article! What is your estimate for the SP for Benitec by the end of 2014? Just want to hear your thoughts,
    20 Mar 2014, 07:15 PM Reply Like
  • Pannobhaso
    , contributor
    Comments (245) | Send Message
    Author’s reply » Martin, the sp is undoubtedly bound by two things over the next 12 months. The first is the results of the TT-034 trial and the second is whether or not the company can secure a partnering deal with a big pharma.


    Management has said that they would like to take TT-034 to a Pllb trial before opening up negotiations with a big pharma. This would rule out a deal this year. If the next tranche of capital is approved, no deal this year would not be a problem.


    As far as the TT-034 results are concerned. I am anticipating good results which will cause a spike in the sp as investors see increased value and less risk in the company. A further spike will occur when the NSCLC program trial starts.


    What the actual price may be is anybody's guess; I really have no better way of knowing. Like everyone else on this thread, my educated guess says that the sp will be much higher at year's end than it is today.


    20 Mar 2014, 08:47 PM Reply Like
  • Pannobhaso
    , contributor
    Comments (245) | Send Message
    Author’s reply » I should have also mentioned that I think Benitec listing on the NASDAQ and not trading on the OTC markets could improve liquidity in the stock and this would affect the share price. If the company is serious about the US market, then I think this move is inevitable.
    20 Mar 2014, 10:10 PM Reply Like
  • subrkaus3
    , contributor
    Comments (98) | Send Message
    This is definitely necessary for Benitec to gain traction in the US market. OTC listed stocks are mostly not bought by many investors!
    20 Mar 2014, 11:04 PM Reply Like
  • GrowthGeek
    , contributor
    Comments (3203) | Send Message
    Agreed, look for the company to work towards making this happen once the stock price maintains above the $2.50 mark.
    24 Mar 2014, 01:48 PM Reply Like
  • Kevin Murphy
    , contributor
    Comments (111) | Send Message
    Here is another company Inovio, (INO), that seems to be involved in using ddRNA in some form for vaccines. It has good backing, enough cash through 2017, and its price has gone from about 70 cents to over $3.50 within the last year.


    Here is a link to their website, and an extract of their overview from their investor page. I would appreciate your comments on their viability in this sphere. I am also an investor in BNIKF.





    Inovio is revolutionizing vaccines. We are advancing a broad portfolio of DNA vaccines, also known as immunotherapies, to prevent or treat cancers, HIV, hepatitis C virus, and other chronic infectious diseases. Our immunotherapies could potentially protect millions of people from sickness or death from diseases for which there are currently no adequate treatments.


    Investment highlights


    - Potentially game-changing phase II efficacy data from lead program mid-2014


    - Exclusive worldwide partnership with Roche to develop and commercialize products from Inovio’s prostate cancer (INO-5150) and hepatitis B (INO-1800) immunotherapy programs


    - Advancing discussions with large pharmaceutical companies regarding partnerships


    - Almost $60M in non-dilutive third party R&D grants and expenditures since 2009


    - Operating capital through 4Q 2017


    - Synthetic DNA vaccines with proprietary delivery technology targeting diseases with multi-billion-dollar markets; designed to extend the powerful capabilities but overcome limitations of conventional vaccines


    - Best-in-class T-cell immune responses displaying killing effect against target cells – designed to fight cancers, HIV, hepatitis and other diseases requiring treatment


    - Universal antibody immune responses to protect against constantly changing viruses such as influenza


    - Favorable safety profile with no serious adverse events in hundreds of subjects to date


    - Vaccine market expected to reach $52 billion by 2016, up from $13 billion in 2007


    - Dominant global patent position


    - Management team and advisors are leaders in the world of vaccines and DNA vaccines


    - Recently recognized with Vaccine Industry Excellence Awards for Best Therapeutic Vaccine (for HPV-associated diseases) and Best Early Stage Biotech Company at World Vaccine Congress.


    Learn More


    [stuff deleted on electronic shareholder features]


    Read our corporate profile.


    Read our investor presentation.


    View Inovio Stock Quote & Chart


    end quote
    20 Mar 2014, 08:32 PM Reply Like
  • subrkaus3
    , contributor
    Comments (98) | Send Message
    Good stuff as usual Panno! It cannot get any better for Benitec!
    20 Mar 2014, 09:15 PM Reply Like
  • Seanl482819
    , contributor
    Comments (8) | Send Message
    Current SP is SOOOO cheap atm!
    The moment a disease is cured from this technology, dont expect to get in for anything less than $10
    20 Mar 2014, 10:09 PM Reply Like
  • SmokeyNYY
    , contributor
    Comments (95) | Send Message
    Fantastic article. Surprised this wasn't published as a main article.
    21 Mar 2014, 12:25 AM Reply Like
  • Pannobhaso
    , contributor
    Comments (245) | Send Message
    Author’s reply »


    More news on Genable
    25 Mar 2014, 12:29 AM Reply Like
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