Sangamo’s Gain, Gilead’s Loss? HIV Treatment Outlook Buoyed by Gene Therapy

3/10/14Follow @Tansey_Xconomy

As the news emerged of Richmond, CA-based Sangamo Biosciences’s progress toward a gene therapy treatment for HIV infection, the media buzzed and investors boosted the company’s share price by 17 percent Thursday.

For the first time, Sangamo (NASDAQ: SGMO) had tried its gene-editing platform on human subjects, and it found encouraging signs in small early stage trials that the treatment could be adapted to push down the population of HIV virus to low or even undetectable levels.

The grabber in the story is that such a treatment might someday allow HIV-infected patients to stop taking antiviral drugs daily for the rest of their lives—eliminating both side effects and a big expense for the healthcare system.

So it’s natural to wonder how such developments would affect the companies taking in billions of dollars a year from sales of those HIV drugs.

The improved prospects for an HIV “functional cure” might have led some investors to turn around and shed shares in Foster City, CA-based Gilead, the world’s largest maker of HIV drugs, whose share price dropped more than 3 percent to $79.92 on Thursday and lost a bit more ground to wrap up the week at $79.58 on Friday. Antiviral drugs made up $9.34 billion of Gilead’s $10.8 billion in product sales in 2013. Gilead’s slide coincided with an overall decline in biotech indexes. It also came amid news from a Boston HIV/AIDS conference, including data from other HIV drug developers as well as Sangamo, that may have affected trading.

Sangamo CEO Edward Lanphier

Sangamo CEO Edward Lanphier

But some investors who have been closely following Gilead might have seen the price dip as an opportunity to snap up more shares in the company, which is also one of the industry’s most broad-based and well-funded new developers of cancer therapies.

While Gilead has continued to nurture its HIV and antiviral franchise—in December it gained FDA approval of the new chronic hepatitis drug sofosbuvir (Sovaldi), and won expanded FDA approval of its once-daily HIV combination drug, Complera—the company has also been buying up oncology companies and assets since at least 2010. And the acquisitions are beginning to bear fruit. Gilead announced in October that a late-stage trial of one of its leading cancer compounds, idelalisib, was halted early because the drug was already showing significant efficacy as a treatment for chronic lymphocytic leukemia. Even with last week’s price dip, Gilead shares are still up more than 75 percent over the past 12 months.

Gilead’s forward-looking moves to become a diversified pharmaceutical company may insulate it from an erosion of HIV drug revenue—if and when an effective gene therapy becomes approved, and accessible. Sangamo’s promising results came from tiny, early stage trials, and the company has said it needs to refine its ZFN gene-editing treatment procedures in future studies to enhance the chance of a cure.

But Sangamo’s moment in the spotlight is one recent reminder that gene therapy is finally emerging as a viable technology that could transform the treatment of HIV, cancer, and a host of other conditions. If the method proves out, it could shoulder aside some maintenance drug therapies by attacking genetic mechanisms closer to the root causes of a range of diseases. Sangamo is trying to do this with its proprietary toolkit of zinc finger proteins for altering the sequences of targeted genes. ZFN stands for zinc finger nucleases, which are molecular tools for homing in on specific small sections of genes and clipping them out.

“Our goal is to use this powerful technology to engineer genetic cures for diseases that have thus far been treated as chronic conditions, including HIV and a wide range of monogenic diseases,” said Sangamo CEO Edward Lanphier in a company release on the first of several studies unveiled this week.

The first paper, published in the New England Journal of Medicine on Wednesday, detailed the results of Sangamo’s inaugural trial of its ZFN gene-editing platform in HIV patients. Through genetic engineering of blood cells called T-cells, Sangamo is trying to mimic the natural immunity to a common HIV strain that a few rare individuals are fortunate enough to inherit with their own genes.

To create its treatment, Sangamo disabled a gene called CCR5 in T-cells from blood samples withdrawn from 12 HIV-positive trial participants. In most people, the CCR5 gene makes the cells susceptible to HIV infection. The modified T-cells with disrupted CCR5 genes were then infused back into the trial subjects.

In such Phase I trials, the first goal is to establish that previously untested treatments are safe. Sangamo reported that its treatment, called SB-728-T, was … Next Page »

Bernadette Tansey is Xconomy's San Francisco Editor. You can reach her at btansey@xconomy.com. Follow @Tansey_Xconomy

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  • jack

    In my opinion scientist can make different groups and different gene editing regimen ( 10-15 grop) from there which is going curing track that can wecan select is not it bettbetter? ? I suprise why only one kind of regregimen follow at time..? We been waiting long time for cure..

  • MineMan

    What was meant by Ed Lanphier when he mentioned “universal donor cells?

    • Sophia Blaze

      its a pleasure for me to write this testimony about this wonderful thing
      that happened to me last week on how i got my HIV aids cured, i have
      been reading so-many post of some people who were cured of HIV, but i
      never believed them, I was hurt and depressed so I was too curious and
      wanted to try dr.Ariba then i contacted him on his email on
      dr.aribaspelltemple@outlook.com, when i contact him, he assured me 100%
      that he will heal me, i pleaded with him to help me out, its a great
      success that he healed just as he promised, he told me that in three
      days time that i should go and check on my HIV status, I was floored
      that when i went to the hospital to check of my status that i was HIV
      negative, i never thought possible that dr.Ariba can do miracles, i
      never really believed in magic but I played along with a little hope and
      and faith and after everything but dr ARIBA changed my life and made me
      a true believer you can contact him on dr.aribaspelltemple@outlook.com
      or call +2348163979933 so he can help you out

  • Sophia Blaze

    i was diagnosed with HIV/AIDS for over 5 years which made loose my job and my relationship with my fiance after he discovered that i was having HIV, he departed from me i tried all my best to make him stays with me, but neglected me until a friend of mine on Facebook from Singapore told me Great healer, who will restore my life back with his powerful healing spell, he sent me the email address to contact (dr.aribaspelltemple@outlook.com) and i quickly contacted him, and he said my condition can solved, that he will treat the disease immediately only if i can accept trust him and accept his terms and condition, i Agreed because i was so much in need of help by all means, so i did all he want from me and surprisingly on Sunday last week He sent me a text, that i should hurry up to the hospital for a checkup, which i truly did, i confirm from my doctor that i am now ( HIV NEGATIVE) my eyes filled with tears and joy, crying heavily because truly the disease deprived me of many things from my life, This is a Miracle, Please do not en-devour to email dr.aribaspelltemple@outlook.com or call 2348163979933 for healing and solution

  • Sophia Blaze

    its a pleasure for me to write this testimony about this wonderful thing that happened to me last week on how i got my HIV aids cured, i have been reading so-many post of some people who were cured of HIV, but i never believed them, I was hurt and depressed so I was too curious and wanted to try dr.Ariba then i contacted him on his email on dr.aribaspelltemple@outlook.com, when i contact him, he assured me 100% that he will heal me, i pleaded with him to help me out, its a great success that he healed just as he promised, he told me that in three days time that i should go and check on my HIV status, I was floored that when i went to the hospital to check of my status that i was HIV negative, i never thought possible that dr.Ariba can do miracles, i never really believed in magic but I played along with a little hope and and faith and after everything but dr ARIBA changed my life and made me a true believer you can contact him on dr.aribaspelltemple@outlook.com or call +2348163979933 so he can help you out

    • Unknown

      Do you know where Dr. Ariba is located at?

  • Isaac John

    I do not believe it until I receive the healing too, I’m from France I want to share a testimony on how Dr. abiola cured my HIV disease, I was having the HIV disease, base on the testimonies I saw on the net I decided to contact him and told him my problems, he required some items in which he will use in preparing the cure for me, I sent him the items money, he bought the items and prepared the cure and he sent it to me via Edo courier deliver service, I drank it and he told me that after drinking it that I will wait for 45 minutes before I can go a conduct a test on myself, faithfully I went for the test and I was tested NEGATIVE, he alone can cure HIV disease, contact him now via deadly.diseases.cure@hotmail.com

  • RODA

    Hi, I work in a communication company I want to share my testimony to the world, my name is roda, i am from united kingdom in belfast north ireland, I was a stripper in a club I got effected with HIV due to the nature of my job, In april 15 2013 i was tested positive to HIV, This is not design to convince you but its just a personal health experience . I never taught doctor lawcy could ever get my HIV-AIDS cured with his healing herb spell, i have tried almost everything but I couldn’t find any solution on my disease, despite all these happening to me, i always spend a lot to buy a HIV drugs from hospital and taking some several medications but no relieve, until one day i was just browsing on the internet when i came across a great post of !Michelle! who truly said that she was been diagnose with HIV and was healed that very week through the help of this great powerful healing spell doctor ,I wonder why he is called the great papa lawcy, i never knew it was all because of the great and perfect work that he has been doing that is causing all this. so I quickly contacted him, and he ask me some few questions and so i did all the things he asked me to do,He ask me to buy some herbs and which I did for my cure,only to see that at the very day which he said i will be healed, all the strength that has left me before rush back and i becomes very strong and healthy, this disease almost kills my life all because of me, so i went to hospital to give the final test to the disease and the doctor said i am HIV negative, i am very amazed and happy about the healing doctor lawcy gave to me from the ancient part of africa, you can email him now for your own healing too on his email: drlawcyspellhome@gmail.com,,,,,,,,,,,,,,,

  • Theodora Fabzz

    Dr. Oraede God will continue to bless you more abundantly, for the good works you are doing in peoples life, I will keep on writng and posting testimonies about you on the Internet, I’m Theodora Fabzz, I was a HIV patient, I saw a blog on how Dr. Oraede cured someone, I contacted him and also got my healing, kindly email him now on Dr.oraedespellhome@hotmail.com or dr.oraedespellhome@gmail.com or email me on theofabzz@gmail.com or call him on +2348161879468.

  • Isaac John

    I do not believe it until I receive the healing too, I’m from France I want to share a testimony on how Dr. abiola cured my HIV disease, I was having the HIV disease, base on the testimonies I saw on the net I decided to contact him and told him my problems, he required some items in which he will use in preparing the cure for me, I sent him the items money, he bought the items and prepared the cure and he sent it to me via Edo courier deliver service, I drank it and he told me that after drinking it that I will wait for 45 minutes before I can go a conduct a test on myself, faithfully I went for the test and I was tested NEGATIVE, he alone can cure HIV disease, contact him now via deadly.diseases.cure@hotmail.com ,,,.

  • Beatrice Mark

    Am Beatrice mark.I was HIV/AIDS positive for 3 years until i saw a testimony of how the lord spiritual has been
    healing people suffering from HIV and other diseases, i contacted him and today am free.i want to express a heart felt

    appreciation to the LORD SPIRITUAL for healing me. I have directed people with Cancer, Barren women,
    and those with different problem to him and he favored them differently.I have not seen any one as powerful as the

    lord spiritual. Just put him to test and see what he can do. (Seen is believing).for more information contact me on beatricemark0032@gmail.com.
    I vowed to testify of what he has done. For any problem just contact or email him on LORDSPIRITUAL@OUTLOOK.COM