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

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generally well tolerated. The next step was to track the fate of the genetically modified, transplanted T-cells by exposing them to the HIV virus during a temporary interruption of antiretroviral drug treatment. Six of the trial participants suspended their HIV drug regimens. The T-cells with disabled CCR5 genes survived better than the unmodified T-cells, Sangamo announced. A key aim of current HIV drugs is to maintain T-cell population numbers at normal levels.

Sangamo also revealed later tweaks to its SB-728-T therapy in data presented at an HIV/AIDS conference on Thursday. In another small early stage trial, investigators used a preconditioning treatment before the trial participants received the infusion of their genetically modified T-cells. The pre-treatment with the drug cyclophosphamide (Cytoxan), which is typically used against cancer or autoimmune diseases, helped the modified T-cells establish themselves in the body, and also enhanced the control of viral levels once antiretroviral drug treatment was suspended in some trial subjects for several weeks, investigators found.

The much-discussed star participant in Sangamo’s trials has ceased taking HIV drugs for about seven months while maintaining undetectable counts of HIV virus. In that individual, however, one of the two original copies (or alleles) of the CCR5 gene is the rare, natural variation that makes T-cells resistant to the entry of the most common form of the HIV virus. After Sangamo’s treatment, it’s assumed, both copies of the CCR5 gene were inactive in many of that individual’s T-cells—and this may be the reason why HIV levels became undetectable.

Sangamo is now planning to alter its ZFN method of editing genes to increase the chances that it can disable both copies of the CCR5 gene in future trial participants. (Every cell in the body, except for eggs and sperm, carries two sets of genes, one set inherited from each parent.)

“To make a therapy, we’ll need to knock out both alleles,” Lanphier told me Thursday during a day of back-to-back media interviews.

While gene therapy may seem to move forward by such inches, tweaks, and increments, the wider story in the Sangamo announcements this week is the growth of a set of technologies shared among a community of scientists and companies working on genetic modifications to treat a variety of different diseases.

A senior author of the New England Journal of Medicine paper about Sangamo’s first HIV trial is Carl June, a professor of immunotherapy at the University of Pennsylvania, who also drew attention at the annual meeting of the American Society of Hematology in December with the favorable results of an experimental gene therapy treatment for two blood cancers, chronic lymphocytic leukemia and acute lymphoblastic leukemia. The Swiss drug giant Novartis (NOVN) has licensed the method, Bloomberg reported from the conference.

Sangamo itself is crossing the jurisdictional lines of disease. Its pipeline includes experimental therapies for the genetically based blood disorders beta-thalassemia and sickle cell disease; in January it announced a partnership on that work with Cambridge, MA-based Biogen Idec (NASDAQ: BIIB). Sangamo is also working on potential gene-altering treatments for hemophilia A, hemophilia B, and Huntington’s Disease in a collaboration with Shire.

“ZFN could be applied to whatever gene we want,” Lanphier says.

Sangamo also isn’t alone in its pursuit of a gene therapy for HIV. Both Sangamo and Los Angeles, CA-based Calimmune have received grants from California’s stem cell research funding agency, the California Institute for Regenerative Medicine, for their work on experimental HIV therapies involving stem cells. Using different techniques, each company is genetically modifying the hematopoietic stem cells that circulate in the blood and give rise to a stable of blood and immune system cells.

In Sangamo’s treatment, the re-engineered stem cells would be re-introduced into the body through a “low-intensity bone marrow transplant,” says Geoff Nichol, Sangamo’s executive vice president of research and development. Theoretically, the modified stem cells would then produce a range of different types of blood-borne cells protected from HIV infection—not only the T-cells that are the targets of Sangamo’s completed trials using modified T-cell infusions. Other immune cells generated by genetically modified stem cells might be enabled to kill the virus, Nichol says. Sangamo plans to seek FDA permission this year for clinical trials of that method.

However, the gene therapy company that succeeds in developing a functional HIV cure may some day face competition from a longtime rival: Gilead. The kingpin of antiretroviral HIV drug sales is conducting its own research programs on potential cures for HIV infection that don’t rely on gene modification. Gilead is studying drug compounds with the potential to eradicate all vestiges of HIV in the human tissues that serve as reservoirs where the deadly virus can hide out.

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11 responses to “HIV Treatment Outlook Buoyed by Sangamo Gene Therapy”

  1. jack says:

    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..

  2. MineMan says:

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

    • Sophia Blaze says:

      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

  3. Sophia Blaze says:

    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

  4. Sophia Blaze says:

    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

  5. Isaac John says:

    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

  6. RODA says:

    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,,,,,,,,,,,,,,,

  7. Theodora Fabzz says:

    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.

  8. Isaac John says:

    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 ,,,.

  9. Beatrice Mark says:

    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