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Calimmune: Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-

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This is an early phase research study looking at whether an experimental gene transfer, LVsh5/C46 (also known as Cal-1), is safe and if it can protect the immune system from the effects of HIV without the use of antiretroviral drugs.

Cal-1 is an experimental gene transfer agent designed to inhibit HIV infection through 2 active parts:

Removing a protein named CCR5 from bone marrow and white blood cells
Producing a protein named C46 on bone marrow and white blood cells
Detailed Description   
It is estimated that 33 million individuals are currently infected with HIV. HIV/AIDS is a disease that impairs immune function, primarily by decreasing CD4+ T lymphocytes. The progression can be contained by daily dosing with antiretroviral therapy (ART) but there are side effects that can be treatment limiting, and the development of HIV drug resistance can force the physician to modify the ART regimen. There are no effective vaccines currently available for HIV.

LVsh5/C46 (also known as Cal-1) is a dual therapeutic, self-inactivating lentiviral vector that encodes for both a short hairpin RNA against the HIV-1 co-receptor CCR5 (sh5) and a HIV-1 fusion inhibitor, C46 and inhibits two processes required for HIV-1 infection:

Binding of the virus to the cellular CCR5 co-receptor and
Fusion of the virus with the host cell
The rationale is that Cal-1 introduced into hematopoietic progenitor/stem cells (HSPC) and mature CD4+ T lymphocytes will protect these cells and their progeny cells from HIV-1 infection and its pathogenic sequelae. This may provide a continuous means of controlling HIV-1 after a single or infrequent dose(s), thereby decreasing or delaying (partially or completely) the need for antiretroviral drug therapy.

It seems they want to repeat the success of the "Berlin Patient" but without a transplantation.

I've been on the list at Quest Research in SF for this trial.  I've been following the Sangamo trials (also at Quest) and am going to wait and see what becomes of them first.

Just got an email from Quest -- they are now recruiting for the CalImmune trial.

Maybe You are "cured" soon. I would be scared if somebody botches on my lovely genes. But CalImmune works with Benitec, and they really have the money and experience with the small hairpin ddRNAi, I think it will be a succes. :)


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