A Major hunter of Mankind from whom our protective weapon have not been ready till now-HIV. Like a child's tiny crawls and steps,many trials started were ran aground.If any minor fortune turns in such development, it could be a giant leap of man kind.
Additionally, AntiRetroViral(ARV)Drugs used in treatment of HIV,fills most of the patients with loathing because of most of the undesirable and intolerable side-effects.
But, a ray of sunshine appeared,through,
Clinical Trial uncovers potential 'functional cure' for HIV/AIDS
Study:
--------
to develop a "functional cure" for HIV/AIDS
Study type:
--------------
Clinical trial-Phase 2
Method-:
-----------
Phase 1:
SB-728-T was given to HIV patients who were on highly active antiretroviral therapy (HAART) but were considered to be "non-responders" — that is, their CD4+ T-cell levels remained low. The patients' HAART therapy was interrupted when they received the SB-728-T therapy.
Subjects on HAART whose CD4+ counts remain below 500 cells per cubic millimeter — and below 450 cells in one cohort — after several years of therapy.
Phase 2:
Two new approaches to increase the number of engrafted T-cells in which both CCR5 gene copies are modified (biallelically modified) in SB-728-T-treated, HIV-infected subjects.
The first, an extension of an ongoing trial (SB-728-902, Cohort 5), is designed to further investigate the effect of SB-728-T treatment on HIV viral load in subjects that are naturally heterozygous for the CCR5 delta-32 gene mutation (i.e. one of their two CCR5 gene copies has the mutation and one is normal).
The second study (SB-728-1101), in HIV-infected subjects without the CCR5 delta-32 mutation, employs a conditioning pretreatment designed to significantly enhance the number of engrafted biallelically modified T-cells.
Findings:
-----------
Explanation:
---------------
ZFN-mediated gene editing technology to replicate a naturally occurring human mutation which renders individuals largely resistant to infection with the most common strain of HIV. CCR5 is a co-receptor for HIV entry into T-cells and, if CCR5 is not expressed on their surface, HIV infects them with lower efficiency.
ZFN modification disrupts the expression of this key co-receptor for HIV entry and renders cells resistant to HIV infection. The approach is based on the observation that a naturally occurring mutation in the CCR5 gene, CCR5 delta-32, provides protection from HIV infection.
Individuals who have the delta-32 mutation in both copies of the CCR5 gene are generally not susceptible to the most common strain of HIV. Those with only one copy of the mutation are identified as "elite controllers" because of their immune systems' ability to resist the progression of HIV without the need for HAART
Study publisher- Sangamo BioSciences, Inc [link]
Author comments:
----------------------
Edward Lanphier, Sangamo's president and CEO:
"We are focused on applying our ZFP Technology platform to develop novel therapeutics to address unmet medical needs.
In addition to the rapid progress that we are making in our clinical program to develop a "functional cure" for HIV/AIDS, we are advancing our preclinical ZFP Therapeutic programs to engineer genetic cures for monogenic diseases including hemophilias and hemoglobinopathies such as sickle cell anemia. Sangamo enters 2012 with a solid cash position which allows us to aggressively pursue our goals while maintaining our historic control on cash burn. As such, we plan to end 2012 with at least $60 million in cash. We look forward to providing further financial guidance for 2012 as well as an update on our clinical and preclinical programs and our corporate partnering activities on our fourth quarter and end of year 2011 call in early February."
Geoff Nichol, M.B. Ch.B., Sangamo's executive vice president, research and development:
"We are delighted to be able to open these two important clinical studies ahead of schedule,Data from earlier Phase 1 trials demonstrated a statistically significant relationship between the number of circulating T-cells in which both CCR5 genes are modified and the reduction in HIV viral load in infected subjects during an interruption of anti-retroviral therapy. Both of these new Phase 2 clinical trials are specifically designed to confirm and further investigate these findings."
Additionally, AntiRetroViral(ARV)Drugs used in treatment of HIV,fills most of the patients with loathing because of most of the undesirable and intolerable side-effects.
But, a ray of sunshine appeared,through,
Clinical Trial uncovers potential 'functional cure' for HIV/AIDS
Study:
--------
to develop a "functional cure" for HIV/AIDS
Study type:
--------------
Clinical trial-Phase 2
Method-:
-----------
Phase 1:
SB-728-T was given to HIV patients who were on highly active antiretroviral therapy (HAART) but were considered to be "non-responders" — that is, their CD4+ T-cell levels remained low. The patients' HAART therapy was interrupted when they received the SB-728-T therapy.
Subjects on HAART whose CD4+ counts remain below 500 cells per cubic millimeter — and below 450 cells in one cohort — after several years of therapy.
Phase 2:
Two new approaches to increase the number of engrafted T-cells in which both CCR5 gene copies are modified (biallelically modified) in SB-728-T-treated, HIV-infected subjects.
The first, an extension of an ongoing trial (SB-728-902, Cohort 5), is designed to further investigate the effect of SB-728-T treatment on HIV viral load in subjects that are naturally heterozygous for the CCR5 delta-32 gene mutation (i.e. one of their two CCR5 gene copies has the mutation and one is normal).
The second study (SB-728-1101), in HIV-infected subjects without the CCR5 delta-32 mutation, employs a conditioning pretreatment designed to significantly enhance the number of engrafted biallelically modified T-cells.
Findings:
-----------
- The viral load of one SB-728-T-treated subject decreased to undetectable levels during a treatment interruption. This subject was found to be an "elite controller."
- Unprecedented improvements in overall CD4+ T-cell counts and the ration of CD4+ to CD8+ cells, as well as engraftment, expansion, trafficking and persistence of the ZFN-modified cells.
- SB-728-T treatment continues to be safe and well tolerated.
Explanation:
---------------
ZFN-mediated gene editing technology to replicate a naturally occurring human mutation which renders individuals largely resistant to infection with the most common strain of HIV. CCR5 is a co-receptor for HIV entry into T-cells and, if CCR5 is not expressed on their surface, HIV infects them with lower efficiency.
ZFN modification disrupts the expression of this key co-receptor for HIV entry and renders cells resistant to HIV infection. The approach is based on the observation that a naturally occurring mutation in the CCR5 gene, CCR5 delta-32, provides protection from HIV infection.
Individuals who have the delta-32 mutation in both copies of the CCR5 gene are generally not susceptible to the most common strain of HIV. Those with only one copy of the mutation are identified as "elite controllers" because of their immune systems' ability to resist the progression of HIV without the need for HAART
Study publisher- Sangamo BioSciences, Inc [link]
Author comments:
----------------------
Edward Lanphier, Sangamo's president and CEO:
"We are focused on applying our ZFP Technology platform to develop novel therapeutics to address unmet medical needs.
In addition to the rapid progress that we are making in our clinical program to develop a "functional cure" for HIV/AIDS, we are advancing our preclinical ZFP Therapeutic programs to engineer genetic cures for monogenic diseases including hemophilias and hemoglobinopathies such as sickle cell anemia. Sangamo enters 2012 with a solid cash position which allows us to aggressively pursue our goals while maintaining our historic control on cash burn. As such, we plan to end 2012 with at least $60 million in cash. We look forward to providing further financial guidance for 2012 as well as an update on our clinical and preclinical programs and our corporate partnering activities on our fourth quarter and end of year 2011 call in early February."
Geoff Nichol, M.B. Ch.B., Sangamo's executive vice president, research and development:
"We are delighted to be able to open these two important clinical studies ahead of schedule,Data from earlier Phase 1 trials demonstrated a statistically significant relationship between the number of circulating T-cells in which both CCR5 genes are modified and the reduction in HIV viral load in infected subjects during an interruption of anti-retroviral therapy. Both of these new Phase 2 clinical trials are specifically designed to confirm and further investigate these findings."
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