Proprietary Enzo-Developed Transducing Vector
Delivers High Levels of Anti-HIV Genes to Human Cells
Enzo Biochem, Inc. (NYSE: ENZ.N) today reported the survival in circulation of genetically engineered blood cells in an HIV-1 human study. The study, a Phase 1 clinical trial sponsored by Enzo Therapeutics, is being conducted at the University of California in San Francisco and is viewed as a critical step forward in the Company's development of a gene medicine for the treatment of HIV-1infection.
Dean Engelhardt, Ph.D., Enzo Senior Vice President, presenting at the 7th Retroviruses and Opportunistic Infections Conference this week in San Francisco, reported that engineered cells have now been observed in circulation for a period exceeding four months. Furthermore, he said that these engineered cells carrying the anti-HIV
genes delivered by Enzo's proprietary HGTV43 vector are producing antisense RNA.
"The trial has progressed to the point where data has been obtained documenting the presence of anti HIV-1 antisense RNA in circulating peripheral blood mononuclear cells. To date, no other trial has reached this point in adult human subjects without first ablating the patient. The engineered cells have persisted incirculation and continue to produce HIV-1 antisense RNA," said Dr. Engelhardt. "We are very encouraged, and indeed even excited, by these
early data. Further studies, as well as an expanded patient
population, plans for which are underway, will be necessary in order to confirm our preliminary findings and determine the long-term effect of this medicine."
Dr. Engelhardt further reported that HGTV43 works to deliver the antisense genes efficiently and quickly to non-growing cells outside the human body. He reported that the process of transduction or adding the genes to the blood stem cells has been reduced to an 18-hour
process, fromseveral weeks previously, thus protecting the stem cells from undesirable differentiation. He also noted that the high rate of transduction in cells from the HIV-1 infected subjects has been consistent throughout the study. "This rapid and extremely efficient method of adding genes to subject blood stem cells played a major role
in our present achievements," said Dr. Engelhardt. "In the trial the stem cells are removed from HIV-1 infected human subject, transduced overnight and the next day injected back into the subject." He reported that the cells producing antisense RNA are designed to evade undesirable immunological responses which means they are benign in
terms of any possible immune reaction. |
