At present, despite an epidemic of staggering proportions, we have no pharmaceutical “cure” for the HIV virus. The Highly Aggressive Anti-Retroviral Therapy (HAART) has been inarguably effective at prolonging life and improving quality of life, but the fact that HIV can remain dormant in the genome in just about any tissue in the body, coupled with its capacity to rapidly change its antigenic determinants, makes a pharmacological cure unlikely. Also, pharmacological regimens come with sometimes unpleasant side effects, and occasionally stop working altogether, necessitating a change in medications. Additionally, although the drug regimens remain quite lucrative for the pharmaceutical industry, and although death rates from AIDS have fallen, the epidemic is still expanding; these medications, at $10,000-$15,000 per person per year (pppy), have to be administered indefinitely, and thus pose an unsustainable economic burden.
We are proposing here a completely innovative approach to gene therapy: an approach which builds on proven research from a number of sources. The FIV-based vector has been validated for gene delivery both in vitro and in vivo. The therapeutic gene has been proven to trap HIV particles and prevent their release from infected cells. We plan to make use of recent advances in CRISPR/cas9 technology to insert the transgenes into the human genome site-specifically, to reduce the risk of oncogenic activation or other positional effects. This vector will confer lasting protection against HIV in uninfected, as well as infected individuals, after a single administration.
Our vector is a radical departure from traditional vaccine-based therapies, which have been slow to bear fruit in clinical trials. Importantly, the vector design is a platform technology: the same auto-regulatory mechanism might be utilized against other viral infections of broad social and medical significance.
Most critically, the vector will enable the delivery of small peptides, called “aptamers,” to neutralize HIV antibodies in HIV-positive individuals, allowing sero-reconversion, i.e. a return to an HIV-negative state.
If you are interested in learning more about our research program, or would like to initiate a discussion about collaboration or investment, we would be very glad to hear from you. If you are aware of any researchers who might be similarly interested, we would ask that you forward this information to them, in hopes of opening a dialogue. Opportunities for researchers who might be interested in participating in this project would include sponsoring applications for a Transformative Research, GOALI, SBIR, STTR and other grants. Thank you very much for your time and attention.