Our Technology for HIV
Gene-Modified Cell Therapies for HIV
Gene-Modified Cell Therapies for HIV
Antiretroviral therapy (ART) has changed HIV-infection from a death sentence to a chronic disease. However, ART requires life-long therapy that is expensive and has the risk of significant side effects. In addition, drug resistance is growing, which requires new and often more costly products. From a patient-centered approach, life-long therapy can be challenging, and unfortunately, stigma and discrimination remain strong throughout the world. More than 40 percent(40%) of the 36.7 million people who need therapy do not have access to it. More than one million people die from HIV every year, and more than 1.8 million become newly infected. A cure and a preventive or therapeutic vaccine could transform the lives of millions of people.
It has been proven that gene-editing to knockdown the expression of CCR5 — a door HIV needs to enter and kill CD4+ T cells — in autologous human stem cells (HSC) combined with transplantation can lead to a cure of HIV. However, the approaches currently available require an expensive and risky ablation of the immune system. Even with that drastic intervention, an insufficient number of gene-modified cells survive to achieve durable control of HIV.
We have pioneered a novel approach that we believe will allow sufficient engraftment of the gene-modified HSC to eliminate the need for ART.
In vivo and in vitro data supporting the scientific foundation of the approach has been presented at the annual meeting of The American Society of Gene and Cell Therapy in May 2020.
Management considered the FDA INTERACT Meeting to be successful with alignment with Enochian’s experimental plan. Additional in vitro and in vivo experiments are underway. Pre-IND submission is possible by the end of 2021, potentially with human studies beginning in 2022.
Boosting a person’s immune system through vaccination can lead to protection from HIV infection in people who are not living with HIV. In persons living with HIV who are controlling the spread of virus with antiretroviral (ARV) treatment, boosting the immune system in a different way than the virus already has through infection, could allow control of HIV after stopping ARVs.
Enochian Bio’s technology uses the powerful induction of an immune response created by cells from another person potentially to induce such a response. Based on promising in vitro results, a study in non-human primates was begun by the renowned HIV and Cancer researcher Dr. Hans Peter Kiem of the Fred Hutchinson Cancer Research Center, Seattle, Washington.
Preliminary results are expected by the end of 2021 or early 2022. If successful, human studies potentially could begin in 2022.
On June 14, Enochian Bio announced that the FDA has accepted a Pre-IND (Investigational New Drug) request for a potential functional cure or treatment of HIV. Written comments are expected this Fall.
Dr. Serhat Gumruçku, co-founder and inventor of Enochian BioSciences, and Director of Seraph Research Institute (SRI), submitted the Pre-IND. The request was based on the results of a 54-year old man living with HIV who had failed to suppress the virus with antiviral therapy. The patient subsequently achieved viral control for 255 days with an innovative treatment of Natural Killer (NK) and Gamma Delta T-cells (GDT) collected from another person. During the entire period, no antiviral drugs were given. It is believed that the GDT cells, a small subset of immune cells that can be infected with HIV, could be a key factor in controlling the virus.
The findings were presented during the Conference of the American Society of Gene and Cell Therapy this past May. Presentations can be found at here.
Enochian BioSciences holds the exclusive license for the proprietary technology.
This innovative cellular therapy could be an important approach to achieve a “functional cure” of HIV, potentially allowing persons with the virus to stop antiviral treatment for extended periods of time. The Pre-IND submission requested that the novel strategy be extended to persons with HIV who have achieved suppression of the virus with antiviral treatment.
HIV is an RNA virus. Based on the Hijack RNA Platform, an approach to “seek and kill” HIV-infected cells has been developed. In vivo and in vitro studies should begin in the near term.