Houston Gene Therapeutics Makes Progress in the Fight Against Diabetes
Houston, Texas - Adeno-associated virus (AAV)-based gene therapy is a new type of medical treatment which is competing favorably with older medical treatment technologies. The medical treatment part comes from the use of human genes as “drugs” to treat disease, as opposed to small molecule drugs, otherwise known as “pills”, which are the main product of “big pharma.” We expect most gene therapies to be more effective (as they use actual human genes), longer lasting (10 years), and safer (has a disease-responsive gene expression system, expressing only where needed).
As a company strategic policy we have chosen to target inflammatory diseases as there are few competitor companies and the market is bigger, as well as more persons die from inflammatory disease than cancer. To this end, recently, we developed and US patented (USP 11,091,524, USP 11,274,134), an AAV-based gene therapy vector, HGT-Cardio-1, to treat cardiovascular disease. HGT-Cardio-1 was a multi-component AAV vector which contained a small inflammation-responsive promoter (eNOX1pr), as well as two anti-inflammatory therapeutic genes (human FoxP3[P1] and human IL-10). The HGT-Cardio-1 vector was able to inhibit, treat, established and ongoing atherosclerosis when tail-vein injected into LDLR-KO mouse/HCD (high cholesterol/high fat diet) in pre-clinical experiments. The markets of patients with atherosclerosis are in the tens of millions in the US.
It is astonishing and somewhat grim that there are so many inflammation-driven diseases of humans, and which increase in number as we age. We considered it fair chance that HGT-Cardio-1 might also treat other such diseases in addition to atherosclerosis. One obvious target inflammatory disease is type 2 diabetes (T2D) which is epidemic in the US, again present in the tens of millions. In mouse experiments with LDLR-KO mice we placed animals on HCD, and added in sugar water to promote/induce diabetes in the animals. After placing these mice on HCD/sugar for two months, we injected the mice with HGT-Cardio-1 or a null-AAV vector. Two months further on we tested the animal groups with the mouse equivalent of glucose tolerance test, feeding them high sugar water, and followed the level of sugar in the bloodstream over time. The glucose tolerance test is routinely given to persons to check for T2D. It was found that untreated HCD/sugar diabetic mice exhibited classical elevated and time–extended blood-sugar levels compared to normal control mice. Opposed to this, animals maintained on the HCD/sugar, but injected with the HGT-Card-1 vector in the middle of the experiment, exhibited the same reduced sugar blood levels over time as the control normal animals. In other words, the HGT-Cardio-1 vector treated the T2D.
The finding that HGT-Cardio-1 was able to treat mouse models of both atherosclerosis and T2D, further strengthens the possibility that other inflammatory disease, beyond just atherosclerosis and T2D, could be treated as well by this one gene therapy treatment product.
Houston Gene Therapeutics is presently entertaining investments towards funding, bringing, the HGT-Cardio-1 product to the clinic, closer towards fulfilling FDA requirements and into clinical trials.
