Innovation Pharmaceuticals Inc (IPIX)

[Whoops]

TP53 gene encodes (makes) p53 mind you we are talking about an individual cell here. Without the gene there is no way for that cell to make p53 in the cell. If Kevetrin is only restoring the pathway from the gene to production of p53 that would mean the gene was never mutated and just blocked. Just restoring the pathway would not be sufficient to produce p53 if the gene is mutated it would have to know how to make it. I would consider restoration of the pathway to a functional gene to be gene therapy as well because it would be literally turning the gene on and off at least in terms of p53 production.

The effect of p53 has to be localized for efficient tumor cell suppression. Big pharma a decade or so ago tried flushing cancer patients with the p53 protein but it quickly became to toxic without much in the way of tumor suppression the attempt here is to get the individual cells to produce p53. I mention this Just in case Kevetrin is simulating p53 itself.

The FDA's definition of Gene therapy: Gene therapy is a medical intervention based on modification of the genetic material of living cells.

Genetic material: The genetic material of a cell or an organism refers to those materials found in the nucleus, mitochondria and cytoplasm, which play a fundamental role in determining the structure and nature of cell substances, and capable of self-propagating and variation.

The TP53 gene, the MDM2 pathway and p53 protein are in the nucleus, through the nuclear envelope and in to the cytoplasm.

I don't care to nit-pick about what Gene therapy is...but I would caution you about announcing that you know the method of action (MOA) of Kevetrin. I am not even sure Dr. Menon knows all those details yet.










Kevetrin doesn't really activate the TP53 gene, rather the gene product p53 (protein of 53 kDa size), via multiple mechanisms. We are not modifying any genes.

Gene therapy would be, as you said, insertion or knockout of genes, or modification of (mutated) genes. Human clinical trials for gene therapy have to go through additional precautions, such as staggered starts, the recombinant DNA advisory committee, and other.

http://osp.od.nih.gov/office-biotechnology-activities/biomedical-technology-assessment/hgt/rac

Gene therapy companies include: SGMO, BLUE, KITE, etc... but not CTIX. Here is a list of all the Gene Therapy trials that have occurred in the US:

http://osp.od.nih.gov/sites/default/files/resources/PROTOCOL_0.pdf