NanoViricides Inc. (NNVC)

[ZincFinger]

Firstly the MonoGenic Diseases (MGD) applications of the IVPR platform are only a small part of what SGMO does.

Secondly the same approach (or a variation of it) being used against HIV could be used for almost any infectious disease.

For acute diseases they could use zinc fingers for regulation (i.e.: the AAV vector codes for zinc fingers that regulate rather than genetically modify genes. Since the ZFs themselves are transient, the drugs effects would last only a few days rather than the lifetime when the ZFs make genetic modifications).

THe relevant genes are for cell surface receptors that are highly significant in many disease: a single variation can often the the overriding determining factor in whether a patient is totally unaffected (never gets infected at all) or dies.

THe relevant receptors are mainly important in reactions to disease and there are many variants of them in the population including ones that are totally dysfunctional (as with the CCR5 deletion variant that provides total resistance to HIV (when homozygous). Homozygotes have no medical problems related to that (other than total resistance to HIV). That would certainly be the case with most such receptors and exceptions could readily be identified

In the relevant receptors there is an extremely high level of variation in the population precisely because of the protection most of them give against various diseases. Often one that protects against one disease confers heightened susceptibility to others. (the gene variation recently identified as conferring very high susceptibility to H7N9 is a good example It is 10 times as common in the Chinese population as in Europeans, almost certainly as a result of the difference profile of infectious diseases each population has been historically exposed to. (If H7N9 ever does get to the USA (the current version) the mortality rate may be far lower here than in China.) (IF and ONLY if it did not acquire the ability to use alleles common in the USA beforehand).

Those naturally homozygous in every single cell in their entire body for the CCR5 deletion that SGMO's drug creates ONLY in specific types of immune cells suffer no adverse health effects. So clearly SB728 is not going to have any "unanticpated side effects" . Much the same thing applies to most of the other relevant receptors.