If you’re an avid Wall Street Daily reader, you already know what molecular scissors are.
But if this is your first time seeing the term, here’s the gist.
It’s a moniker we use to describe Sangamo Biosciences’ (Nasdaq: SGMO) gene therapy called “zinc finger nuclease” (ZFN), which allows them to cut harmful genes from cells in the body.
And one type of ZFN, known as SB-728-T, has our attention.
That’s because Sangamo recently conducted a successful clinical trial using the treatment to fight HIV.
During the trial, scientists took cells from patients with HIV and used SB-728-T to remove a gene that’s linked to the progression of the virus. Then they reintroduced the treated cells into the bloodstream.
At that point, they took patients off their standard HIV drugs to monitor how the new cells combated infection.
Sangamo discovered two important findings during the trial:
1. Treated patients experienced a boost in healthy T-cells that “no other treatment has been shown to do,” according to the company. And this includes patients who had shown resistance to current HIV drugs.
2. One participant, who I’ll call Patient X, with a mutation known as Delta 32 (which suppresses the HIV gene naturally), ended up with undetectable levels of the virus after treatment.
And now the company is using the data to conduct two new studies that take SB-728-T to the next level.
Delta 32 Mutation Put to the Test
First, Sangamo’s on the hunt for 20 more candidates with the Delta 32 mutation. Then it plans to conduct a similar study to further verify that those with the gene can more effectively repress viral load like Patient X.
This time, though, Sangamo is taking them off their HIV drugs for four more weeks to discover if ZFNs can keep the virus in check for longer.
About 10% of the population has the same mutation as Patient X, so this knowledge could at least ensure that ZFNs work for them.
But it’s the other new trial that really has me tuned in.
Replicating Patient X’s Response
For the second study, the company is testing a key hypothesis from the previous trial: If half of Patient X’s cells were already resistant to HIV to begin with, scientists believe that upping the amount of ZFN-treated cells should work for everyone else.
Makes sense. And to do this, they’re actually removing some of the patients’ own infection-fighting T-cells. This essentially makes room for the newly modified replacement cells to grow and multiply in the body.
Needless to say, if this proves effective, it would be huge for Sangamo. Because it would mean that every HIV-positive patient – with or without a special genetic mutation – could eliminate signs of the virus altogether.
As one researcher on the trial, Dr. Carl June, says, if this test proves successful, Sangamo “will achieve a ‘functional cure’ and eliminate the need for continued [antiretroviral therapy].”
Well, since ZFNs are simply making cells strong enough to keep the virus constantly at bay, it’s not exactly the same as zapping it from the body entirely. But it’s pretty darn close.
There’s also the fact that Sangamo doesn’t know how long the treated cells can keep the virus suppressed. Ideally, the treatment would eventually need to be administered once. Then the new HIV resistant cells would replicate enough to make the body self-sufficient against the virus.
At that point, Sangamo would have a powerful new treatment that could single-handedly turn the $15 billion HIV drug industry upside down.
Miracle cure or not, the company has already proven that ZFNs are capable of helping patients who are resistant to current drug therapies. And according to Bloomberg, as of 2010 that still represents 11% of HIV cases.
So either way, Sangamo’s at the cutting edge of a new revolution in gene therapy that I’m convinced will generate massive revenue streams for the company in the coming years.