>Wasn't there a European trial a few years back that set back gene therapy because of leukemia in some patients?<
Sirna-027 has hardly anything in common with those early gene-therapy experiments. It is administered locally in the eye and does not use a viral vector.
>It sure seems like a lot of people are working on wet AMD, does anyone know any promising dry AMD candidates (outside of treatments to stop progression to wet)?<
Arresting the progression to wet AMD is the most important thing a patient with dry AMD can do, so that’s where the attention will be focused. Dry AMD per se is not such an awful condition that it warrants the kinds of aggressive interventions that are justified for people with the wet form of the disease.
SGMO - Wasn't there a European trial a few years back that set back gene therapy because of leukemia in some patients?
Yes, the problem was that they used a viral vector to modify the DNA. Not enough specificity. SGMO is getting close to clinical trials in HIV that will once again attempt DNA modification. In this case, the modifications will be performed by SGMO's Zinc Finger Proteins (ZFPs) that can precisely target the exact spot where the modification is to occur. They also use a viral vector, but only to get the ZFPs into the cell. The virus isn't involved in the DNA modifications. The link below describes their current status.
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