gene therapy

“And last year saw the approval of the first gene therapy for a treatment of a rare form of blindness.”

“So gene therapy has been the holy grail for treating the approximately 5000 known diseases that are caused by a single gene mutation.”

“So as we advance our ability to deliver gene therapy, as we advance our ability to actually very precisely edit the genes, and only the genes that we need to edit, gene therapy will become applicable to a far broader range of diseases, not just limited to thos…”

“Gene therapy, an area that historically has been considered to be very risky, has been shown to be so effective in this form of treating inheritable form of blindness, That that passed the FDA panel recommendation unanimously, a 13 to zero vote. CAR T similarl…”

“We've done fifteen billion dollars of acquisitions just last year in the space, not, not including all of our internal work in each of these areas.”

“If you actually think about gene therapy and new technologies, they are creating the moat. Meaning, for example, Avexis and SMA, they are the, probably the leading gene therapy program out there, and they want to continue building that franchise and have not o…”

“To jump in, probably the natural place to start is on the AV side, as these adeno-associated viruses are the only clinically approved vector so far for gene therapy.”

“If we're able to make a medicine that does that, that can deliver one gene to a given cell type, it becomes increasingly likely that we'll be able to deliver a different gene to a different cell type for a different disease.”