AveXis Reassures SMA Community is Safe and ‘Transformative'

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    • #11196

      AveXis executives late last week reassured everyone in the SMA community anticipating their promising experimental AVSX-101 SMA therapy that, in spite of a recent study that showed a similar viral vector was toxic when used in high doses on animals, it remains both safe and “transformative.”

      The AveXis comments were published as part of an exclusive interview by SMA News Today. You can read the article here.

      This recent study in question, which was conducted on monkeys and piglets by researchers at the University of Pennsylvania Perelman School of Medicine, revealed that high doses of the AAV9 vector caused life-threatening toxicity. AAV9 is a harmless virus that AVXS-101 uses to cross the Blood Brain Barrier in SMA patients and deliver gene therapy that impacts SMN function.

      The news last week sent the gene therapy community into a swoon, crashing stock prices of several pharma companies that are developing treatments based on the use of viral vectors. One of these is AveXis, whose AVXS-101 therapy is highly anticipated by the SMA community.

      In their SMA News Today interview, AveXis’ Chief Medical Officer, Sukumar Nagendran, explained that, while similar, there are “many nuances” between the AAVhu68 vector used in the study, and the AAV9 vectors being used in the SMA patient trials, making it like comparing apples and oranges.

      AveXis once again stated their strong safety profile for AVXS-101, with company CSO Brian Kaspar noting that “. . . some of the children have been on the therapy for nearly four years” with no serious health issues caused by the therapy whatsoever.

      Tell us what you think about the recent AveXis news.


    • #11197

      Get a little closer as to who will be funding Wilsons gene therapy for SMA – big company named BIOGEN. Does it all make sense now as to why there was this scare? What AveXis and Kasapar said believe it.

      • #11201

        AveXis has definitely faced an uphill battle at times in developing its SMA therapy. But I think that the data continues to look really, really promising. It will be an amazing breakthrough if the SMA community can get two complementary therapies approved that can potentially be administered together (or at least at the same time).

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