The CEO of AVI Biopharma is speaking in Geneva tomorrow and the topic of the presentation is very interesting to me, as a father of a boy who is not in the "hot-zone" for current exon-skipping clinical trials.
Gaining approval of AVI or Prosensa treatments as a class is the only way those drugs can help my son in time.
Is anybody at PPMD going attending this conference? If so, can you publish the presentation?
I'm also very interested in this. My grandsons don't fall in the "hot-zone" either.
Hello David & Terry!
I checked in with everyone-- Although PPMD will not be attending this meeting, Sharon Terry from Genetic Alliance (USA) will be participating. After the meeting is over, we'll ask around to see if we can get a recap of Chris Garabedian's presentation.
When I asked Pat about this, she also wrote:
Discussions around this topic and efforts are underway to encourage regulatory agencies to approve AON (antisense oligonucleotides) as a class. It is believed that approving the backbone chemistry (prosensa’s 2 Omethyl and/or AVI’s morpholino) would facilitate and expedite approvals of specific sequences. It is estimated, that this approach, could potentially treat 80% +/- of the boys with Duchenne.
This does not mean that the regulatory agencies would step aside, rather they would require a modified toxicology package rather than the full tox package on every chemistry. The difficulty is that each chemistry will express (if successful) a different dystrophin protein and potentially different off target issues.
In general conversations, regulatory agencies in the US and Europe understand this high unmet need, the need to expedite therapies that are safe and effective. In general conversations both have suggested that once one or two of these AON’s are approved, they would have significantly more data and in a better position to discuss the concept of approving AON as a class.
I hope this helps!