Pushing forward a venture that will concentrate on that.
Location: Kibbutz Gesher Haziv, Israel
Latest Activity: Apr 5, 2016
Started by Vinu Joseph Apr 5, 2016.
Dear friends,I hope that you all are fine by grace of God.We are looking in search for any advancement in Exon 11 skipping. I have two children 9 and 5 years old both DMD with exon 11 skipping…Continue
Tags: to, 25, deletion, 12, exon
Any updates on this. My son is 9 years old and has deletion of 44 exon. The concept of patient specific exon skipping is very interesting. How is it progressing.
A short update regarding the initiative. Still struggling to get some funding together. I wish I had better news as I know this patient specific exon skipping one of the routes that needs to be explored more seriously. and now.
Dear Nimi, can you update us please about this initiative?
I think your focus on regulatory change is good strategy.
Good luck in your venture!
Keith thanks for your note
You are totally right the cost of the antisense is very expensive especially Morpholino. 2'OMe would be ~40K. BUT:
My hope is that by the time that we will manage to convince regulators to draft for us patient specific clinical protocol (that will pull like investors in) also delivery will improved and we will reduce the price of Cost of Manufacturing.
Take care and we will be in touch,
A scientist told me that the manufacturing cost of an exon skipping compound was over $100k per year. Which, to me, seems like it will keep the n=1 study scenario from ever taking place. These drugs will not be accessible to people unless they're FDA approved.
Hi to all:
I wanted to update you.
I just came back from the Action Duchenne Conference. The conference was wonderful and above all I met excellent people both families from our community and professional people as well.
Regarding Patient Specific Exon Skipping-
The vibe at the conference was that n=1 is the closest bet that we have and if you will enhance our commitment towards this aim it will come about sooner. And sooner is so much better. Sooner in this case could be a life saver.
I got a partially green light from an investor to start working on the project. I'm saying partially because I still have to show some milestones and matching funds on the level of 5:1 (the investor will invest 500k if there is another 100K from another investor or NFP. I hope I will manage to conclude all condition that will allow me to focus on the project and by that push the industry and regulators towards this aim.
I hear you parents that the process is frustrating and that we don't want to wait until Prosensa nor Avi will push this one forward. So I'm not waiting and that is what I'm doing.
I hope I'll have good news for you in the near future (February). It's annoying that it takes so much time to bring in the funds needed.
Take care to you all,
Thanks for all your comments.
Our study in Israel suggests a methodology for patient specific exon skipping treatment.
We are not better and even not close to have the experience and knowledge held by Avi or Prosensa…
What seems to me is that us parents need to put as much pressure and our loudest outcry towards regulatory agencies around the world to design a clinical protocol for patient specific therapy NOW!
Money from investors will not go in patient specific therapy until the regulatory agencies will make a move. The regulatory agencies won't make a move before someone negotiate with them professionally on a protocol and nobody negotiate because nobody gets paid to do so and nobody gets paid to do so because there are no investors and there are no investors because the regulatory agencies aren’t suggesting a clinical protocol economically feasible for patient specific therapy…
PPMD is working hard on this and I hear that Prof Wilton and his team are doing the best they can in Australia and I read what Prof Muntoni said and many other professors and scientists, but we need to join them and work globally on this one.
Patient specific therapy is expected to have a great impact especially on orphan diseases. Nobody chose to be in this community but now that we are here in an extended family, we have this great responsibility.
And this is why I opened the parent group. Let's think about ways to push this forward and take us, as fast as we can outside of this loop.
Tomorrow a new (Jewish) year starts. Happy new year to all.
What study is in pre-clinical phase?
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