Patient Specific Exon Skipping -NOW!

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Comment by Nimi Langer on March 17, 2012 at 3:14pm

Dear all:

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.

Comment by Ofir Arad on March 9, 2012 at 10:23am

Dear Nimi, can you update us please about this initiative?

Comment by Ambrish Kapadia on November 10, 2011 at 11:11pm

Hello Nimi,
it is nice to hear that u are working extremely hard in this direction of exon skipping.
I wish u all the best in your endevours.
My son is born 9th may 2007.His missing exons are 52 to 54 and is eligible for exon skipping 55.
All the best to u and your family.
Rgs ambrish (from mumbai india) tel+91 9870304838

Comment by Jon Moulton on November 10, 2011 at 12:46pm

I think your focus on regulatory change is good strategy.

http://www.myspace.com/127542408/blog/477387107

Good luck in your venture!

Comment by Nimi Langer on November 10, 2011 at 4:34am

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:

1. Don't forget that based on improved efficacy using the methodology of patient specific exon skipping we would need less AON.
2. The cost reduction of cost of manufacturing will come also with improve methodologies of delivery such as peptides and AVV. Both still have some challenges with the main one being toxicity.

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,

Nimi

Comment by Keith Van Houten on November 9, 2011 at 6:54pm

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.  

 

Thoughts?

Comment by Nimi Langer on November 8, 2011 at 2:21am

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,

Nimi

Comment by Nimi Langer on September 27, 2011 at 2:56pm

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…

But…

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.

Nimi

 

 

 

Comment by cheryl cliff on September 27, 2011 at 12:06pm

Nimi,

What study is in pre-clinical phase?   

Comment by Debbie Bowman on September 27, 2011 at 11:02am

Does the press release mention how readily available this would be in the US, if they go to clinical trials?

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