Why have we not been bought out? I know I will be crucified for this, but here it is.
The naked PMO's patent has been expired for years. We are now working on Wilton's and Koles' patents, which are the targets. Recently the Supreme Court has found that DNA targets, cannot be patented. We won the patent in the USA, for the morpholino. The FDA said that our patent was good, while Prosensa's patent based on antisense for exon skipping was too general. Therefore, in the USA, we can use the morpholino for exon skipping, but so can prosensa, as long as they don't use the morpholino. In Europe, just the opposite for several exons. More bad news. The naked PMO has difficulty entering the cells. That's why we can give such large doses. Probably 90% of what we give is cleared by the kidneys intact. If we add a delivery system, PPMO, we can also just deliver 6mg/Kg, and then we become toxic as well. Posters have pointed to our Marburg trial, stating how well we enter cells. Nonsense, we have to use a charge, PMO+, to gain entry to the cells and kill the virus.
All and all, with all these problems that have held us back, we have viable drugs, but they are not problem free. I don't believe this board influences the market, but I just want you folks to know what I know. Please don't kill me. I still think we have a viable platform going forward, and I am still invested...ruby
If anyone wants to dispute anything I've said, I welcome it.
Sentiment: Strong Buy
Arthur Rubinstein (Ruby): "Recently the Supreme Court has found that DNA targets, cannot be patented"
"rediculously" (as the non-pianist Rubenstein spells it)......wrong.
Not the famous romantic pianist again:... "Therefore, in the USA, we can use the morpholino for exon skipping, but so can prosensa, as long as they don't use the morpholino."
Perhaps he should change his name to Sammy Beckett or one the more slapstick absurdists.
Micro found the above (and I just quoted the shortest pieces of nonsense from it) "a good summary"....and then went on to assure us the PMO only gets into DMD cells because they are "leaky"...even though a recently published (2 weeks ago if not one week ago and talked about here on this board and on I.V. too, I believe) posits and seems to show that the PMO actually enters cells at the earliest stages of a new muscle cell's growth cycle. And so can be useful in a number of muscles diseases other than supposedly 'leaky" DMD cells as was once thought.
But Ruby's probably intentionally factually inaccurate post - designed by Ruby's creator (Piney) to stir up trouble and disciussion - elicited no intelligent discussion here at all........and so the Simp id was given the job. The discussion Simp stirred up looks like it's not even worth reading. God's knows what tired old pro Chris anti-Chris blather the "New" Regime posting stirred up. Any wonder that only a few newbie suckers get roped into the faux discussions here anymore? .... aside from the usual slew of fake ids - all created by the same nut-case who dominates the I.V. board as well.
Someday we have to hope Chris gets Sarepta to a point where the actual company provides the interesting stuff regarding this company .....and not one old nut on it's poster boards
Are you listening Chris? Save us!.
You are an ignorant person who has need of extensive DD. BTW...I have nothing to do with Piney...Heck, I find her annoying.
"Not the famous romantic pianist again:... "Therefore, in the USA, we can use the morpholino for exon skipping, but so can prosensa, as long as they don't use the morpholino. Perhaps he should change his name to Sammy Beckett or one the more slapstick absurdists"
Prosensa's claim to the U.S. patent office was that they have patented Antisense for exon skipping, while Serepta, claimed their patent was the use of morpholinos for exon skipping. The patent office found for Sarepta, saying, that Prosensa's claim was to general, while Sarepta was properly more specific. Therefore, if Prosensa wants to treat with their drug in the U.S. they are free to. In Europe they found for Prosensa for 2 exons, accepting the "antisense" term, therefore we cannot market in Europe.
Sentiment: Strong Buy
Eteplirsen 2025 (patents) – 2030 (patents)
Other DMD exons 2025 (patent applications) – 2030 (patents)
Exon-skipping 2013 (patents) – 2023 (patents)
Antivirals (Ebola, Marburg, Dengue and Influenza) 2022 (patents) – 2030 (patent applications)
chemistry (PPMO, PMOplus and PMO-X ) 2024 (patents) – 2032 (patent applications)
Antibacterials 2018 (patents) – 2031 (patent applications)
Other rare diseases 2025 (patent applications) – 2032 (patent applications)
Other targets and programs 2019 (patents) – 2032 (patent applications)
Some of our patents on core technologies expired in 2008, including a patent for our basic PMO chemistry. However, as we continue to advance the
research supporting our PMO-based technologies, we believe that the patented and likely patentable improvements we are developing will provide the necessary
basis to develop and exclusively commercialize our products. We also rely on trade secrets and proprietary know-how, especially when we do not believe that
patent protection is appropriate or can be obtained. Our policy is to require each of our employees, consultants and advisors to execute a confidentiality and
inventions assignment agreement before beginning their employment, consulting or advisory relationship with us. These agreements provide that the individual
must keep confidential and not disclose to other parties any confidential information developed ...........................................................................................-----------------------------##############
Sarepta has many patents with the use of pmo s ,the pmo itself is free to use but the certain patents with pmo-s are not free to use.....as far as ppmo ,Sarepta owns it as a whole and are great to use as a short term solution or much better long term than second generation antisense.Sarepta also owns 100 percent of pmoplus,pmo-x .......................
Good summary. If I recall correctly they are working on a different PPMO that they hope to be non toxic. The proof will be in the pudding.
Obviously as far as the naked PMO goes, everything depends on applications and any intellectual property that may protect them.
Further exon skipping targets, if restricted to the naked PMO would depend on cases where the cells were leaky enough to permit PMO entry.
There is the chemistry - PMO-X. I am posting on IV a L,I,N,K
There is also the antibacterials and antivirals.
What Sarepta needs to do is get DMD across the finish line in order to fund development in a wide range of applications and chemistry's.
The reason Dystrophin levels are almost impossible to relate to clinical benefit, becomes obvious in this example. My muscles have normal levels of Dystrophin. So does a track and field champion. Who do you think would perform better in a 6 MWT? There just isn't a direct relationship between levels of Dystrophin and physical performance.
Also, I believe the PPMO, is usable in any application, where the length of treatment is relatively short, like antivirals, but certainly not where lifetime dosing is required. The toxicity would not be of clinical significance over the course of a few weeks of treatment. JMHO
Sentiment: Strong Buy
Nice post MJ.
As we consider what would attract a partner:
Besides DMD what other disease can exon-skipping be expected to target?
Besides DMD what other disease can an antisense drug target?
The only partner for an antiviral program has been the US government. Besides the viral hemorrhagic programs what other viral programs can an antisense drug target?