hsimp....berry is taking action against the people on this Yahoo board. Corrective action. And he's done. He accomplished that mission in one post - right here! Re-read his first sentence, Way to miss the point and go off on a tangent! Get some sleep.
What's a "contrast agent"?............
Non-ionic, Low-Osmolar Contrast Agent
 As disclosed herein non-ionic, low-osmolar contrast agent refers to any non-ionic, low-osmolar substance used to enhance the contrast of structures or fluids within the body in medical imaging. It is understood and herein contemplated that non-ionic, low-osmolar contrast agents bind to a target tissue or other molecule through hydrogen bonds between the contrast agent and the charged surface of the proteins on the surface of the tissue or to another nucleic acid, amino acid, peptide, or protein. Such agents include but are not limited to iobitridol, iohexyl, iomeprol, iopamidol, iopentol, iopromide, ioversol or ioxilan. Thus, for example, disclosed herein are compositions comprising an ASO and a non-ionic, low-osmolar contrast agent, wherein the non-ionic, low-osmolar contrast agent comprises iohexyl."
I guess it's just what they inject you with before a medical imaging procedure is performed. Kind of an interesting discovery - that it enhances PMO delivery, maybe as well as anything else we've discovered.....? It's nice that we apparently discovered this and are trying to patent it in this usage.
NON-IONIC, LOW OSMOLAR CONTRAST AGENTS FOR DELIVERY OF ANTISENSE OLIGONUCLEOTIDES AND TREATMENT OF DISEASE ........from two months ago.
Here's an excerpt that tells the story pretty well:
"Previously, single bolus injections of morpholino were performed at PND30 via stereotactic injection into the ventricle without any marked improvement in survival. However, this initial experiment was done with naked morpholino and in adult animals there is minimal spread of the naked morpholino from the injection site (FIG. 3D). This problem is solved (FIG. 3D) by complexing the morpholino with Iohexyl. This Reagent is compatible with clinical practice and has a known toxicity profile. It can also be noted in FIG. 3D that annealing the morpholino with a complementary DNA molecule resulted in increased delivery, but was not as effective as Iohexyl. The negatively charged clamped morpholino (Dimer) can distribute in a similar manner to MOEs. Thus naked morpholinos do not distribute as widely as MOEs. However, this is easily overcome by the novel addition of Iohexyl. Furthermore, due to their low toxicity, morpholinos can be used at much higher concentrations than MOEs, even when complexed with Iohexyl."
....a bit more:
"The development of successful gene therapies for motor neuron disease will likely require widespread transduction within the spinal cord and motor cortex. Two of the most common motor neuron diseases are spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS), both debilitating disorders of children and adults, respectively, with no effective therapies to date. Recent work in rodent models of SMA and ALS involves gene delivery using viruses that are retrogradely transported following intramuscular injection. However, clinical development may be difficult given the numerous injections required to target the widespread region of neurodegeneration throughout the spinal cord, brainstem and motor cortex to effectively treat these diseases."
Trying to earn that lousy Christmas bonus, pathetic one? That took a lot of time to think up and type! Last night's homework? Way to show us how desperate you are. Glad you did buy some at $14. Your pay scale could use the augmentation. good luck on getting that low this week. Next week are you off?
Chris has balanced the company's financials perfectly as the FDA dragged it's feet, got pushed forward a bit, dragged i's feet again, got pushed forward a bit again, dragged it's feet yet again ... and on it goes.
Meanwhile Chris has husbanded well and also carefully employed the 300 million he very cleverly wrung out of the Street during this ongoing saga to, in fact, work on the other aspects of the company's future. He has saved a fortune by holding FDUSA over the head of the FDA - and his supposed "delays" in starting large extremely expensive trials (that some here crazily think we should have jumped at the chance to rush into!), trials that FDUSA is specifically designed to eliminate the need for! - Chris's moves (or "delays") are and were exactly the RIGHT strategy for this company.
We now will be giving the FDA their lousy 12 to 24 new kid safety data - culled from the two trials now in progress - and with approval coming "on schedule" after that NDA submission in June, and the incredible expense of the drugs were now shelling out for in these 4 or 5 new trials will be soon covered by insurance companies. This is the way Chris is squeezing as much FDUSA benefit out of a FDUSA law that that FDA is stubbornly ignoring. It's really not that hard a strategy to see. And, of course, it's pretty ingenious ... and it seems to be working.
Of course, the complexity and time taken could all have been - and still be in future - avoided at any time had the FDA chosen to use the powers granted then by FDUSA. At any time! As Chris has noted repeatedly. But they chose not to and so Chris had to work around them. And he did. And he raised over 300 million doing it. And built a very strong foundation for a lasting company in the meantime. And you geniuses snarl at him every day! Amazing.
A.F. stands for "a fool". Lives in a dream world of random thoughts and emotions ... much like Starfe, actually. I'm glad Sarepta confuses him so much. Shows him for the fool he apparently is.
He raised and husbanded some 300 million in his game with the FDA using FDUSA. without that money how would we have afforded these 3 or 4 trials they are demanding? Before we got the 300 million Chris had a very good "excuse" as you call it. No money. That's why the FDUSA law was passed. Even without the FDA adhering to that law, Chris played that law and over time managed to get the money needed. He used a law that he probably suspected would never be actually adhered to to none-the-less get the money the law was intended to make unnecessary for a small company to come up with to bring a new kind of drug to market. He used the threat of FDUSA to give him a few moments of FDA head fakes to raise the money anyway. Get it?
What's "the incompatibility of the FDASIA law" supposed to refer to? We know what the law says. The law is clear. It's also clear the FDA has always intended to dance around it. The problem is the FDA MAY ALWAYS INTEND TO DANCE AROUND ANY SUCH "LAW". ... If some future president makes up his mind to fix that problem once and for all, then perhaps we can have hope. Until that happens, forget it. In 7 months or so they may apply FDUSA to our drug but it will be a one shot deal. That's pretty obvious. By the time our next drug is awaiting their blessing we may have elected a competent and determined can-do president to clean out those stables once and for all. Not yet another lazy distracted go along to get along nobody
Our drug is "small company (us) killing expensive". Big trials earlier may have wounded us or killed us. That's the rationale behind the FDUSA law. Chris has been trying to leverage that law to bring this drug through this "valley of death". The FDA has been fighting that law the whole way. Now we've started the big expensive trials. Now we wait until it pleases the FDA to make their peace with FDUSA. We are being squeezed monetarily and the FDA is being squeezed by the parents and the Congress and the impotent law. In about 7 months the ketchup will become unblocked from the bottle's neck. Once approval comes the company can stop bleeding money feeding drug to these trials. (Insurance companies then come to our rescue).
That's been the game here. Luckily, Chris has seized on the moments the FDA has had to relent and pretend they are following the intent of the FDUSA law and Chris has raised mucho dollars at those moments to raise the cash we've needed to get us to where we can afford to live without FDUSA. If we have to.
For playing this game as long as he has, and winning it, Chris deserves immense praise. Many fools here never understood what this game was or how Chris was playing it. That's pretty lame.
We can repeat this today, tomorrow and everyday until the drug is approved. As we've repeated it now for oh maybe 5 years now. If you are trying to prevent "the shorts" from any plan they may have to disparage the 168 week data you can forget it. They will do it if they want to. They will manipulate the stock price down as if it was all a real concern. And you will all cry bloody murder and rend your garments, wring your hands and cry to the heaven above for justice. You won't get justice if the shorts don't want you to get justice when that data comes out. Resign yourself to your fate. You can not win trying to out-manipulate the perceptions the big boys want to create. Really. Forget it. Five years of explaining that the drug only seeks to stabilize or improve natural history expectations is enough, I think. Enough. Through up your hands right now. Walk away. And wait for approval.
Mina Therapeutics looks like interesting science. Upregulation of proteins. Seems like there is a lot of potential in what they are doing. Unique idea as far as I can tell. And they are getting unexpected added results (positive) from their experiments. Much more interesting than merely blocking a gene. Non-public as of now. Worth watching.
There's a patent or patent application on it from a while ago ... and, as I say, Chris was asked about it at the end of his Stifel presentation. It's going to be for real.
Yeah well...Google yields one single result for Conrad's "Sarepheth". But of course Conrad may have written "his" bible for himself.
Elijah from Tishbe, who was one of the settlers in Gilead, said to Ahab, “As surely as the Lord lives, Israel’s God, the one I serve, there will be neither dew nor rain these years unless I say so.”
2 Then the Lord’s word came to Elijah: 3 Go from here and turn east. Hide by the Cherith Brook that faces the Jordan River. 4 You can drink from the brook. I have also ordered the ravens to provide for you there. 5 Elijah went and did just what the Lord said. He stayed by the Cherith Brook that faced the Jordan River. 6 The ravens brought bread and meat in the mornings and evenings. He drank from the Cherith Brook. 7 After a while the brook dried up because there was no rain in the land.
Elijah and the widow from Zarephath
8 The Lord’s word came to Elijah: 9 Get up and go to Zarephath near Sidon and stay there. I have ordered a widow there to take care of you. 10 Elijah left and went to Zarephath. As he came to the town gate, he saw a widow collecting sticks. He called out to her, “Please get a little water for me in this cup so I can drink.” 11 She went to get some water. He then said to her, “Please get me a piece of bread.”
12 “As surely as the Lord your God lives,” she replied, “I don’t have any food; only a handful of flour in a jar and a bit of oil in a bottle. Look at me. I’m collecting two sticks so that I can make some food for myself and my son. We’ll eat the last of the food and then die.”
13 Elijah said to her, “Don’t be afraid! Go and do what you said. Only make a little loaf of bread for me first. Then bring it to me. You can make something for yourself and your son after that. 14 This is what Israel’s God, the Lord, says: The jar of flour won’t decrease and the bottle of oil won’t run out until the day the Lord sends rain on the earth.” 15 The widow went and did what Elijah said. So the widow, Elijah, and the widow’s household ate for many days. 16 The jar of flour didn’t decrease nor did the bottle of oil run out, just as the Lord spoke through Elijah.
17 After these things, the son of the widow, who was the matriarch of the household, became ill. His sickness got steadily worse until he wasn’t breathing anymore. 18 She said to Elijah, “What’s gone wrong between us, man of God? Have you come to me to call attention to my sin and kill my son?”
19 Elijah replied, “Give your son to me.” He took her son from her and carried him to the upper room where he was staying. Elijah laid him on his bed. 20 Elijah cried out to the Lord, “Lord my God, why is it that you have brought such evil upon the widow that I am staying with by killing her son?” 21 Then he stretched himself over the boy three times and cried out to the Lord, “Lord my God, please give this boy’s life back to him.” 22 The Lord listened to Elijah’s voice and gave the boy his life back. And he lived. 23 Elijah brought the boy down from the upper room of the house and gave him to his mother. Elijah said, “Look, your son is alive!”
24 “Now I know that you really are a man of God,” the woman said to Elijah, “and that the Lord’s word is truly in your mouth.”
No version with an "S".
zar'-e-fath (tsarephath; Sarepta): The Sidonian town in which Elijah was entertained by a widow after he left the brook Cherith (1 Kings 17:9). Obadiah refers to it as a Canaanite (probably meaning Phoenicia) town (Obadiah 1:20). It appears in the Greek form Sarepta in Luke 4:26 (the King James Version)
As I recall, Chris was asked a question about it too....As to whether it could serve as a combo therapy for DMD. And he said that that was the idea. (If memory serves.)
jrrt -- did you find the myostatin pmo drug listed in the stifle slide?