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Biotech / Medical : Gene therapy -- Ignore unavailable to you. Want to Upgrade?

To: SnowShredder who wrote (126)	8/13/1999 5:35:00 PM
From: Mike McFarland	Read Replies (1) \| Respond to of 319

AAV--"site specific integration (if they can get it to work efficiently" In the recent Ariad-Genovo paper it was said that there was no reduction in hGH peak levels after each administration of the promoter--for more than ten months. Plus they said they could precisely control the circulating levels of hGH by controlling the rapamycin dose...and if they don't give the drug, hormone levels are undetectable. Is that efficient--or was your question something about directing the therapy to a given site and not having it bleed all through the system? I just assumed you shoot the stuff into a big muscle and most of it stays there. I guess I should go through the papers and keyword search for 'liver'? These were immunocompetent mice that did so well--I assume they're still boppin around with hGH spiked at will. My wife made me sell most of her ARIA when I explained they were working with monkeys, er, non-human primates seems to be the code so the animal rights folks don't perk up...but she let me buy her shares back when it was mice in the latest paper. Funny how nobody seems to be impressed with mice--it's not like they're growing unrealistic tumors on their backs, geeze!

To: SnowShredder who wrote (126)	8/13/1999 6:47:00 PM
From: Mike McFarland	Read Replies (1) \| Respond to of 319

p.s. I was also under the impression that the promoter and the gene were being delivered seperately, but I may be messed up on my jargon alltogether--three pieces right, the gene, the activation domain (promoter, which is inserted at a different time) and the dimerizer drug. Honesty, the Materials and Methods section of PNAS pp8657 goes right over my head. In fact the only things I recognized were FKBP12 and NF-kB (well I know easy terms, cDNA, CMV, BALB/c) but the rest of it is Greek to me. Say, speaking of the Greeks, is Ariad named for Ariadne? I posted some nonsense over on Yahoo to try and draw somebody out on that, but never got a reply. Anyone?

To: SnowShredder who wrote (126)	8/14/1999 12:55:00 AM
From: Jongmans	Read Replies (1) \| Respond to of 319

Cell Genesys has lentiviral vector +.. cellgenesys.com Martin

To: SnowShredder who wrote (126)	11/12/1999 4:47:00 PM
From: Mike McFarland	Read Replies (1) \| Respond to of 319

Where'd and gang: I really need to get a copy of the recent Avigen PNAS paper and get caught up on that one...it must be the only stock in my Gene Therapy watch lists that is consistently moving upward, geeze this momentum guys are bold...or maybe they've all read the paper and liked it (somehow I do not think that all the Yahoos subscribe to PNAS) Melissa Burton, Hiroyuki Nakai, Peter Colosi, Janet Cunningham, Rachel Mitchell, and Linda Couto. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proceedings of the National Academy of Sciences 1999; 96(22):12725-12730. But for now maybe you can help. As I understand it, contaminating wild type AAV triggers immune response and limits transgene expression and finding the best AAV play is finding the company which can manufacture the purest? What about recombination with wild-type after administration, all AAV is supposed to be disabled, tricky. Do you suppose the Avigen Hemophilia Canine model is immune privledged in some way? It is not at all likely I'll jump on to Avigen for the conclusion of this momo ride, but I can't ignore this stock either...clearly the market likes it, and sometimes the market is right...even if for the wrong reason. I saw a post which said that PBS may be running a show with something about Gene Therapy, if I find the name and time, I'll post. Finally, Wilson apparently spoke at the NatureBiotech Gene Therapy meeting on Monday--regarding EPO. I wonder if he gave an update on the Upenn (Ariad?) EPO monkeys? Any lukers oout there know? Also Carter of TGEN spoke on Tuesday--anybody see that presentation--it should have been about tgen's aav-CF in sinusitis model. I do not know if Avigen had anybody presenting at the meeting, sorry. I'd hoped to see a pulse in Ariad and TGEN this past week on that meeting, TGEN did do a PR, market yawned. I never expect any PR from Ariad or Genovo...last time I checked in August the two were still cooperating. Finally, I sure hope I am not missing out on some great gene therapy discussion over on the listserve, if so please PM me.