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Biotech / Medical : VD's Model Portfolio & Discussion Thread -- Ignore unavailable to you. Want to Upgrade?

To: Mike McFarland who wrote (5619)	9/15/1998 1:12:00 PM
From: Pseudo Biologist	Read Replies (2) \| Respond to of 9719

Mike, two companies I can recall doing work on AAV are Avigen (AVGN) and Cell Genesys (CEGE). This is from CEGE's most recent 10K: <<During 1997, Cell Genesys acquired Somatix Therapy Corporation ("Somatix"),establishing a leadership position in gene therapy. Product research and development programs of the two companies were highly complementary, with Cell Genesys focused on the treatment of AIDS and cancer, and Somatix focused on cancer, central nervous system diseases and other disorders. Technology research and development programs were also highly complementary, with synergies in the retroviral and adenoviral vector programs of both companies and with new adeno-associated viral and lentiviral vector programs contributed by Somatix. Integration of the two companies was completed by the end of 1997, having prioritized the most promising programs, significantly reducing overall expenses of the combined businesses. With the combined portfolio of product opportunities, technologies and intellectual property, Cell Genesys believes it has substantially increased its ability to attract new corporate collaborations, to advance product candidates through development and commercialization and also has expanded its opportunities to license additional therapeutic genes from other companies and research institutes needed to create new gene therapies.>>

To: Mike McFarland who wrote (5619)	9/15/1998 11:22:00 PM
From: poodle	Respond to of 9719

Mike, I would be careful with AAV. Idea was very good because of AAV specific integration site, but vector has low capacity. If my memory is correct, some article described system of 3 different AAV constructs used simultaniously to overcome this problem. Unfortunately, that's not the only problem. This spring I talked to gene therapy experts about different vectors, mainly adenoviruses, AAV and retroviruses. I have been told that AAV demonstrated low efficiency in vivo. More than, expression of the inserted constructs in vivo declined sharply. Retroviruses looked better for them, but very far from medical appl. either. I had been told also that there are many psychological problems yet. Gene therapy, in it's "classical" or broader meaning, will be the most serious advantage of medicine, if not the only real medicine at all, but effective methods for DNA transfer should be developed first.

To: Mike McFarland who wrote (5619)	9/16/1998 3:32:00 AM
From: SnowShredder	Respond to of 9719

Mike, Here are the US gene therapy companies that use AAV that I know of: Somatix, Targeted Genetics, Avigen, & Applied Immune Sciences. In Europe: Intro Gene & MediGene. In my opinion AAV vectors are not the best gene delivery vehicle available. The reason why people started to use AAV was because of their site specific integration, but in the vector version AAV does not have specific integration...the integration is random (the wildtype has specific). Right now if you are looking for integration I would look @ the MLV based retroviral vectors or even the lentiviral vectors. If you need transient expression look toward the adenoviral vectors or non viral vectors. Each vector system has its pros & cons...if you here of an integrating Ad vector...then let me know. If you have any specific ?'s I'm sure that I can find an answer for you or @ least point you in the right direction (if I don't know it)...I work in a gene therapy lab. Right now I'm keeping my $ out of gene therapy companies, but some day I'm sure that I will change my mind. Best of Luck, Where'd He Go?