Targeted Genetics Corp. announced Wednesday that it hassigned a collaborative research agreement with the JohnsHopkins University School of Medicine to develop adeno-associated virus (AAV) vectors for use in gene therapy.
Targeted Genetics (TGC) of Seattle will have the first optionunder the agreement to negotiate an exclusive worldwidelicense to any inventions resulting from the collaboration. Inreturn, TGC will pay royalties to Johns Hopkins.
TGC, a gene therapy R&D company spun off from ImmunexCorp. (NASDAQ:IMNX) in 1989, is developing integrated genetherapies for treating AIDS, cystic fibrosis, cytomegalovirus andcancer.
The AAV vectors that TGC is now developing with JohnsHopkins are among a small number of recombinant virusvector systems that have been shown to have utility as in vivogene transfer therapy. TGC is hoping to use these vectorsparticularly for treating cystic fibrosis, where gene therapyinvolves delivering the gene for the missing protein (cysticfibrosis transmembrane regulator protein) directly into thelungs of affected people.
And TGC is already in Phase I/II trials with an ex vivo genetransfer approach to modifying the CD8 component of T cells ofHIV-infected patients.
TGC was the first commercial company to receive permissionfrom the National Institutes of Health's Recombinant DNAAdvisory Committee (RAC) to conduct this gene transfer trial,which combines a marker gene and a suicide gene that can beused to eliminate the altered cells if they present anyproblems. -- Jennifer Van Brunt
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