In the 60-day comment period after the National Institutes of Health'sOffice of Technology Transfer (OTT) posted a notice in the FederalRegister last July 30, announcing its intention to grant an exclusive licenseon an adeno-associated virus vector (AAV) to Targeted Genetics Corp. ofSeattle, OTT has received at least two comments from other companiesasking to be cut in on the deal. OTT is reviewing these demurrers, theagency's licensing specialist, Steven Ferguson, told BioWorld, and shouldreach a decision in several weeks.

The vector's principal inventor is Barrie Carter, who left the NIH's Instituteof Diabetes, Digestive and Kidney Diseases last year to become executivevice president and director of research and development at TargetedGenetics. Two of Carter's other AAVV inventions were proffered toattendees at last week's annual meeting of the Licensing ExecutivesSociety on a non-exclusive basis.

Targeted Genetics stands to obtain exclusivity to the invention describedin its pending patent as "Modified Adeno-Associated Virus Vector Capableof Expression From a Novel Promoter." The company's licensure rightswould be limited to gene therapy for the treatment of cystic fibrosis (CF).

"The invention's main discovery," Ferguson said, "is that a truncated CFgene, packaged with a novel promoter is small enough to fit in the AAVvector's cargo bay." He added that the CF application is "a very discretefield of use," assuming other applications for the promoter exist.

Jon Case, director of corporate development at Targeted Genetics, toldBioWorld that the AAV p5 promoter, plus its inverted terminal repeat (ITR)form a 263-base-pair cassette, which mediated efficient expression in aCF bronchial epithelial cell line. Moreover, the ITR itself can mediate geneexpression.

-- David N. Leff Science Editor

(c) 1997 American Health Consultants. All rights reserved.