Firm will develop Pseudomonas therapeutics and continue to exploit existing AAV vector assets.

Targeted Genetics is changing its name to AmpliPhi Biosciences, which it says more accurately reflects its core bacteriophage technology, which is being exploited to develop antibacterials. The technology was acquired through Targeted Genetics’s takeover of U.K.-based Biocontrol, earlier this year. Biocontrol’s lead product, BioPhage-PA, has completed Phase I/II evaluation as a treatment for chronic ear infections caused by Pseudomonas aeruginosa. A second candidate, BioPhage-PR, is being developed for the treatment of Pseudomonas lung infections in cystic fibrosis patients. A grant has been secured from the Cystic Fibrosis Foundation of America to support clinical trials of BioPhage-PR.

AmpliPhi says it aims to progress both these lead bacteriophage programs and also evaluate the potential for combining bacteriophage-based therapeutics with conventional antibiotics as multitarget, indication-specific therapeutics.

“AmpliPhi Biosciences is the first group to advance bacteriophage for human therapeutic applications through a Phase I/II clinical trial to modern regulatory standards,” comments Jeremy Curnock Cook, board chairman. “We believe the company’s expertise will prove valuable in seeking to provide reliable, safe, efficacious, and scalable solutions for controlling antibiotic-resistant bacterial infections.”

The firm also hopes to continue capitalizing on its existing AAV vector-based gene therapy assets that include royalty-bearing product licenses held by Amsterdam Molecular Therapeutics (AMT), Celladon, and Genzyme.

Targeted Genetics sold manufacturing technologies and other adeno-associated viral (AAV) vector technology to Genzyme as part of an asset purchase agreement signed in September 2009. Genzyme agreed to pay up to $7 million for the assets and the successful transfer of the technology and also agreed to pay a portion of sublicensing revenue received during a specified timeframe and royalties on net product sales of products if it successfully commercializes products utilizing the acquired Targeted Genetics technology.

Targeted Genetics separately entered into a nonexclusive patent license agreement with AMT in 2006, which covers the latter’s lipoprotein lipase deficiency therapeutic, Glybera®, which is currently undergoing regulatory review by the EMA. The license agreement provides for AMT to pay royalties on net product sales, milestone payments upon the achievement of certain product development targets, and annual maintenance payments. Celladon is exploiting the AAV vector technology for the development of Mydicar®, a clinical-stage genetically targeted enzyme-replacement therapy for advanced heart failure.

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