Pharmaceutical Business review

Genzyme gains rights to Asklepios’ duplex gene therapy vector technology

Used by Genzyme for its internal product development and external sublicensing, the technology allows a gene therapy Adeno-Associated Viral vector (AAV) to package and deliver more potent therapeutic DNA cargo to a patient’s cells.

An intracellular-synthesis step necessary for converting a single-stranded DNA genome into a double-stranded genome can be by passed with the Duplex vectors resulting in quick onset and increased levels of gene expression.

Further, the agreement provides AskBio the rights to Genzyme’s Intra-Strand Base-Pairing technology that supports DNA double-stranded genome packaging within an AAV-based gene therapy vector.

AskBio vice president Jade Samulski said, "This cross-license with Genzyme represents our ninth license of the Duplex vectors and further validates AskBio’s platform of vector technologies and their significance and potential for future patient benefit."

Genzyme Gene Therapy R&D head Sam Wadsworth said, "We believe our efforts will be enhanced through AskBio’s technology as we continue to pursue targeted solutions to address unmet medical needs."