Pharmaceutical Company Sued For Patent Infringement In Gene Therapy Drug

A suit was filed Friday in the District of Delaware by plaintiffs Genzyme Corporation and Aventis, Inc. against defendants Novartis Gene Therapies, Inc., Novartis Pharmaceuticals Corporation, and Novartis AG (collectively, Novartis). The plaintiffs are alleging that the defendants infringed on five separate patents when they engaged in the “unauthorized manufacture, use, and sale of recombinant adeno-associated virus vectors(“rAAV vectors”) for their gene therapy drug Zolgensma.” Genzyme is a wholly-owned subsidiary of Aventis.

The violated patents involve the use of rAAV vectors in gene therapy, said to modify a person’s genes in order to treat genetic diseases. rAAV vectors are used in gene therapy to package and deliver the copies of a gene (often called transgenes) to the cells. Oftentimes, the transgenes are not expressed or take a while to be expressed because of the difficulty in attaining sufficient levels of the transgene.

A challenge with transgene expression is the fact that the incoming DNA is single-stranded and must be converted into double-stranded, which is described as a “key rate-limiting step.” The aforementioned patents protect a method by which “rAAV vector DNA can be engineered to self-adopt a double-stranded conformation upon delivery to a target cell.” This method effectively eliminates the conversion from single to double-stranded DNA step that often inhibits the transgene expression. The patents refer to this technology as intrastrand base pairing. The patents-in-suit are described by the plaintiffs as representing “a significant leap forward that allows functional gene therapy.”

Zolgensma is a gene therapy product “intended to treat certain children less than two years of age with SMA [spinal muscular atrophy].” After the defendants obtained FDA approval to market their gene therapy product, Zolgensma, they began to sell and promote the product in the United States. Zolgensma infringes on the patents-in-suit by using self-complimentary rAAV vectors which are both covered by and based on the innovations of the violated patents.

The suit cites five separate counts of patent infringement, leading the plaintiffs to seek favorable judgement on all counts, compensatory damages, litigation fees, and any other relief deemed proper by the court.The plaintiffs are represented by Wilks Law Firm.