Category: Gene Therapy
On September 23, 2019, the Patent Trial and Appeal Board (“PTAB”) issued a decision dismissing Sigma-Aldrich’s interference petition related to the revolutionary CRISPR-Cas9 biotechnology. The claims at issue in Sigma-Aldrich’s petition were directed to methods of genetically modifying a eukaryotic cell using a CRISPR-Cas9 system. The petition was unusual because it sought an interference although none of Sigma-Aldrich’s claims had yet been allowed. On this basis, PTAB denied the petition as premature (and on other procedural grounds) and dismissed it without prejudice to refiling.
On July 19, 2019, Sigma-Aldrich filed a petition with the Director of the U.S. Patent and Trademark Office (“USPTO”) and the Chief Administrative Patent Judge (“CAPJ”) of the Patent Trial and Appeal Board (“PTAB”) seeking an interference between itself and the Regents of the University of California (“UC”) that would parallel an interference that was recently declared between UC and the Broad Institute (“Broad”). The claims at issue are directed to methods of genetically modifying a eukaryotic cell using a CRISPR-Cas9 system. The petition is unusual because it seeks to provoke an interference although Sigma-Aldrich’s pending claims have not been allowed, contrary to 37 C.F.R. § 41.102 and MPEP § 2303. Sigma-Aldrich argues that the circumstances here are extraordinary and warrant an exception to the rule.
Today FDA approved the first-ever “small interfering RNA” (siRNA) product, marking a significant milestone in the story of RNA interference (RNAi) technology and clearing the way for a new type of therapeutic. Alnylam® secured approval and Orphan Drug Designation for its siRNA product Onpattro (patisiran), a therapy for the rare hereditary disease transthyretin-mediated amyloidosis in adult patients. The disease is caused by mutations in a protein called “transthyretin” which leads to symptoms of neuropathic pain, loss of sensation in the hands and feet, and wheel-chair confinement. In Alnylam’s Phase III clinical trial, Onpattro improved multiple clinical manifestations of the disease and demonstrated safe administration of a siRNA product.
On December 19, 2017, FDA approved Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl), the United States’ first gene therapy approved to treat an inherited genetic disease. This approval follows that of Novartis’ Kymriah (tisagenlecleucel) and Gilead Sciences’ Yescarta (axicabtagene ciloleucel), both gene therapies approved earlier this year to treat certain forms of cancer. Gene therapy has arrived, and although the genetic material central to these new treatments is not expressly listed in the statutory definition of “biological product,” FDA is regulating these products as biologics, giving them twelve-year non-patent exclusivity.