Gene Therapy and Patents – Upcoming Issues and Concern

Joseph Guthrie, KLJ Staff Editor

In the 1990’s, gene therapy was viewed as the next major step forward in medical treatment, with the potential to permanently cure a wide variety of genetic disorders.  Despite early successful trials, gene therapy suffered a major setback in 1999 following the death of Jesse Gelsinger, an otherwise healthy 18-year-old suffering from a manageable genetic disorder, during a clinical trial.  Considering the technology potentially unsafe, the FDA suspended many ongoing trials and researchers largely abandoned the field.

Fifteen years later, gene therapy is back in a huge way, having been used in human trials to successfully cure HIV, cancer, and various rare genetic disorders.  Since the beginning of 2013, investors have poured nearly $700 million into companies focused on developing gene therapy treatments. Like in the pharmaceutical industry, development and testing of these kinds of treatments involves huge amounts of money, and these companies will eventually seek patents to protect their discoveries.  What remains to be seen is whether these treatments can be patented, and if so, whether these patents will spur innovation or merely stifle it. In its most common form, gene therapy involves the use of viruses to deliver DNA to a patient’s cells, replacing mutated, dysfunctional genes with functional ones.  In order to develop these therapies, researchers need to first identify the genes responsible for the disease, find a suitable virus to deliver a healthy gene, and finally tailor that virus to specifically target the patient’s affected cells. Biotech firms can try to patent these therapies in one of two ways: by patenting the method of delivering the DNA to the patient or by patenting the genes or modified viruses themselves.  Two recent Supreme Court decisions, however, create a great deal of uncertainty about whether either of these approaches will work. In Mayo v. Prometheus, the Court invalidated a wide swath of diagnostic method patents on the ground that they involved “well-understood, routine, conventional activity previously engaged in by researchers in the field” and risked “tying up the use of the underlying natural laws”.   Recognizing the far-reaching effects this decision might have, the Federal Circuit in Classen Immunotherapies v. Biogen IDEC clarified that method claims could overcome this obstacle by including an additional treatment step. These cases matter for gene therapy patents because genetic disorders are not always identical across all patients.  Every treatment will require genetic diagnostics to tailor each therapy to each specific patient.  Thanks to Classen, however, the diagnostic aspect of gene therapy method claims seem to be safe as long as they are coupled with the treatment itself. Patentability of the DNA and viruses themselves presents a more serious problem, however.  In Association for Molecular Pathology v. Myriad Genetics, the Supreme Court upset nearly two decades of patent practice by holding that isolated DNA sequences were unpatentable subject matter.  At the same time, the Court found that cDNA, a mirror image of naturally occurring, edited DNA, was patentable since, unlike isolated DNA, it was created in the laboratory and not simply extracted from a human subject. Myriad has important implications for gene therapy patents because many developing gene therapy treatments require the use of naturally occurring DNA.  While Myriad allows patents on cDNA sequences, they are not entirely useful in gene therapy since they are copies of an edited form of DNA found outside the cell nucleus.  For gene therapy to work, it needs to use natural, unedited DNA sequences to overwrite the faulty sequences in the patients’ chromosomes.  Only claims covering the lab-created, genetically engineered viruses are likely to survive post-Myriad. In the end, the gene therapy industry might benefit from limiting the scope of these patents, but this level of uncertainty about it isn’t going to help.