Another section of this newsletter includes an item that discusses how supporters of the human genome project advocate probing yet further “beneath the skin” to demonstrate that the massive investment in genome-wide association studies and the coalescence of these findings into polygenic scores will open up new avenues for prevention. As described in the January 2022 issue of the journal Nature Biotechnology, a goal of advancing science and technology should be to solve global health challenges by developing new therapies, treatments, and preventive measures that directly could contribute to the enhancement of the collective well-being of societies. Human genome editing innovations should endeavor to move in this direction, and any that did not, or that were potentially harmful, should not be allowed to continue. Technologies that should not be allowed at all include those attempting heritable germline human genome editing because of the risks and safety concerns surrounding current technology and also because of the new and possibly unique ethical challenges they would introduce.
Instead, somatic gene editing therapies, by contrast, do not present the same risks and challenges, promising to provide treatments for a wide range of diseases and conditions, including cancers, blindness, and hemophilia. The era of genetic therapies, both gene-editing treatments and gene therapies, several of which currently are on the market, has arrived for rare disease. As more of these therapies come online, however, it is time to explore how current business models based on patents and restrictive licensing limit access to treatments. It also is time to explore whether there are other ways in which patents can still reward innovators and protect investments while ensuring that the widest number of patients who need these treatments can receive them. An example of a strategy possibly worth pursuing would be to reduce the cost of premium-priced gene therapies through the use of patent pools, which have been applied successfully in the technology and electronics world.