Zone in with Zon—Modified mRNA Mania, Clinical and Commercial Potential

Dr. Gerald Zon’s latest “Zone in with Zon” blog post, dated December 2, 2013, and published by TriLink BioTechnologies of San Diego, provides a fascinating discussion of the developing use of modified mRNAs in a wide variety of key applications, including gene therapy, nucleic acid vaccines, and cellular reprogramming, as well as the possibly tremendous commercial potential of modified mRNA technology in these and other areas. Dr. Zon begins by discussing the intellectual simplicity of gene therapy, i.e., to simply replace a broken gene with the DNA of the normal gene and thus ultimately generate the normal version of the missing or altered protein. Unfortunately, it has proven remarkably difficult over three decades of work to achieve this effectively and safely. Dr. Zon attributes this in part to the challenges for cell- or tissue-specific delivery, as well as concern for adverse events generally ascribed to unintended vector integration leading to neoplasias. Nevertheless, there are presently more than 1700 clinical trials of gene therapy taking place around the world. However, as a consequence of the slow progress of DNA-based gene therapy, a number of researchers have recently turned to the study of modified mRNAs that might be used to produce the missing protein directly by translation. This field is called “mRNA therapeutics.” Another application is in the use of mRNAs as cancer vacccines. Here the idea is to use mRNAs coding for tumor-associated antigens to induce specific immune responses to the tumor. Dr. Zon notes that a 2013 review from Novartis Vaccines & Diagnostics, and entitled “RNA: the New Revolution in Nucleic Acid Vaccines,” claims that the “prospects for success are bright.” The reasons for this, Dr.
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