The rule of three, page-7

One question which troubled me was whether RNA therapeutics was going to be superseded by gene therapy. For instance, is gene therapy more sophisticated and more powerful than RNA therapeutics? Sarepta while continuing with their RNA programs appear to be highlighting and creating a focus upon their gene therapy development programs. Is this a portend of the future?

The answer appears to be there is room for both approaches and each type of therapy will have its own area of competitive advantage. RNA therapeutics has been held back by the delivery problem which may also apply to gene therapy for certain targets but with additional complications. For instance, gene therapy utilises viral vectors but consider the problem of re-dosing once the body gains immunity to the virus. Further, there are delivery challenges for gene therapy in some targets such as penetration into the back of the eye; and then there are the long term concerns about the safety of gene therapy in terms of the risk of integration into the genome.

Sometimes the disease can only be corrected by RNA modification rather than adding a gene. Splice switching oligonucleotides are perfectly designed for a significant number of rare and ageing diseases. Lastly but not least the answer to many rare and ageing diseases will lie in the combination of RNA and gene therapy approaches.