A lot of investors have assumed that the RNA franchise would be cannibalised with the arrival of gene therapy. Not my words but a paraphrase of a comment by Doug Ingram from the Sarepta Q4 2019 Earnings Call. Are global investors right or has the heir apparent to RNA therapeutics got DNA all over its face. Its not a competition mind you but which is the superior technology: gene engineering to replace defective genes with healthy genes or gene modification.
Hottod has pointed out the recent series of setbacks for gene therapy including a clinical trial in haemophilia run by Biomarin where the FDA expressed doubts about the durability of its gene therapy drug. The prospect is raised that re-dosing may not even be possible given the issues of immunogenicity using viral vectors.
We can grasp some of the issues through the article Using CRISP to improve viral vectors for gene therapy, written by Angus Liu and published on FierceBiotech on 3 September 2020. The author points out that that many people have pre-existing immunity against adeno-associated viruses (AAVs) and even if they don't the first inoculation of a vector could induce an immune response.
Simply put, gene therapy even at this early stage has to re-invent itself before it can realise its potential. Meanwhile, RNA therapeutics is overcoming the one real problem that has held it back - an efficient delivery system. Does one sense from the market capitalisation for PYC, that sophisticated investors believe PYC is leading developments in delivery systems for antisense oligonucleotides and the field of RNA therapeutics?
PYC Price at posting:
12.5¢ Sentiment: Buy Disclosure: Held
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