RNA therapeutics have come of age, but their ongoing success is...

RNA therapeutics have come of age, but their ongoing success is impeded by inefficient or toxic delivery inside cells. PYC believes that its cell-penetrating peptide delivery platform solves this problem @hottod

There have been stunning successes but also spectacular flops in the application of mRNA. I was re-looking at a post back in August 2020 where I asked the question - in relation to neurodegenerative diseases - what could PYC do differently. I entirely agree with Hottod in pointing out the delivery problem but recognise at the same time that diseases like Parkinson's and Alzheimer's and possibly even Huntington’s disease re: Wave and Roche clinical failures, may not be one disease but several or multiple disease subtypes lumped together under one disease classification.

Reference was made to an article Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era; published Wiley Online Library in August 2020, authors include Sue Fletcher. From Post #: 46746791;

The authors highlight the problem of the heterogeneity in PD and the difficulty of evaluating treatments where patients have different disease profiles including different genetic-molecular pathogenesis. Not all PD patients are the same and response to treatments will vary. There is increasing recognition of the need to stratify patients into homogeneous groups based upon genes and/or molecular pathways and underlying pathophysiology.

We may find that PYC will stratify patients into subtypes in order to find targets. If neurodegenerative diseases have different pathways or problem protein expressions one size does not fit all. I think that our CSO is fully aware of the need for a novel approach to tackling neurodegenerative disease.