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Message: Re: What else can RVX treat that we don’t know about yet?

"We just have not seen market hype in epigenetics and we have and continue to see it in genetic editing."

Theoretically, gene editing targets precise location(s) in the genome to alter the nucleotide sequence. And non-gene editing gene therapies typically facilitate the expression of a single known gene product. Of course, there are worries about long term effects, concerns about permanance or irreversibility, and potential problems in the fidelity of gene editing leading to off-target effect. However, in general gene editing and gene therapies are much easier concepts to grasp and less of a concern about unexpected or unintended or off-target effect. Not completely free of these concerns, but mostly. 

Epigenetic therapies, on the other hand, have a much higher hurdle to clear. Epigenetics is a less understood concept and the research is not as advanced. In general, epigenetics is part of every process in biology. From cell/tissue differentiation during development in utero and postnatal growth, to masculinization/feminization of cells/tissues during development, to puberty initiation, to aging and to several different disease processes. Not an exhaustive list by far; just examples of how pervasive and essential epigenetics is to multiple, if not all, facets of biology. Epigenetics goes beyond acetylating the lysines of histones and being read by bromodomains of BET proteins. Methylation, demethylation, acetylation and deacetylation are all some of the more commonly discussed epigenetic mechanisms, but that list of epigenetic modifications is not exhaustive. Epigenetic modifications are very dynamic processes that happen in both normal healthy, and abnormal unhealthy states. 

Scientific/medical professionals, regulatory agencies, investors, etc are likely to approach and accept epigenetic therapies, including BET inhibition, with both more caution and more skepticism as compared to more "conventional" therapies. I'm not trying to be the Boogeyman or anything, just the reality of drug development and fear of the unknown and lack of understanding of novel mechanisms of action. Aside from BETonMACE, epigenetics drugs have been limited to oncology (someone correct me if I'm wrong). So I expect apabetalone and BETonMACE to be scrutinized hardcore, both fairly and unfairly. Being first in class has its advantages and disadvantages.

BearDownAZ

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