Toniv,

No, I don't think CRISPR has been discussed on here before. Although CRISPR is a powerful technology for editing the sequence of genes (either for creating/fixing a mutation, or inactivating a gene) or for gene siliencing, it is absolutely no threat for BET inhibitors. Apples and oranges. Or maybe more like apples and meatloaf. Very different.

CRISPR is amenable to manipulating cells ex-vivo, not in vivo. For example, cultured immortalized cell lines or primary cell cultures could be manipulated via CRISPR in the lab for various applications, including delivering modified cells back to the patient. Or CRISPR allows for very efficient manipulation of embryonic stem cells for generation of genetically modified organisms (like mutant mice, etc). However, one huge hurdle that limits CRISPR to ex vivo applications right now is that the CRISPR system necessitates getting the CRISPR/Cas9 ribonucleoproteins into the cells using techniques such as electroporation. This is much different than the using an orally available, cell-permeable small molecule like BET inhibitors and other pharmaceuticals.

Could CRISPR be used by RVX to accelerate progress? Of course! Basic research will continue to benefit enormousely from the applications of CRISPR to elucidate biological mechanisms in a faster, less expensive and more efficient manner. However, IMO there is absolutely no worry about competitors with deep pockets outpacing RVX or Zenith for MACE or their other indications by using CRISPR. If anything, RVX and Zenith may be using or will use in the future CRISPR technology in house themselves to better understand and characterize the mechanisms by which their BET inhibitors are eliciting their biological effects.

Best regards,

BearDownAZ