transcription factors as drugs. same delivery problems and then some new problems, but it is still a good idea. why?
most diseases are caused by small changes in protein expression. we can't solve a lot of diseases because we see a 10% upregulation in 40 proteins, a 30% upregulation in another 30% proteins and 20% deregulation in another 5.
Users in the replies call transcription factors an exciting new drug idea for complex diseases because such concepts are rare in biopharma, while noting immunogenicity and PK/PD control challenges.
don't forget immunogenicity. controlling PK/PD here seems very hard. still exciting new idea and those don't come up very often in biopharma. Sangamo and a few others once got around this hoop but never exactly on the TF-or-bust wavelength
personally i think there are too few liver diseases left to battle with nucleic acids (ask NTLA), drugging ALD would be a huge win, no doubt a 2B+ drug, but PK/exposure here may be tricky. if you drug with a mRNA encoding the TF you might have a better chance
eventually getting tissue-specific delivery could result in tx for tough diseases like IPF where there is no 'master fibrosis enzyme'. you have a huge ensemble to adjust: think like a music producer smoothly adjusting several EQ bands vs. the drug which annihilates one band
TFs have always been tantalizing & we know they're implicated in disease via genetics. generally no one has tried to drug TFs because of cellular delivery problems. for the liver, moderna et al solved this. that's why all the CRISPR companies target liver diseases (Wilson etc)
TFs have always been tantalizing & we know they're implicated in disease via genetics. generally no one has tried to drug TFs because of cellular delivery problems. for the liver, moderna et al solved this. that's why all the CRISPR companies target liver diseases (Wilson etc)
transcription factors as drugs. same delivery problems and then some new problems, but it is still a good idea. why?
most diseases are caused by small changes in protein expression. we can't solve a lot of diseases because we see a 10% upregulation in 40 proteins, a 30% upregulation in another 30% proteins and 20% deregulation in another 5.
personally i think there are too few liver diseases left to battle with nucleic acids (ask NTLA), drugging ALD would be a huge win, no doubt a 2B+ drug, but PK/exposure here may be tricky. if you drug with a mRNA encoding the TF you might have a better chance
eventually getting tissue-specific delivery could result in tx for tough diseases like IPF where there is no 'master fibrosis enzyme'. you have a huge ensemble to adjust: think like a music producer smoothly adjusting several EQ bands vs. the drug which annihilates one band
@MartinShkreli Are AI models being designed to break through the various protein expression data
@MartinShkreli
@MartinShkreli AAV delivery with epicrispr for FSHD
@MartinShkreli ALD will be treated via engineered microbes
transcription factors as drugs. same delivery problems and then some new problems, but it is still a good idea. why?
most diseases are caused by small changes in protein expression. we can't solve a lot of diseases because we see a 10% upregulation in 40 proteins, a 30% upregulation in another 30% proteins and 20% deregulation in another 5.