Jacob Kimmel: evaluation

Transcript context

…How big will the payload have to be? How many transcription factors? I think it's just a countable number. Some of those that we've found today that have efficacy are somewhere between one and five. That's a small enough number that you can encapsulate it in current mRNA medicines. Already in the clinic today, there are medicines that deliver many different genes as RNA. There are medicines where, for instance, it's a vaccine as a combination of flu and COVID proteins, and they're delivering 20 different unique transcripts all at the same time. When you think about that already as a medicine that's being injected into people in trials, the idea of delivering just a few transcription factors is seemingly quotidian. Thankfully, I don't think we'll be limited by the size of the payloads that one can deliver. One other really cool thing about transcription factors is that the endogenous biology is very favorable for drug development. The expression level of transcription factors in your genome relative to other genes is incredibly low. If you just look at the rank-ordered list of what are the most frequently expressed genes in the genome by the count of how many mRNAs are in the cell, transcription factors are near the bottom. That means you don't actually need to get that many copies of a transcription factor into a cell in order to have benefits. What we've seen so far, and what I imagine will continue to play out, is that even fairly low doses of these medicines, which are well within the realm of what folks have been taking for more than a decade. They are able to induce really strong efficacy. We're hopeful that not only will the actual size of the payload in terms of number of base pairs not be limiting, but the dose shouldn't be limiting either. Would it have to be a chronic treatment, or could it just be a one-time dose?…

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