Arrakis Therapeutics is named for the desert planet in the legendary “Dune” series of science-fiction novels. It’s an apt name because the Waltham, Mass., startup is trying to achieve a feat once thought to be biotech fantasy: Develop oral medicines — pills — that can target RNA, the messenger-like molecules that turn genetic instructions into proteins.
Scientists at Arrakis have devoted the past two years to building new computer models and rejiggering existing drug-screening tools to create a platform capable of discovering compounds that work against RNA instead of traditional disease-causing proteins.
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I don’t believe these people are out to actually develop “rSMs”. A look at their website and one gets the feel fast that they are really trying to license some bioinformatic software approaches. I’m assuming that’s what the money is really funding. This idea of orally deliverable rSMs is beyond naive wishful thinking. “…serial biotech entrepreneur…” summed it up pretty well.
Unless the compound is able to target specifically the mRNA encoding for Myc (and, I don’t see how), this thing is just vaporware.
Just wishful thinking, and getting easy money from ignorant investors
Omomyc, has a more rational and realistic ground. But, being a peptide it has its fair share of issues concerning delivery
I’m not sure what type of inhibitor they are using, but in theory, specificity should be attainable if using an antisense oligo as the drug, which has Watson Crick complementarity for the mRNA sequence. I read this and assumed they are using siRNAs.
read about this crew in late boston globe business report last night. boston has become such an incubator of biotech with its many universities. the angel investors behind these early stage outfits are almost as impressive as these silicon valley tech start ups. tyvm AF for your update, always enjoy your work
There are two dangers with this “naive” RNA approach. The first is financially, and easy to solve as it’s about own taken financial risks – possible of course that the whole thing is in best case; an over-belief in a goal by a group of over-optimistic dreamers. The second, and significantly more dangerous and possible long-term severe threat is the (human specie) biological one. Stem cell is one thing well researched, where often the main controversy is on an ethical ground – is it “right” and according to our own beliefs to use cells from embryons. However, going this RNA “deep” into the body cells / functions, can cause “effects” these scientists never had thought about. There exist already several real cases where we live after such “outbreaks”. For instance, inter-gene-connect bees, where the “clever” scientists’ dream was to create a less aggressive and more honey productive one. None of these scientists even considered the result could be the total opposite(!) which it did! And they created a so-called “killer” bee. We can also argue that some of today’s deceases are also created by scientists, such as AIDS etc (this comment is not about such discussion though:). I guess what I’m trying to expose is that scientists are very often very “smart” within a very short-/narrow-sighted “pipe-line” (not mention their own personal financial needs), which can make them more dangerous than a cancer cell! To consider a breakthrough of stopping cancer cells is a bold great dream. Yet, the same scientists should also at the same time always considered that the total opposite could happen. Especially, when it comes to the later stage of making human tests and produce a pill! In short, having bold dreams can be good, but it also need to consider all unimaginable (less positive) results. Highly, possible such consideration should be taken by independent scientists (financed by the industry), who have a blue-print worse-scenario-cases outlined when they visit these scientific (over-)optimistic “dreamers”. They, should also be able to stop/shut down any plant if they would see/understand the “dreamers” are fake/scammers and/or in worse case not consider a potential danger the do/cause our specie. In short, big bold dreams are possible good, but should not be solely let in the hands of the dreamers!
To quote from the Wikipedia entry “RNA-targeting small molecule drugs”:
“David Wilson and David Draper were the first to suggest that RNA structures could be targeted by small molecules. They hypothesized that RNA could be “druggable” by targeting the 3D structure in the same way as protein 3D structures are used as drug targets and furthered the idea that targeting RNA could be used to treat diseases.[8] “
Ref 8=
Ratmeyer, L. S; Vinayak, R; Zon, G; Wilson, W. D (1992). “An ethidium analogue that binds with high specificity to a base-bulged duplex from the TAR RNA region of the HIV-I genome”. Journal of Medicinal Chemistry. 35 (5): 966–8. doi:10.1021/jm00083a024. PMID 1548686.
That was 27 years ago!
My educated guess is, although targeting mRNA molecules is feasible, targeting just one specific mRNA is just not possible. At least, by a simple chemical molecule
If they are using an antisense oligonucleotide, why shouldn’t it have selectivity towards a target mRNA over other ones?