If future is RNA, Future is here
It didn’t take long for CRISPR developments to work their way through the approval process, and ultimately face a likely “passthrough” final stage with the FDA.
October 18, 2023 at 7:00 AM, Intellia reported that they were cleared for a phase III investigation of new drug for rare cardiological disorder, ATTR Amyloidosis. The rare indication has been studied for years, so to get to this stage is an accomplishment in and of it’s own right. However, the candidate known as NTLA-2001 is the first ever in-vivo gene-therapy candidate to hit phase III in the US.
It’s incredible how time flies. Why – it felt like just a couple week’s ago we were lauding Nobel prize winners, Katalin Karikó and Dr. Drew Weissman for their accomplishments in advancing mRNA research.
….Because it was. Make no mistake – it took the original research of Jennifer Doudna to get to where we are today with many astounding therapies breaking the mold of what we thought was possible in modern medicine. The platform though is a somewhat empty canvas. Further developments would show that RNA and mRNA manipulation have unveiled so much more to us than we would have otherwise know [without the CRISPR technology].
What does this mean for other CRISPR driven studies?
It means you can begin to get excited. Earlier in the year, the FDA expanded CIBR by over 25% headcount to deal with the increase of cell and gene therapy studies. They didn’t just do this so that they could approve everything, though. They’re going through their typical due diligence – and potentially have even more scrutiny on gene therapy studies – for the sake of making sure we get things right prior to future submissions. For such a rare indication like ATTR though, with now so much robust data, it’s a strong sign that it’s made it this far.
What’s next?
I need to let my non-scientist people know, you won’t see another approval for another couple of years, but that can still be considered rather quick. The good thing is, it’s at this stage and there are plenty other groups like Pfizer and IONIS battling to get out strong data. Intellia is partnered with Regeneron for this particular candidate, but there are several other independent Gene therapy compounds they presume will be equally as successful.
In the meantime, those searching for answers to the dip in the market, look no further than the clear future of medicine.
Intellia gets FDA OK to trial in vivo CRISPR therapy in the US (fiercebiotech.com)
