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In a first for genome editing, Intellia Therapeutics reports positive early data on in vivo CRISPR therapy
From Stat+: link
I'm finding myself wanting to spend more time reading about biotech as I believe it's a huge opportunity, so I just subscribed to Stat+ on a monthly plan, and we'll see how much it can help shape my investment point of view.
In a major milestone for the still-young field of genome editing, Intellia Therapeutics said Saturday that the first six patients to receive a CRISPR-based treatment for a genetic nerve disorder have safely had the DNA inside their liver cells edited.
Preliminary results from the study — the first to show that CRISPR-based gene editing can be delivered systemically and performed in vivo, or inside the body — found that the treatment reduced levels of a disease-causing protein by an average of 87% in the higher dose cohort with only mild side effects. The encouraging interim Phase 1 results, presented at a conference on Saturday, were published simultaneously in the New England Journal of Medicine.
.. The Intellia therapy, called NTLA-2001, is being co-developed by Regeneron Pharmaceuticals.
The patients, enrolled in an ongoing clinical trial in the U.K. and New Zealand, suffer from a form of transthyretin amyloidosis — a progressive, fatal disease caused by a mutation to the TTR gene. The condition leads to the liver producing misshapen, unstable versions of the protein transthyretin that accumulate into toxic plaques in the peripheral nervous system. NTLA-2001 successfully turned off that problematic gene, leading to drastic reductions in levels of circulating transthyretin.
Although Intellia didn’t directly measure the amount of editing in their clinical trial participants — which would have involved an invasive liver biopsy — there were other signs that the lipid nanoparticles used to deliver the therapy, known as LNPs, performed their job well. In the three patients treated with the lowest dose of NTLA-2001, CRISPR successfully knocked down the levels of TTR by more than 50%. In the three patients who received the higher dose, TTR levels dropped by an average of 87%.
Intellia is the first of the so-called “CRISPR companies” to present clinical data showing it’s possible to precisely alter the DNA of particular cells within the body to treat genetic disease with a one-time intravenous infusion of CRISPR.
Other companies developing treatments based on CRISPR or similar genome-editing technologies either perform the work on cells in a lab and then re-administer them to patients, or inject them directly into the target tissue.
“We’ve been yearning for space flight and while we’ve had our first space walk and our first moon landing, and now it feels like we’re going to Mars,” said genetic engineer Fyodor Urnov, of the University of California, Berkeley, who was not involved in the research. “The fact that Intellia solved the CRISPR-specific challenges of what it takes to inject a human being with a lipid nanoparticle carrying an mRNA encoding Cas9, is in of itself a landmark.”
This company's stock is pretty volatile and interesting to trade.
Here is the 1 month chart:
The stock sold down to $75 on June 17th, after a double top at $89 on June 14 and 15, so a pretty drastic sell down in only two days, or about 16 percent.
At the time, the only news I could see was that a Director sold about 200k shares at $87.04 through an automated selling program, reducing his shares from about 765k to 565k, or about 25% of his holdings.
form 4 link: here
I thought at the time the market over-reacted, so I added 50% to my position at around $78. I would have bought more, but I'm still uncertain over how best to play the CRISPR space - now owning NTLA, CRSP and BEAM, so I didn't buy more. In hindsight, I wish I would have doubled my position.
Looking forward, it will be interesting to see how the market reacts next week.
Clearly $89 is a resistance point, and if we can break it on volume, then I will add another tranche (maybe 30%) to the position.
If it sells off (ie buy the rumor, sell the news), then I'll hang tight for the next catalyst.
I actually think it will pop - or that's what I'm hoping.
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