Summary
The first in vivo clinical trial results using in vivo CRISPR gene editing technology were reported yesterday (6/26/21) by Intellia (NASDAQ:NTLA) and its partner Regeneron (NASDAQ:REGN).
The trial is for a rare disease called transthyretin amyloidosis that is treated by decreasing levels of transthyretin (TTR), the causative agent.
The results showed a dose-dependent reduction in serum levels of TTR after a single dose of NTLA-2001, NTLA’s CRISPR therapy targeting inactivation of the TTR gene.
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Yesterday, at the annual meeting of the Peripheral Nerve Society (PNS) annual meeting, Intellia (NTLA) reported the first clinical results for in vivo delivery of a CRISPR technology. The CRISPR therapeutic candidate, called NTLA-2001, targets the causative protein, transtheyretin (TTR), or a rare disease called rare disease called transthyretin amyloidosis (ATTR). The results were impressive.
Transthyretin amyloidosis (ATTR) is a progressive and fatal disease that results from the build-up of a misfolded form of the transthyretin (TTR) protein, leading to peripheral neuropathy and cardiomyopathy. It is known that treatments that reduce the levels of TTR in serum can effectively treat the disease (See e.g., Adams et al, NEJM (2018) Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis). The liver is the primary source of circulating tetrameric transthyretin protein (See Adams et al., 2018). So it is a good candidate disease for a proof-of-concept study NTLA-2001, which targets liver cells using this very hyped gene editing technology called CRISPR.
The results of the first two dosing levels of NTLA-2001, appear to establish proof of concept for NTLA-2001 and NTLA’s in vivo CRISPR technology for knocking down a protein produced by liver cells. The results, which were presented at the PNS biomedical meeting, an accompanying investor event (See presentation HERE), and an accompanying New England Journal of Medicine article (Gillmore et al. 2021 “CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis”), showed that NTLA-2001 was able to reduce levels of TTR in serum in a dose-dependent manner and to an extent that should be clinically meaningful. The 80%+ reduction in TTR levels measured at 28 days after administration of the highest dose tested (FIG. 1) compares well with the approx 75% reduction seen with RNAi technology Partisan (ONPATTRO®) (Adams et al. 2018) marketed by Alnylam (See HERE). Plus unlike the RNAi therapy,
FIG. 1
This is an exciting proof of concept for NTLA-2001 and especially for in vivo CRISPR technology. Next up for NTLA-2001 will be a report-out later this year of more data from this Phase 1 study (See HERE). Investors will be looking at whether the reduction in TTR serum levels is sustained over long periods and whether further reduction can be achieved. This is important data when comparing NTLA-2001 to approved RNAi therapeutics for this disorder, which require re-administration every 3 week (See Onpattro label HERE).
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This article may be updated from time to time
Article History:
6/27/21 Created
6/28/21 Updated format
This article is NOT investment, tax, or legal advice. Please use this as a starting point for your detailed diligence and consult professional advisers before making investment decisions.
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