Pharmaceutical

Intellia Therapeutics (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR technology, announced financial results and operational progress for the fourth quarter and full year of 2017. As quoted in the press release: Using our proprietary lipid nanoparticle (LNP) delivery system, Intellia has achieved near-complete knockout editing of the transthyretin amyloidosis …

Intellia Therapeutics (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR technology, announced financial results and operational progress for the fourth quarter and full year of 2017.

As quoted in the press release:

Using our proprietary lipid nanoparticle (LNP) delivery system, Intellia has achieved near-complete knockout editing of the transthyretin amyloidosis target gene (TTR) in rodents and most recently shared data on the progress of non-human primate (NHP) studies. In the fourth quarter, our NHP studies continued, yielding higher levels of editing as the Company improved the LNP formulation and dosing regimens, including repeated doses. Ongoing efforts in the NHP studies focus on consistently achieving a therapeutically relevant level of editing for the TTR gene as determined by transthyretin level reductions and we continue to observe and, as in prior animal studies, gather long-term durability data.

Click here to read the full press release.

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