Intellia Therapeutics and Regeneron Pharmaceuticals have released the latest positive interim data from an ongoing Phase 1 clinical study of NTLA-2001 (NCT04601051), which aims to treat transthyretin (ATTR) amyloidosis with a single-dose. The interim data released includes patients treated at Richmond Pharmacology’s London unit.

The Phase 1 trial assessed the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and the optimal biologically active dose. This commenced in 2020 with the first patient dosed at Richmond Pharmacology – 15 participants have now received the treatment at two additional sites across the world.

The results demonstrate that the precision gene editing therapy has successfully reduced the production of misfolded TTR protein which builds up in the body. At all four dose levels, NTLA-2001 was generally well tolerated. Most adverse events were mild in severity with 73% (n = 11) of patients reporting a maximal adverse event severity of Grade 1. The most frequent adverse events included headache, infusion-related reactions, back pain, rash, and nausea. There were no clinically significant liver findings observed.

Part 2 of this study will build on these findings and assesses the safety and tolerability of NTLA-2001 in patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN) and Patients with Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM).


About NTLA-2001

NTLA-2001 is the first CRISPR/Cas9-based therapy candidate to be administered systemically for precision editing of a gene in humans. It is designed to inactivate the TTR gene in liver cells to reduce the production of misfolded TTR protein, which accumulates in tissues throughout the body and causes the debilitating and often fatal complications of ATTR amyloidosis.

For more information about the interim results click here.

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