Transthyretin amyloidosis, also known as ATTR amyloidosis, is a life-threatening condition marked by the accumulation of misfolded transthyretin (TTR) protein in tissues, primarily the nerves and heart. NTLA-2001 is a gene-editing therapeutic drug that works in vivo to cure ATTR amyloidosis by lowering TTR levels in the blood. It is based on a clustered model. CRISPR-Cas9 is an acronym for regularly interspaced short palindromic repeats and associated Cas9 endonuclease, and it consists of a lipid nanoparticle encasing Cas9 protein messenger RNA and a single guide RNA targeting TTR.
TTR knockdown was shown to be persistent after a single
dosage in preclinical investigations. During the first 28 days following
infusion, serial safety assessments in patients revealed few adverse events,
and those that did occur were minor in severity. Pharmaco dynamic effects were
shown to be dose-dependent. The mean reduction in serum TTR protein
concentration from baseline was 52 percent (range, 47 to 56) in the group that
got a 0.1 mg per kilogramme dosage and 87 percent (range, 80 to 96) in the
group that received a 0.3 mg per kilogramme dose at day 28.
Administration of NTLA-2001 to a small sample of patients
with hereditary ATTR amyloidosis and polyneuropathy was accompanied with fairly
minor side effects and resulted in lower serum TTR protein concentrations due
to targeted TTR deletion.
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