Imetelstat

Excerpt

Originally known as GRN163L, imetelstat sodium (imetelstat) is a 13-mer N3’—P5’ thio-phosphoramidate (NPS) oligonucleotide that has a covalently bound 5’ palmitoyl (C16) lipid group. The proprietary nucleic acid backbone provides resistance to the effect of cellular nucleases, thus conferring improved stability in plasma and tissues, as well as significantly improved binding affinity to its target. The lipid group enhances cell permeability to increase potency and improve pharmacokinetic and pharmacodynamic properties.

The compound has a long residence time in bone marrow, spleen and liver. Imetelstat binds with high affinity to the template region of the RNA component of telomerase, resulting in direct, competitive inhibition of telomerase enzymatic activity, rather than elicit its effect through an antisense inhibition of protein translation. Imetelstat is administered by intravenous infusion.

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Telomerase

Excerpt

Cell division plays a critical role in the normal growth, maintenance and repair of human tissue. Telomeres are repeated sequences of DNA at the ends of each chromosome, and are key genetic elements involved with the regulation of cell division. Telomeres shorten every time a cell divides, and once telomeres reach a critically short length, the cell either dies by apoptosis or stops dividing and senesces.

Telomerase is a naturally occurring enzyme that maintains telomeres and prevents them from shortening during cell division. Telomerase consists of at least two essential components: an RNA template, which binds to the telomere, and a catalytic subunit with reverse transcriptase activity, which adds the specific DNA sequence to the chromosome ends each time a cell divides.

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