pharmaphorum
JULY 13, 2021
The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a life-threatening disease affecting the heart and nervous system.
Delveinsight
AUGUST 6, 2020
Transthyretin (TTR) is a protein that the liver produces to facilitate the transfer of thyroid hormone and vitamin A in the blood. However, due to specific reasons, the TTR protein begins to misfold and aggregate as fibrils, a process known as amyloidosis of Transthyretin (ATTR). Gene-silencing Therapies.
Roots Analysis
OCTOBER 22, 2023
Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.
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