This treats a form of amyloidosis by inhibiting the production of the transthyretin protein. A lot of research still needs to be done for LNPs to act as optimal carriers in all their varied roles.
Scripps Research scientists discovered that some sections of the hormone-carrying molecule transthyretin (left) are in constant motion (right), which may explain why the protein can misfold and ...
Here, the authors explore how composition and structure of protein aggregates affect dormancy and exit from this state. Cryo-EM reveals how transthyretin moves, offering insights into ligand ...
whereby amyloid fibrils made from misfolded transthyretin protein accumulate in the heart. In most patients, death is caused by sudden heart failure or heart-related issues. The trial revealed ...
NI006 is an antibody that binds to the misfolded forms of the transthyretin protein that form the fibrils, and cause them to be cleared away by immune cells like macrophages. Tafamidis is thought ...
Similarly, nex-z is in late-stage trials for ATTR amyloidosis, a rare condition caused by the accumulation of misfolded transthyretin protein. Intellia is running two Phase III studies ...
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