transthyretin-mediated amyloidosis
Improved Cytosine Base Editing Tech Eliminates Problem of Undesired Bystander Mutations
The percentage of perfectly modified alleles using the new base editor is more than 6,000-fold for disease correction compared with the current BE4max editor.
Alnylam Pharmaceuticals and collaborators this week published data from phase I trials of the siRNA-based transthyretin amyloidosis drugs ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine.
Alnylam Pharmaceuticals this week said that it has begun dosing patients in a phase I study of its siRNA-based transthyretin-mediated amyloidosis treatment ALN-TTR02.
Alnylam also released its first-quarter financial results, reporting higher losses and lower revenues.
Jan 27, 2011
Jan 6, 2011