Important data reveals are expected from Madrigal, Arrowhead and Ascendis, with details from Biogen and Eisai due at a conference.
Though broad questions intensify, NTLA-2001 continues showing clinically what it is meant to.
Novel approaches including gene editing are creeping towards approval.
With pivotal sepofarsen data due, the group will hope to get one up on Editas.
SAR445136 could take on Crispr and Vertex’s CTX001, but plenty of other groups have the same idea.
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.