With pivotal sepofarsen data due, the group will hope to get one up on Editas.
Praxis, Proqr and Nordic Nanovector are awaiting important data.
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.
The group’s antisense project is the only game in town for Usher syndrome, but its latest trial is not a slam-dunk.
Several key data readouts slated for the first quarter remain unannounced, from Roche, Lilly, Axsome and others, but the Covid-19 pandemic puts many at risk of delay.
Roche's Mirros study should prove idasanutlin's worth on top of chemotherapy, while Proqr hopes to Usher in a rare disease cure.
Promising data with Fibrocell’s epidermolysis bullosa gene therapy send the company down as funding worries kick in.
Three gene therapy contenders, Abeona, Fibrocell and Krystal, are taking different approaches in the quest to cure epidermolysis bullosa.