Proqr eyes its first big test
With pivotal sepofarsen data due, the group will hope to get one up on Editas.
Key data reveals for the small players
Praxis, Proqr and Nordic Nanovector are awaiting important data.
Editas falls well short of Brilliance
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.
Proqr hopes to usherin a new ophthalmic therapy
The group’s antisense project is the only game in town for Usher syndrome, but its latest trial is not a slam-dunk.
Will pending clinical readouts make it across the line?
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.
Upcoming events – pivotal data for idasanutlin and an interim look at Proqr's exon-skipper
Roche's Mirros study should prove idasanutlin's worth on top of chemotherapy, while Proqr hopes to Usher in a rare disease cure.
Fibrocell gets a reality check
Promising data with Fibrocell’s epidermolysis bullosa gene therapy send the company down as funding worries kick in.
Gene therapies go skin deep to tackle epidermolysis bullosa
Three gene therapy contenders, Abeona, Fibrocell and Krystal, are taking different approaches in the quest to cure epidermolysis bullosa.
