ANA 2021 – Ionis rocked by tofersen flop
After the Valor defeat, the group and its partner Biogen will hope that going earlier could improve the amyotrophic lateral sclerosis project’s chances.
Crispr’s reminder about allogeneic Car-T redosing
Crispr follows Allogene with backing for off-the-shelf Car-T therapy – and adds big concerns over lack of durability.
Idorsia’s Fabry flop puts focus on gene therapies
But questions about gene therapy safety could benefit the enzyme-replacement therapy incumbents.
Takeda gets the pipeline blues
A big selloff on a mid-stage stumble seems harsh, but Takeda has problems on the horizon.
Biopharma’s stock market winners and losers so far this year
Moderna is the standout gainer, but the stragglers are becoming more apparent as the pandemic tide retreats.
Editas falls well short of Brilliance
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.
Pfizer adds to gene therapy woes
But another safety scare for the big pharma’s Duchenne muscular dystrophy project could be good news for Sarepta.
