The first clinical evidence for in vivo gene editing, and a surge in valuation, is to be celebrated. But there is much more to do.
A small molecule for alpha-1 antitrypsin deficiency faces a key clinical test, and its chances of success aren’t good.
A rollercoaster year for biopharma ended with investors firmly deciding that the pandemic was an opportunity, not a threat.
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
Reaction to data from Sangamo's ongoing genome-editing trial shows that small numbers can equal big risks.
Beam Therapeutics is developing a new method of Crispr genome editing, but it could get caught up in the same old patent problems.