The regulator stopped dosing in the U.S. for a drug that’s meant to be a more potent version of Sarepta's marketed medicine Exondys 51, after a patient experienced dangerously low magnesium levels.

Breyanzi is now approved in the second-line setting, matching Gilead’s Yescarta, which won a similar clearance from the FDA earlier this year.

After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.

Two investment firms, Gurnet Point Capital and Patient Square Capital, have agreed to pay as much as $890 million for Radius, which has come under pressure amid share price declines.

The bulk of the investment will go toward advancing development of three antimalarial drug candidates meant to combat resistance to artemisinin, a common treatment.

The company says a recent coverage decision by Medicare has forced it to end an observational trial of the Alzheimer's drug after enrolling just 29 participants.

The pharma will pay $75 million to access Precision’s gene editing technology, adding another program to the lengthening list of drug development efforts targeting the inherited blood condition.

The Belgian drugmaker will spend more than $100 million to acquire two companies, CellPoint and AboundBio, in a move meant to “disrupt” CAR-T treatment.

Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing.

A confirmatory study of PTC Therapeutics’ muscular dystrophy treatment Translarna missed its main goal, although the company highlighted the trial’s overall positive results. 

Valneva will receive $95 million from Pfizer to support development of a vaccine candidate as the French company’s COVID-19 work sputters.

A medicine at the center of a multibillion-dollar alliance between the two companies passed a major test in transthyretin amyloidosis. Full study details were not disclosed, however.