The firm announced deals with UMass Chan Medical School and Quiver to further its ambitions to develop transformative treatments for the condition.

The treatment has been used in a trial to treat children with GM1 gangliosidosis, but if the FDA allows, this would be the ...

The baby boy with a rare urea cycle disorder received the bespoke therapy within a few months of birth and is doing well, ...

The baby boy received the bespoke therapy within a few months of birth and is doing well, according to a presentation at ASGCT.

The trial aims to enroll 40 patients with FGFR alterations across the US, who won't need to travel far to receive care.

The study tests the autologous CAR T-cell therapy LEU011 in patients who express NGK2DL in at least 10 percent of tumor cells.