Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.

Moving forward, the biotech said it will focus its resources on developing treatments for genetic liver diseases and on its partnered programs.

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

In a Phase Ia trial in ovarian cancer, a strong neutralizing antibody response correlated with better disease control and ...

The firms launched the global Phase II bladder cancer trial for autogene cevumeran after seeing early success in pancreatic ...

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

Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

The ASO for myotonic dystrophy type 1 was well tolerated and improved splicing and function in a study presented at ASGCT.

From decoy DNA sequences to plasmapheresis, researchers are testing strategies to get around anti-AAV antibodies that hinder ...

The European Commission approval was based on results showing the immunotherapy regimen improved event-free survival in a Phase III trial.

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

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