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

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.

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

The firm is prioritizing development of its anti-DKK1 antibody sirexatamab following promising Phase II results in microsatellite-stable colorectal cancer.

The firm must deprioritize a mitochondrial disease program in order to fast-track PBGENE-DMD to a Phase I study.

The firm will test CAP-002 in a Phase I/IIa trial involving patients with syntaxin-binding protein 1 developmental and epileptic encephalopathy.

The funds will support multiple new and ongoing clinical trials of Tilt's oncolytic adenoviral therapy TILT-123.

Researchers found the therapy downregulated VEGF expression and improved retinal thickness in neovascular age-related macular degeneration patients.

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