A "productive" meeting with the FDA has given the firm confidence in its regulatory filing and commercialization plans for ST-920.

4D Molecular Therapeutics (4DMT) has said a trial of its gene therapy for Fabry disease has been placed on a clinical hold by the FDA, a few weeks after it reported three cases of atypical ...

Sangamo Therapeutics Announces Important Derisking Milestones in Pathway to Anticipated BLA Submission for ST-920 in Fabry Disease ...

This would be in terms of the development of its Fabry Disease program, which uses gene therapy isaralgagene civaparvovec to treat these patients. The reason why investors should care about this ...

is leading the gene therapy study in Calgary.Dr. Jeffrey Medin, Affiliate Scientist at UHN, and MACC Fund Professor at the Medical College of Wisconsin, pioneered the Fabry gene therapy treatment ...

Galafold (migalastat) is an oral precision medicine for adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant, based on in vitro assay data.