Fabry disease
UniQure Doses First Fabry Disease Patient in Phase I/IIa Gene Therapy Trial
The company plans to enroll up to six men with Fabry disease in each of two cohorts testing different dose levels of the investigational gene therapy.
FDA Lifts Hold on 4D Molecular Therapeutics' Fabry Disease Cardiomyopathy Gene Therapy Trial
Now that the clinical hold has lifted, 4DMT said it expects to resume enrollment in the Phase I/II trial evaluating 4D-310 in the second half of 2024.
Sangamo Therapeutics Gets FDA Feedback on BLA for Fabry Disease Gene Therapy
Although the regulatory path for isaralgagene civaparvovec is becoming clearer, the firm won't advance a registrational trial without a commercialization partner.
UniQure Fabry Disease Gene Therapy Gets FDA IND Clearance
Following clearance of the investigational new drug application, the company is planning a first-in-human dose escalation trial in the US.