FDA releases hold on second of Intellia’s ATTR-CM gene therapy trials
Two Phase III trials were put on clinical hold by the FDA in October 2025.
Cytiva Navigating cell therapy WP 4
FDA tells uniQure to conduct another study of Huntington’s gene therapy
Gene editing takes centre stage in FDA’s new rare disease approval pathway
BioRestorative concludes Phase II BRTX-100 trial enrolment for cLDD
UniQure pauses dosing in Fabry disease trial amid safety concerns
Lilly stops development of three drugs, including gene therapy
Affinia receives FDA approval for Phase I/II trial for dilated cardiomyopathy
Ultragenyx’s gene therapy shows sustained improvements in MPS IIIA
