UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms

BioSpace·Mar 5

✦ NabkaNews BriefAuto-summarized from multiple outlets · verify with the source

UniQure's path forward for its Huntington's gene therapy is uncertain due to ethical questions and disagreements with the FDA. The company may be seeking approval in the UK after encountering issues with the FDA, and there are conflicting reports on the status of the therapy's potential phase 3 trial. The situation has highlighted broader concerns about the FDA's approach to data sharing and external controls in the cell and gene therapy space.

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