AvroBio receives rare paediatric disease designation from the US FDA for first in class gene therapy for Gaucher disease

AvroBio

27 October 2022 - AVR-RD-02 has previously received fast track status from FDA, orphan drug designation in the US and EU, and ILAP designation from the UK MHRA.

AvroBio today announced that the US FDA has granted rare paediatric disease designation to AVR-RD-02, a first in class investigational gene therapy that genetically modifies patients’ own haematopoietic stem cells to treat Gaucher disease, a rare lysosomal disorder that can lead to multi-organ pathology, clinical morbidity and early mortality.

Read AvroBio press release

Michael Wonder

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Michael Wonder