CAMBRIDGE, Mass.– AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) to investigational AVR-RD-02, a first-in-class gene therapy that uses patients’ own hematopoietic stem cells to treat Gaucher disease, a rare lysosomal disorder that can lead multiorgan pathology, clinical morbidity and early mortality.
The Innovation Passport is the first step in the ILAP process, triggering the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and National Health Service (NHS) England, to chart a roadmap for regulatory and development milestones with the goal of early patient access in the U.K.
AVROBIO’s Gaucher disease program includes Gaucher disease type 1 and type 3, which collectively impact an estimated 30,000 patients worldwide. The company expects to provide an interim clinical data update for its Phase 1/2 Gaucher disease type 1 clinical trial, as well as an outline of the development and regulatory strategy for its Gaucher disease type 3 program, in Q4 2022.
