Challenges With Conducting Clinical Trials in Gaucher Disease: Neil Weinreb, MD


The chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.

“Many of the more severe genotypes and phenotypes that we see in Gaucher disease are actually concentrated in economically challenged countries... whereas for example, in the US, the overwhelming majority of patients have type one Gaucher disease and many of them tend to have very mild disease which doesn't even need to be treated. That’s not necessarily the best population to recruit for trials... There needs to be a regulatory decision to determine how many patients are really needed to be able to reach a conclusion about whether a therapy is effective and safe.”

Gene therapy could serve as a 1-time alternative solution to regular enzyme replacement therapy (ERT), a standard-of-care treatment for Gaucher disease. Companies such as AVROBIO and Prevail Therapeutics (a subsidiary of Eli Lilly) are investigating possible alleys for gene therapy in Gaucher disease.

AVROBIO is conducting a phase 1/2 study (NCT04145037) assessing the lentiviral AVR-RD-02 therapy in patients with type 1 Gaucher disease and is currently enrolling. Prevail is enrolling patients with type 2 Gaucher in a phase 1/2 study (NCT04411654) assessing the adenoviral therapy PR001.

GeneTherapyLive spoke with Neil Weinreb, MD, chair, International Collaborative Gaucher Group Registry, and safety committee, AVROBIO, to learn more about the potential of gene therapy to treat Gaucher disease and other lysosomal storage disorders. He discussed the challenges of conducting clinical trials in Gaucher disease.

AVROBIO Reports Second Quarter 2021 Financial Results and Provides Business Update. News release. AVROBIO. August 5, 2021.
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