Challenges in Developing Gene Therapies for Gaucher Disease: Neil Weinreb, MD


The chair of the International Collaborative Gaucher Group Registry discussed the place of gene therapies in the Gaucher treatment landscape.

“Other [lysosomal storage] diseases are a little bit easier to discuss in terms of gene therapy, because there's so little available to help people, whereas traditional type one Gaucher, or non-neuropathic Gaucher disease, we actually have almost a wealth of therapies right now. And then convincing somebody who was being well controlled on 1 of the available treatments that they should try something which is still investigational and not without hazard is a bit of a challenge.”

Gaucher disease, while in many cases adequately controlled by enzyme replacement therapy (ERT), represents another area of opportunity to develop gene therapies as a curative solution for patients whose disease may be inadequately controlled by ERT. 

Companies such as AVROBIO and Prevail Therapeutics (a subsidiary of Eli Lilly) are investigating gene therapies for Gaucher disease. AVROBIO is currently enrolling patients with type 1 Gaucher disease in a phase 1/2 study (NCT04145037) assessing the lentiviral AVR-RD-02 therapy and 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 challenges of developing and investigating gene therapies in LSDs and Gaucher disease, including benefit-risk profiles and timing treatments.

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