Avrobio touts early clinical data and plans new gene therapy trial in 2023
Those with Gaucher disease have to practically live at the hospital to receive bi-weekly, hours-long infusions of enzyme replacement therapy — for the rest of their lives. It’s inconvenient and can be costly.
Avrobio, a clinical-stage gene therapy company, is trying to change how Gaucher is treated by developing a one-time gene therapy called AVR-RD-02 that has shown promising, albeit very early, results for Gaucher disease Types I and III in two separate trials.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.