Experimental Treatments for Gaucher Disease
Several therapies are being developed for Gaucher disease, including gene therapies designed to address the underlying genetic cause of the disease and chaperone therapies that aim to improve the function of the faulty glucocerebrosidase enzyme. Newer versions of enzyme replacement therapy and substrate reduction therapy are also being studied.
PR001
PR001 is a gene therapy being developed to ease symptoms and slow or halt the progression of Gaucher disease type 1. Prevail Therapeutics is currently testing it as a one-time infusion into the bloodstream in a Phase 1/2 clinical trial.
FLT201
FLT201 is an investigational gene therapy designed to ease symptoms and stop or even reverse disease progression in people with Gaucher disease type 1. It is being developed by Spur Therapeutics to be given as a one-time intravenous infusion.
Ambroxol
Ambroxol is an experimental oral chaperone therapy that may help ease both neurological and non-neurological symptoms of Gaucher disease. It has been tested in several clinical studies across all types of Gaucher disease and is sometimes used off-label in regions outside the U.S.
Venglustat
Venglustat is an experimental oral substrate reduction therapy from Sanofi that is currently in Phase 3 trials for the possible treatment of Gaucher disease type 3.
