Freeline unveils early efficacy signal for Gaucher gene therapy: #ASGCT24
BALTIMORE — Four patients with Gaucher disease were able to stop their regular treatments after receiving a one-time infusion of a gene therapy developed by Freeline Therapeutics.
The four patients have maintained or seen improvements in certain biomarkers of Gaucher disease following the gene therapy, according to new results shared Thursday morning by Freeline at the American Society of Gene & Cell Therapy annual meeting. None of the four have had to restart either enzyme replacement therapy or substrate reduction therapy, which are standard treatments for the disease.
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