Voyager Posts Encouraging Preclinical Data For CNS-Directed Gene Therapy

Voyager Therapeutics Inc VYGR has announced the presentation of preclinical data of GBA1 gene replacement therapy.
The data demonstrated that a single intravenous (IV) dose of GBA1 gene replacement therapy achieved widespread distribution in the central nervous system (CNS) and peripheral tissues and substantially raised levels of the GCase enzyme in animal models.
The results were presented at the European Society of Gene and Cell Therapy (ESGCT).
The increase over endogenous GCase levels in the range of 300% - 660% suggested a promising rise in GCase levels of 30% - 50% that is anticipated to be clinically impactful.
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