FRLN - Freeline gains 8% on gene therapy data in Gaucher and Fabry disease

Freeline Therapeutics (FRLN) perks up 8% premarket in reaction to the announcement of data from its gene therapy programs in Gaucher and Fabry diseases.FLT201 for Gaucher Disease: Data demonstrate potential of FLT201 to deliver sustained levels of ?-glucocerebrosidase (GCase) variant 85 to Gaucher target tissues not sufficiently addressed by standard-of-care enzyme replacement therapy ((ERT)). FLT201 resulted in robust GCase expression in the liver and sustained GCase secretion into the plasma.Restoration of GCase activity after FLT201 injection was observed in difficult-to-reach tissues as shown by decreased levels of disease causing substrate, lyso-Gb1.Freeline expects to initiate first-in-human dose finding studies of FLT201 in late 2021.FLT190 for Fabry Disease: Early development and qualification data suggest that the assay performance is acceptable and accurate in detecting NAbs in Fabry disease samples.These results support further development and validation as a robust, standardized assay for use in gene therapy trials. The company will present these data at 17th AnnualWORLDSymposium,

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Freeline gains 8% on gene therapy data in Gaucher and Fabry disease