Freeline Publishes Preclinical Proof-of-Concept Data for FLT190, its AAV Gene Therapy Candidate for Fabry Disease, in the Nature Journal Gene Therapy
Data show increased enzyme activity and reduction of harmful substrate, an established biomarker of efficacy in Fabry disease, in key tissues and organs
FLT190 currently being investigated in MARVEL-1 Phase 1/2 clinical trial
LONDON, Jan. 19, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced the publication of preclinical proof-of-concept data for its gene therapy candidate for Fabry disease, FLT190, in the Nature journal Gene Therapy. Fabry disease is a debilitating genetic disorder in which an enzyme deficiency leads to a harmful build-up of fat in the cells that causes progressive organ damage and can result in early death.
“People living with Fabry disease can experience debilitating symptoms and disease progression, even with current treatments. There is a pressing need for therapies with more lasting efficacy that are less burdensome on Fabry families,” said Pamela Foulds, M.D., Chief Medical Officer at Freeline. “We are encouraged by these preclinical data that support our ongoing Phase 1/2 clinical trial of FLT190 and our belief in FLT190 as a potential life-changing, one-time therapy for people with Fabry disease.”
Key findings that support development of FLT190 based on the preclinical evaluation in a Fabry mouse model and non-human primates following a single intravenous dose include:
A link to the publication, “Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease,” can be found here: https://www.nature.com/articles/s41434-022-00381-y
Freeline is currently enrolling patients in the second dose cohort of MARVEL-1, an international, multicenter, adaptive dose-escalation and dose-expansion Phase 1/2 clinical trial in adult men (= 18 years) with classic Fabry disease. The company expects to share initial safety and efficacy data from the second dose cohort of MARVEL-1, as well as updated data from the first cohort, in the first half of 2023.
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