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4D Molecular Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of 4D-310 for the Treatment of Fabry Disease
EMERYVILLE, Calif., March 09, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: 4DMT), a clinical-stage gene therapy company harnessing the power of
About this update from 4d Molecular Therapeutics, Inc.
[{"type":"text","content":"EMERYVILLE, Calif., March 09, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: 4DMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-310 for the treatment of Fabry disease. Fabry disease is an inherited lysosomal storage disease with high unmet medical need that results from loss of function mutations in the alpha-galactosidase (AGA) enzyme. “Through our Therapeutic Vector Evolution platform we apply the principles of directed evolution to invent targeted and evolved AAV vectors for the delivery of genes to specific tissue types. Dosing the first patient in the Phase 1/2 clinical trial of 4D-310 marks the third product candidate to be administered to patients, all of which utilize proprietary vectors derived from our Therapeutic Vector Evolution platform,” said David Kirn, MD, chief executive officer, co-founder and president of 4DMT. “4D-310 is designed with the goal of achieving a novel dual mechanism-of-action for these patients. 4D-310 is designed for both stable therapeutic AGA enzyme activity in the blood, as well as for intracellular production within affected tissues, including in cardiac muscle cells.” “The unmet need for patients with Fabry disease is significant. Current standard of care enzyme replacement therapies are associated with a high-treatment burden, and due to the short half-life in the blood, patients lack therapeutic concentrations of AGA between infusions. Cardiovascular disease is still the leading cause of death in patients despite standard therapy. Gene therapy represents a promising therapeutic modality for patients with Fabry disease because of its potential as a one-time therapy that can deliver stable and sustained levels of AGA activity,” said Dr. Raphael Schiffmann, MD, Senior Vice President & Therapeutic Area Head, Cardiology at 4DMT. “The targeted organ distribution of 4D-310 in Fabry disease makes it likely that this therapy can also potentially treat the cardiomyopathy associated with Fabry disease, a critical disease manifestation that other therapies have been unable to address.” The Phase 1/2 open-label, dose-exploration and dose-expansion study is expected to enroll up to 18 Fabry disease patients. The study is designed to assess ...