Gene Therapy 4D-310 Safe, Restores Gal A Enzyme in 3 Dosed Patients

Gene Therapy 4D-310 Safe, Restores Gal A Enzyme in 3 Dosed Patients

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Gene Therapy 4D-310 Safe, Restores Gal A Enzyme in 3 Dosed Patients

A single dose of 4D-310, an investigational gene therapy from 4D Molecular Therapeutics (4DMT), appears generally safe and restored the levels of alpha-galactosidase A (Gal A), the faulty enzyme in Fabry disease, in the first three men enrolled in a Phase 1/2 clinical trial. Notably, all three patients were free from enzyme replacement therapy (ERT) at their last follow-up assessment, ranging from six weeks to six months. Moreover, their levels of globotriaosylsphingosine, or Lyso-Gb3 —…

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