Gal A Activity Nears Normal in 2nd Man Given FLT190 Gene Therapy

Gal A Activity Nears Normal in 2nd Man Given FLT190 Gene Therapy

312465

Gal A Activity Nears Normal in 2nd Man Given FLT190 Gene Therapy

FLT190, Freeline Therapeutics’ experimental gene therapy, led to sustained increases in alpha-galactosidase A (Gal A), the faulty enzyme in Fabry disease, in the first two patients given the one-time therapy in the Phase 1/2 MARVEL-1 clinical trial. Notably, the second patient, dosed early this year, has reached near-normal Gal A levels and remained free of enzyme replacement therapy (ERT) four months post-treatment. These “highly encouraging” results were achieved in the trial’s low-dose group “and already FLT190 appears to…

You must be logged in to read/download the full post.