FLT201: Initial Clinical Data

FLT201 has potential to deliver continuous level of enzyme and penetrate deeper tissues that existing therapies do not reach SINGLE DOSE Potent liver-directed AAVS3 capsid N Novel transgene for longer-acting engineered GCase variant Easier to reach organs: liver, spleen Harder to reach organs: bone, lung O O 00 O O 00 GCase variant 85 with prolonged half-life = more enzyme available to be delivered to cell and longer time in the cell itself FREELNE 10

View entire presentation