ART001: targeting ATTR amyloidosis
ACCUREDIT THERAPEUTICS DELIVERS BEST
IN VIVO GENE EDITING PRODUCTS IN RECORD SPEED
> 90% (up to 98%) serum TTR reduction after 4 weeks
No infusion-related reaction observed
No > Grade 1 liver enzyme elevation observed
No off-target editing detected at up to ~80 times of EC90
No off-target-related translocation detected
No large indel affecting genomic region with known functions (except for TTR) detected
