PFIC2 is a rare, degenerative pediatric liver disease — and 90% of rare diseases have no cure. Current standards of care fail 66% of patients, often leading to liver failure or cancer before age 5. Available therapy involves daily medications that address symptoms, not the cause.

AMB-01 is a safe, single-dose gene therapy designed to be curative. It targets the genetic root cause by delivering a corrected version of the defective gene directly into affected liver cells — eliminating the need for lifelong symptomatic treatment.