Apic Bio Inc.'s APB-101 is a dual-function gene therapy for AAT deficiency that silences the mutant AAT gene and augments wild-type AAT expression, which the company believes gives it greater efficacy and lower liver toxicity than other gene silencing or gene replacement therapies for the disease.
Apic's John Reilly and Christian Mueller said APB-101, which was designed using Apic's THRIVE platform, differs