Apic: Dual-gene therapies

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 from other treatment modalities because it incorporates both gene silencing and gene augmentation into a single adeno-associated viral (AAV) vector. Reilly is co-founder and CEO, and Mueller is co-founder and scientific adviser.

Unlike single-function therapies that either silence or replace a gene, Mueller said, THRIVE is tailored for diseases like AAT deficiency, where a gene mutation results in the loss of functional protein and the...