Apic Bio is a gene therapy company spun out of the University of Massachusetts Medical School that develops dual-function gene therapies using its proprietary THRIVE™ silence-and-replace platform. Its lead programs include APB-101, a liver-sparing one-time therapy for Alpha-1 antitrypsin deficiency, and APB-102, a second-generation vector program for SOD1 ALS that builds on a compassionate use IND. The company is advancing preclinical Alpha-1 and ALS programs, including gene insertion of AAT and targeting of the C9orf72 mutation, and plans discovery work leveraging THRIVE. Apic holds an exclusive license to U.S. Patent No. 10,077,452 from UMass Medical School covering rAAV-based compositions and methods. The company is based in Cambridge, Mass., and emphasizes developing scalable manufacturing approaches, with plans to rethink AAV production and oversee CDMO relationships. Recent leadership hires include CTO Scott Loiler, bringing cGMP manufacturing and regulatory experience to support IND-enabling toxicology and human clinical studies. Apic Bio is a pre-clinical gene therapy company leveraging a proprietary dual-function AAV (df-AAV) platform to treat rare diseases with complex mechanisms, initially targeting Alpha‑1 Antitrypsin Deficiency (Alpha 1). Its lead product, APB-101, uses an AAV-delivered Dual Function Vector to silence the Z‑AAT mutant protein while augmenting normal M‑AAT, and has demonstrated efficacy in vitro and in vivo. APB-101 is undergoing pre-clinical GLP toxicology studies in non-human primates and the company reports a pre-IND meeting with the FDA that outlines a path to a first-in-human Phase 1/2 study. Apic Bio launched with seed financing that provided an initial tranche to secure key intellectual property rights and operational support, led by the venture philanthropy arm of the Alpha‑1 Foundation and a private investor with the disease. The df-AAV platform is positioned to address other diseases where a mutant gene product must be reduced while augmenting the normal gene product, and the company frames APB-101 as a potentially "liver sparing" approach compared with augmentation alone. Apic Bio is a spin-off from the University of Massachusetts Medical School; its scientific founders’ research is funded in part by an $11M grant from the National Heart, Lung, and Blood Institute.

Total raised

$40M

Funding rounds

2

Latest round

Series A

Latest activity

Jan 2019