Biotech Gene Therapy Startup Apic Bio Launches in Cambridge, Massachusetts

August 23, 2017
By Mark Terry, BioSpace.com Breaking News Staff

Cambridge, Mass. – Apic Bio launched today with $1 million seed funding by the venture philanthropy arm of the Alpha-1 Foundation and a private investor, Ed Krapels, who is living with Alpha 1. The Alpha-1 Foundation invested $500,000 in the company, as did Krapels.

The company will focus on gene therapy approaches to treat Alpha-1 Antitrypsin Deficiency (Alpha 1). Its lead product is APB-101, which targets the liver by way of an AAV delivered Dual Function Vector (df-AAV). In this process, the Z-AAT protein is silenced and M-AAT protein is augmented.

APB-101 has shown proof-of-concept in the preclinical setting in vitro and in vivo. The company is presently evaluating preclinical GLP toxicology studies in non-human primates.

“We are grateful to TAP and A1AT Investors, LLC who have supported the successful start of Apic Bio by providing the first tranche of our seed financing round allowing us to secure key intellectual property rights and operational support,” said John Reilly, Apic’s co-founder and president, in a statement. “With such strong support from the advocacy and patient community, we are confident that we will identify the right corporate partners to help us achieve our business development goals and bring this exciting new therapy to patients.”

People with Alpha 1 do not have sufficient levels of circulating AAT protein to protect lung tissue against damage from proteases. This results in the accumulation of mutant AAT polymers in the liver. Patients show progressive emphysema and the accumulation of abnormal AAT polymers increase the risk of liver cirrhosis.

The company is a spinoff from the University of Massachusetts Medical School (UMMS). It is built on the gene therapy research of Christian Mueller, associate professor of Pediatrics and a member of the Horae Gene Therapy Center at UMMS and Terence Flotte, the Celia and Isaac Haidak Professor in Medical Education, dean of the School of Medicine and provost and executive deputy chancellor of the UMMS. They are the scientific founders of Apic. In part, the research is funded by an $11 million grant from the National Heart, Lung, and Blood Institute (NHLBI).

“We are encouraged by the feedback that we have received during our pre-IND meeting with the FDA that there is a clear path for us to conduct a first-in-human Phase I/II clinical study,” Mueller said in a statement. “Furthermore, we are very much looking forward to demonstrating the benefit of APB-101 to patients that have been living with alpha-1 and have had very little hope for a cure. Our data suggests this is a ‘liver sparing’ approach for gene augmentation which may exceed the therapeutic and safety margins when compared to a strict gene augmentation without gene silencing that may exacerbate the underlying liver disease.”

Ed Krapels is the chief executive officer of Anbaric, a power line developer. A former financial advisor and risk management consultant, he was a member of the Department of Energy Secretary Steven Chu’s Electricity Advisory Committee from 2010 to 2012. His brother, Peter Krapels, died at the age of 45 from Alpha-1 antitrypsin deficiency.