Rare Daily Staff
Apic Bio said it completed a $40 million financing to advance its experimental gene therapy programs and support additional discovery efforts.
Morningside Venture Investments led the round. The ALS Investment Fund also participated in the funding with existing investors The Alpha-1 Project and A1ATD Investors. Patients living with Alpha 1 lack sufficient levels of circulating Alpha 1 protein (AAT) to protect lung tissue against damage from proteases and experience the accumulation of mutant AAT polymers in the liver. Clinically, the deficiency is manifested by progressive emphysema and the accumulation presents a significant risk of liver cirrhosis.
The company is developing APB-101 as a one-time gene therapy treatment for Alpha-1 Antitrypsin Deficiency patients. In pre-clinical studies it has demonstrated the ability to reduce levels of the mutant Alpha-1 protein (Z-AAT) and at the same time program liver cells to produce the correct AAT protein (M-AAT).
The company is also developing a gene therapy, APB-102, as a one-time treatment for amyotrophic lateral sclerosis (ALS) caused by a mutation in the SOD1 gene.
In connection with the financing, Jason Dinges from Morningside joins the board and Felix von Coerper from the ALS Investment Fund will become a board observer.
Additionally, Scott Loiler, who was previously the Scientific Director of Vector Development at Nationwide Children’s Hospital, joins the company as CTO. Loiler is a recognized gene therapy expert who was at the center of multiple translational gene therapy programs during his time at the NCH.
The company said the funding will also be used to advance its THRIVE gene therapy platform, which both silences mutant gene production and replaces a normal gene product in a single, dual-function vector. Numerous diseases are associated with inherited or somatic mutations. In many cases, these mutations can lead to both a toxic gain of function and loss of function as in the case for Alpha-1 antitrypsin deficiency. In other cases, mutations in a single allele lead to dominant toxicity without a clear loss of function but approaches to reduce their toxicity by non-specifically silencing both alleles can unmask or result in a loss of function.
“Rare diseases with both toxic gain of function and loss of function remain a major area of unmet medical need in the gene therapy field,” said John Reilly, CEO of Apic Bio. “We believe that our breakthrough approach that both silences mutant genes and simultaneously replaces normal, beneficial genes for monogenic diseases, could provide patients with an effective single treatment therapy.”
January 7, 2019
Photo: John Reilly, CEO of Apic Bio