Apic Bio is committed to finding cures for patients with genetic diseases. The company is a spin-off from the University of Massachusetts Medical School (UMMS) and is based upon nearly 30 years of gene therapy research by Apic’s scientific founders. Apic is developing best-in-class treatment options for rare, devastating neurological and liver diseases. Its current pipeline focuses on new and effective treatments for Alpha-1 Antitrypsin Deficiency (Alpha-1, or AATD) and genetic Amyotrophic Lateral Sclerosis (ALS.)



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. In both these scenarios, the THRIVE™ platform provides a superior therapeutic approach than simple gene silencing or gene replacement approaches. The THRIVE™ platform both silences a mutant gene product and replaces a normal gene product in a single
“dual function” vector.



John Reilly M.S., M.B.A.
Co-Founder and CEO

Scott Loiler, Ph.D.
Chief Technology Officer

Daniel Geffken
Interim Chief Financial Officer



Dr. Robert H. Brown Jr., M.D.
Scientific Co-Founder

Dr. Christian Mueller, Ph.D.
Co-Founder and Scientific Advisor



Jason Dinges, Ph.D., J.D.
Investment Advisor,
Morningside Technology Advisory LLC

Felix von Coerper
Managing Partner,
ALS Investment Fund

John Reilly M.S., M.B.A.
Co-Founder and CEO,
Apic Bio

Dr. Christian Mueller, Ph.D.
Co-Founder and Scientific Advisor, Apic Bio




APB-101 is a “liver-sparing” gene therapy designed as a one-time treatment for Alpha-1 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 Alpha-1 protein (M-AAT).


This Apic sponsored program now employs a second-generation vector design with a novel delivery route for the one-time treatment of genetic (SOD1) ALS. This program is based on a compassionate use IND study begun by Robert H. Brown, Jr., DPhil, MD; and Christian Mueller, PhD, in 2017 at the University of Massachusetts Medical School, with collaborators at the Massachusetts General Hospital.




BioCentury, January 2019
Apic: Dual-Gene Therapies (Subscription required)

Wall Street Journal, January 2019
Apic Bio Nabs $40 Million for New Gene-Therapy Approach (Subscription required)

BioWorld, January 2019
Apic Bio secures $40M to fund silence & replace gene therapy (Subscription required)

Chemical & Engineering News, January 2019
UMass gene therapy spin-off Apic Bio launches

MetroWest Daily News, November 2018
UMass Medical Researchers near ALS breakthrough

Wall Street Journal, August 2017
Apic Bio Gets Financing to Tackle Rare Lung Disease (Subscription required)


Published RESEARCH

October 2018
Science Translational Medicine

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

November 2017
Molecular Therapy
Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency
December 2015
Human Gene Therapy
Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates
December 2015
Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice
December 2015
Molecular Therapy
Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors
March 2012
Molecular Therapy
Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles


Apic Bio is always seeking individuals who are talented, dynamic, and passionate about working in an entrepreneurial environment. We offer a fast-paced, challenging, and rewarding experience with a competitive salary plus attractive stock options and benefits. To be considered for a position, please submit your CV:


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