News

Advances in Neurodegenerative Disease Research and Therapy

Keystone Resort, Keystone, Colorado, USA Antibody-enzyme fusions degrade pathogenic glycogen in Lafora disease and demonstrate a novel cell-penetrating platform for treating neurological disorders Kathryn Brewer1, Annette Uittenbogaard1, Grant Austin1, John McCarthy1, Anna DePaoli-Roach2, Peter J. Roach2, Brad Hodges3, James R. Pauly3, Tracy McKnight3, Dustin D. Armstrong3*, Matthew S. Gentry1   1University of Kentucky, 2Indiana University, 3Valerion Therapeutics *dustin.armstrong@valerion.com   […]READ MORE...

Valerion Presents Preclinical Proof-of-Concept Data for VAL-0417 for the Treatment of Lafora Disease

– High Unmet Need for this Rare and Fatal Form of Epilepsy – – Planning Underway for Initiation of Clinical Studies in Lafora Patients – CONCORD, Mass., Sept. 6, 2017 /PRNewswire/ — Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced preclinical proof-of-concept results providing early […]READ MORE...

Novel approaches to improved treatments for orphan genetic diseases: Enhanced intracellular delivery of protein therapy through natural transport mechanisms

The development of a means to restore missing proteins to diseased cells consistently and efficiently remains a prominent goal in molecular medicine. While gene therapy has the potential to accomplish this task, considerable obstacles must be overcome before it becomes clinically applicable. Protein therapy on the other hand, comprises the direct delivery of missing proteins […]READ MORE...

Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease

– Potential New Treatment Option Designed to Improve Glycogen Clearance for Better Patient Outcomes – – Top-line Clinical Data Expected in the Fourth Quarter of 2017 – CONCORD, Mass. July 11, 2017—Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced that it has initiated dosing […]READ MORE...

MLSC Portfolio Spotlight

Valerion Therapeutics, based in Concord, MA, is a biotechnology company focused on developing targeted therapies for orphan genetic diseases. Valerion has developed a first in class and multifunctional antibody-delivery platform capable of efficiently targeting biotherapeutics to key tissues including neurons and skeletal muscle fibers. More importantly, the company’s proprietary platform delivers functional cargo to intracellular […]READ MORE...

Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium

CONCORD, Mass., Sept. 3, 2015 /PRNewswire/ — Valerion Therapeutics, LLC yesterday announced positive results from a non-clinical “proof of concept” pilot study for a novel humanized antibody and acid alpha glucosidase (GAA) fusion candidate for the treatment of Pompe disease. The studies performed with Valerion’s proprietary product candidate [VAL-1221 (3E10Fab-GAA)] are part of a broad […]READ MORE...