News

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...

Catalent And Valerion Therapeutics Collaborate To Bring New Treatments For Orphan Genetic Disorders To Market

Somerset, N.J. – December 2, 2014 — Catalent Pharma Solutions, the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products, today announced that it is to collaborate with Valerion Therapeutics, LLC, an emerging science-driven company, focused on the development of bio-therapeutics for orphan genetic diseases, to develop […]READ MORE...

MTM: MDA Funds Development of Myotubularin-Based Treatment

Valerion Therapeutics (formerly 4s3 Bioscience), with support from MDA, will continue testing its experimental myotubularin-based compound in mice and will begin producing a pharmaceutical-grade compound. The compound, 3E10FV-MTM1, has shown benefit in myotubularin-deficient mice with a disorder mimicking human MTM.READ MORE...