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Valerion Provides Proof-of-Concept Data for VAL-0417 for Treatment of Lafora Disease.

M2 PHARMA-September 7, 2017-Valerion Provides Proof-of-Concept Data for VAL-0417 for Treatment of Lafora Disease


- US-based clinical-stage biotechnology company Valerion Therapeutics has provided preclinical proof-of-concept results showing early validation of VAL-0417 as a potential treatment for Lafora disease, a rare and fatal genetic form of epilepsy, characterized by an accumulation of aberrant glycogen-containing deposits known as Lafora bodies, the company said.

VAL-0417 is a novel fusion protein that combines Valerion's delivery antibody linked to amylase, to uniquely target Lafora bodies present in all tissues of Lafora disease patients.

In a presentation titled "Lafora Body Degradation by a Therapeutic Enzyme for the Treatment of Lafora Disease," Valerion researchers and collaborators from the University of Kentucky demonstrated, in vitro and in vivo, the ability to deliver VAL-0417 to the skeletal muscle and brain with retention of up to 24 hours following intracerebroventricular (ICV) injection in Lafora mouse models.

Lafora Disease, a rare, progressive, autosomal recessive neurodegenerative disorder characterized by intractable seizures, difficulty walking, muscle spasms, neurological deterioration, rapid cognitive decline, dementia, and death typically within ten years of onset, is caused by loss-of-function mutations in either the laforin gene (EPM2A) or malin gene (EPM2B) and is associated with gradual accumulation of Lafora bodies, aggregates of poorly branched, hyperphosphorylated, insoluble glycogen also known as polyglusan.

Valerion Therapeutics, part of the Alopexx Enterprises portfolio of companies, specializes in the development of therapies for orphan genetic diseases through its proprietary antibody-mediated delivery platform, which enables enhanced intracellular delivery of a range of active therapeutic molecules by way of a transport mechanism present in muscles and neurons.

The company's product candidates target disease tissues via a novel antibody with cell-penetrating properties dependent on a tissue-localized membrane transporter.

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Publication:M2 Pharma
Article Type:Disease/Disorder overview
Date:Sep 7, 2017
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