Galectin 1: A novel small protein therapy for Duchenne muscular dystrophy

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R41AR067014-01
Agency Tracking Number: R41AR067014
Amount: $225,000.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: NIAMS
Solicitation Number: PA13-235
Timeline
Solicitation Year: 2014
Award Year: 2014
Award Start Date (Proposal Award Date): 2014-09-01
Award End Date (Contract End Date): 2016-08-31
Small Business Information
1664 N VIRGINIA ST, A.R.F. MS/328, Reno, NV, 89557-0001
DUNS: 079173703
HUBZone Owned: N
Woman Owned: N
Socially and Economically Disadvantaged: N
Principal Investigator
 DEAN BURKIN
 (775) 784-6288
 dburkin@medicine.nevada.edu
Business Contact
 DAVID MAINE
Phone: (775) 223-6169
Email: david@strykagen.com
Research Institution
 UNIVERSITY OF NEVADA RENO
 204 ROSS HALL MAILSTOP 325
RENO, NV, 89557-0001
 Nonprofit college or university
Abstract
DESCRIPTION provided by applicant Duchenne Muscular Dystrophy DMD is a fatal muscle disease affecting in every male births DMD results from mutations in the gene encoding the dystrophin a kDa scaffolding protein responsible for providing a mechanical link between the muscle fiber actin cytoskeleton and a transmembrane protein complex called the dystrophin associated glycoprotein complex DGC Although life expectancy for DMD patients has gone up in recent years the only currently approved treatment remains corticosteroids which have limited positive effects including inflammation inhibition and counterproductive side effects Recent research studies have focused on using virally mediated gene replacement direct gene repair myoblast cell transfer small molecule protein enhancement exon skipping small molecules and protein therapeutics The Burkin lab has recently shown that recombinant mouse Galectin is capable of stabilizing and restoring the normal protein levels of dystrophin associated proteins normally in DMD Galectin treatment also leads to elevated levels of both utrophin and integrin in skeletal muscle critical modifying proteins that act to protect the fragile sarcolemmal from damage in the absence of dystrophin The enhanced sarcolemmal stability leads to a decreased myofiber regeneration and damage Galectin also functions in muscle to limit inflammation and thereby fibrosis Finally Galectin treated mdx mice displayed elevated strength and activity levels relative to controls Strykagen plans to further develop Galectin protein therapy towards an IND application by producing purified human Galectin protein capable of getting FDA approval for phase I clinical trials In collaboration with our academic partner the purified recombinant galectin will be used in preclinical studies in the mdx mice to demonstrate muscle protecting properties Results from this study should lead directly into a phase II STTR grant for preclinical studies in the GRMD dog model of DMD Together these studies will lead to an IND application with the FDA and clinical trials to develop Galectin protein as a novel treatment for DMD PUBLIC HEALTH RELEVANCE Duchenne Muscular Dystrophy DMD is a fatal muscle disease that currently has no cure and limited treatment options Several studies have demonstrated utrophin and the integrin are major modifiers of disease progression in DMD and targets for drug based therapeutics Dr Dean Burkin has identified that mouse galectin a small kDa protein increases both utrophin and integrin to therapeutic levels in the mdx mouse model of DMD This study aims to produce recombinant human galectin protein and determine if this therapeutic can prevent muscle disease in the mdx mouse model of DMD

* Information listed above is at the time of submission. *

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