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Optimization of an integrin enhancing molecule for the treatment of Duchenne muscular dystrophy

Award Number: R41AR067019
ORGANIZATION: NATIONAL INSTITUTE OF ARTHRITIS & MUSCULOSKELETAL & SKIN DISEASES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 04/01/2015
PERIOD OF PERFORMANCE END DATE: 03/31/2017

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Optimization of an integrin enhancing molecule for the treatment of Duchenne muscular dystrophy - ¿ DESCRIPTION (provided by applicant): Duchenne muscular dystrophy is caused by mutations in the dystrophin gene resulting in the loss of the dystrophin glycoprotein complex in skeletal muscle. Loss of dystrophin results in reduced sarcolemmal integrity, progressive muscle damage and compromised muscle function. There is currently no cure and limited treatment options for DMD and all affected children will die from this genetic disease unless new treatments are discovered soon. The ¿7¿1 integrin is major laminin receptor which can act as a surrogate for the dystrophin complex in skeletal muscle. Studies have demonstrated that enhanced transgenic expression of the ¿7 integrin in skeletal muscle can improve muscle strength, function and longevity of mouse models of DMD. Together these studies indicate that drugs that target an increase ¿7 integrin in skeletal muscle may serve as novel therapies for DMD. The Burkin laboratory has developed and used a muscle cell-based assay to identify small molecules that increase the ¿7 integrin in skeletal muscle. Strykagen Corp. has the option to license Stryka-969, the top "hit" compound identified from this screen. In this Phase I STTR application we will screen Stryka-969 compound analogs using the muscle cell based assay and develop structure activity relationships (SAR), perform medicinal chemistry and identify lead compounds with optimal activity that target the ¿7 integrin in mouse and human myogenic cells for further development. Second we will confirm on-target and off- target activity of each lead compound in human DMD myogenic cells. This proposal will translate exciting studies in transgenic mice into the development of a new line of ¿7 integrin-targeted therapeutics for the treatment of DMD.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2017 ( Subtotal = $0 )
2017	2015	STRYKAGEN CORP.	1664 N VIRGINIA ST ROSS HALL 106 MS 321	RENO	NV	89557	WASHOE	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	8/3/2017	NEW	$0
														Subtotal = $0

Issue Date FY: 2015 ( Subtotal = $224,999 )
2015	2015	STRYKAGEN CORP.	1664 N VIRGINIA ST ROSS HALL 106 MS	RENO	NV	89557	WASHOE	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	3/18/2015	NEW	$224,999
														Subtotal = $224,999

Grand Total All Awards = $224,999

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Optimization of an integrin enhancing molecule for the treatment of Duchenne muscular dystrophy

Award Number: R41AR067019

ORGANIZATION: NATIONAL INSTITUTE OF ARTHRITIS & MUSCULOSKELETAL & SKIN DISEASES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 04/01/2015

PERIOD OF PERFORMANCE END DATE: 03/31/2017

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