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Galectin-1 Protein Therapy for Congenital Muscular Dystrophy

Award Number: R41AR073696
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: 07/01/2018
PERIOD OF PERFORMANCE END DATE: 06/30/2020

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Galectin-1 Protein Therapy for Congenital Muscular Dystrophy - Abstract Laminin-¿2 related congenital muscular dystrophy (LAMA2-CMD), also known as Merosin Deficient Congenital Muscular Dystrophy (MDC1A), is a fatal muscle wasting disease that affects patients from birth. LAMA2-CMD results from mutations in the LAMA2 gene resulting in loss of laminin-¿2 protein. Laminin-¿2 is required for the formation of the heterotrimers laminin-211 (¿2, ¿1, ¿1) and laminin-221 (¿2, ¿1, ¿¿), which are major constituents of the skeletal and cardiac muscle basal lamina. LAMA2-CMD patients exhibit severe muscle weakness from birth, are confined to a wheelchair, require ventilator assistance to breathe and have reduced life expectancy. There is currently no effective treatment or cure for LAMA2-CMD and all affected children will die from this genetic disease. Galectin-1 is a 14kDa protein localized in the myomatrix that interacts with laminin and ¿7¿1 integrin and has been shown to stabilize dystrophic muscle. The Burkin lab has recently shown that treatment with recombinant human Galectin-1 protein leads to elevated levels of ¿7¿1 integrin in the skeletal muscle of the dyW mouse model of LAMA2-CMD. Galectin-1 treatment increased the capacity for muscle repair and functioned to limit muscle inflammation and fibrosis. Galectin-1 could interact with other laminin isoforms that are up-regulated in LAMA2-CMD to stabilize their interaction with muscle. What is unclear from these studies is the mechanism of action, the optimal dosing and pharmacokinetic and pharmacodynamic (PK/PD) and toxicology profile of exogenously delivered Galectin-1 to prevent disease progression in laminin-¿2 deficient muscle. In the proposal Strykagen will optimize Galectin-1 dosing, define the PK/PD profile, toxicology and determine the effectiveness of optimized Galectin-1 protein treatment on disease progression in a mouse model of LAMA2-CMD. Together these studies will contribute to an IND application to develop recombinant human Galectin-1 protein as a novel treatment for patients with LAMA2- CMD.


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: 2021 ( Subtotal = $0 )
2021	2018	STRYKAGEN CORP.	1664 N VIRGINIA ST ROSS HALL 106 MS 321	RENO	NV	89557	WASHOE	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	12/4/2020	NEW	$0
														Subtotal = $0

Issue Date FY: 2018 ( Subtotal = $225,000 )
2018	2018	STRYKAGEN CORP.	1664 N VIRGINIA ST ROSS HALL 106 MS 321	RENO	NV	89557	WASHOE	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	6/22/2018	NEW	$225,000
														Subtotal = $225,000

Grand Total All Awards = $225,000

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Galectin-1 Protein Therapy for Congenital Muscular Dystrophy

Award Number: R41AR073696

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: 07/01/2018

PERIOD OF PERFORMANCE END DATE: 06/30/2020

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