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Treatment of muscular symptoms in Pompe rare disease via lectin assisted ERT delivery

Award Number: R43AR077353
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: 08/15/2020
PERIOD OF PERFORMANCE END DATE: 07/30/2022

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Treatment of muscular symptoms in Pompe rare disease via lectin assisted ERT delivery - The current inability to effectively deliver corrective doses of lysosomal enzymes to key cells involved in muscular disease symptoms remains a significant hurdle for rare lysosomal disorders (LSD) such as Pompe disease and other similar diseases with significant muscular-skeletal pathologies. BioStrategies LC is developing the plant lectin RTB as a carrier capable of expanding enzyme delivery to “hard-to-treat” organs and tissues including the brain, heart, and skeletal muscle tissues. Lectin-mediated ERT delivery has recently shown promise in other LSDs including MPS I and GM1. This SBIR is focused on developing a “delivery-enhanced” enzyme replacement therapy (ERT) for patients with Pompe disease. Pompe is an autosomal recessive LSD caused by genetic deficiencies in acid alpha-glucosidase (GAA) leading to progressive multi-organ pathologies particularly focused on severe symptoms in smooth, cardiac and skeletal muscles. Pompe disease in its severe forms can lead to death due to extensive cardiomyopathy and respiratory muscle failure. Our long-term goal in this research project is to bring an ERT capable of treating the full spectrum of progressive muscular and other disease manifestations to Pompe patients. Objectives of this Phase I SBIR feasibility study are to produce bioactive GAA:RTB fusions and demonstrate ERT product delivery into human myocytes, correction of lysosomal phenotype in Pompe cells, and biodistribution to skeletal muscle, heart, and other tissues in the Pompe mouse model. Success in Phase I feasibility goals will support moving on to rigorous Phase II SBIR follow- up preclinical assessments aimed at moving this promising ERT product to an IND. The feasibility established here will also support expanding the RTB carrier system to additional ERT drugs and therapeutics for other diseases having life-threatening muscle pathologies.


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: 2023 ( Subtotal = $0 )
2023	2020	BIOSTRATAGIES LC	504 UNIVERSITY LOOP E	JONESBORO	AR	72401	CRAIGHEAD	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	5/2/2023	NEW	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $250,000 )
2020	2020	BIOSTRATAGIES, LC	504 UNIVERSITY LOOP EAST	JONESBORO	AR	72401	CRAIGHEAD	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	8/11/2020	NEW	$250,000
														Subtotal = $250,000

Grand Total All Awards = $250,000

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Treatment of muscular symptoms in Pompe rare disease via lectin assisted ERT delivery

Award Number: R43AR077353

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: 08/15/2020

PERIOD OF PERFORMANCE END DATE: 07/30/2022

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