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Rapid egg bioreactor construction for Fabry disease therapeutics

Award Number: R41GM146516
ORGANIZATION: NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/08/2022
PERIOD OF PERFORMANCE END DATE: 08/07/2024

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Rapid egg bioreactor construction for Fabry disease therapeutics - Abstract Fabry disease is a lysosomal storage disease (LSD) affecting 1 in 40,000 to 117,000 live births. The current treatment is enzyme replacement therapy (ERT) with recombinant human α-galactosidase A (GalA). A major healthcare burden from this treatment is the high drug cost at $350k per patient-year. To reduce the drug cost for Fabry disease, we propose to develop a rapid approach to construct cost- effective chicken egg bioreactor for GalA production, which has the potential to produce 50 mg recombinant protein per egg for less than $10. Previously, a similar egg bioreactor was developed for another LSD, lysosomal acid lipase (LAL) deficiency. The drug sebelipase alfa for LAL deficiency has been the only FDA- approved therapeutics from transgenic chicken eggs. The traditional blastoderm transgenesis of chicken is limited by its efficiency and thousands of chickens will be screened to obtain a positive clone. Another mainstream approach with primordial germ cells (PGCs) suffers from the fact that PGCs contribute to the germline but not somatic cells, and only generate male G0 chimeras, delaying the assessment of chimerism until six months later when their semen is genotyped. Our novel approach will generate chimeric roosters and hens with both somatic and germline chimerism, which greatly accelerate transgenic chicken construction. In Phase I, we will engineer chicken cell lines with GalA expression cassette knock-in at the highly transcribed OVA locus and construct the chimeric chickens. The chimeric hens will provide eggs for rapid assessment of GalA abundance and activity. This validation will show the feasibility of this approach for the future generation of chicken with heterozygous and homozygous OVA-GalA knock-in.


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: 2025 ( Subtotal = $0 )
2025	2022	ATGC INC	3670 NORTHBROOK DR	SUPERIOR TOWNSHIP	MI	48198	WASHTENAW	USA	Biomedical Research and Research Training	000	1	11/18/2024	NEW	$0
														Subtotal = $0

Issue Date FY: 2022 ( Subtotal = $259,520 )
2022	2022	ATGC INC	540 AVIS DR	ANN ARBOR	MI	48108	WASHTENAW	USA	Biomedical Research and Research Training	000	1	8/5/2022	NEW	$259,520
														Subtotal = $259,520

Grand Total All Awards = $259,520

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Rapid egg bioreactor construction for Fabry disease therapeutics

Award Number: R41GM146516

ORGANIZATION: NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 08/08/2022

PERIOD OF PERFORMANCE END DATE: 08/07/2024

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