Tuesday, October 7, 2025 10/7/2025

Skin-targeted Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa

Award Number: R01AR063070
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: 04/01/2013
PERIOD OF PERFORMANCE END DATE: 08/31/2030

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Skin-targeted Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa - Recessive dystrophic epidermolysis bullosa (RDEB) is a rare inherited mucocutaneous disease caused by mutations to the COL7A1 gene. Advancements in gene therapy have opened avenues for treatment by introducing a functional copy of the mutated gene in the patient’s cells. Viral vectors, gene editing nucleases, base editors, and prime editors are potential strategies for restoring expression of COL7A1. Lenti-, retro-, and herpes simplex virus (HSV) approaches are all being employed clinically. Ex vivo cell correction with integrating vectors (i.e. lenti- or retroviral) requires specialized facilities for engineering cells and these systems pose insertional mutagenic risks that are a consideration in the RDEB phenotype that has a predisposition to aggressive squamous cell carcinoma development. HSV-1 is a non-integrating vector that reduces genome toxicity related safety concerns but episomal nucleic acid could be lost over time. All of these viral vector systems mediate supranormal gene expression levels. In situ, locus specific correction is desirable as it retains the endogenous gene regulatory network and minimizes insertional mutagenesis. Here we will evaluate highly promising genome engineering and delivery approaches as part of a strategy to restore COL7A1 expression in cells ex vivo and in vivo. In the first aim, we will define and optimize evolved engineered Prime Assisted Site- Specific Integrase Gene Editing (eePASSIGE), a powerful addition to the genome engineering armamentarium. Polymeric nanoparticles (PNP) will be employed for the delivery of eePASSIGE nucleic acids that will install a full length COL7A1 cDNA into the endogenous locus ex vivo and in vivo. Mesenchymal stromal cells (MSC) that express ATP-binding cassette superfamily member (ABCB5) display homing and tissue repair properties and we will evaluate the ability of eePASSIGE corrected autologous ABCB5+ MSC to correct RDEB pathology in mice in vivo. In the second aim, we will develop a novel microfluidics-based epidermal poration device to deliver eePASSIGE PNPs topically to mice for in vivo, permanent COL7A1 gene correction. Both aims will then be synergized to assess the combinatorial potential of topical and cell-based therapy to ameliorate the cutaneous and internal disease manifestations toward filling a therapeutic gap in current treatment options. Our proposed experiments will develop a novel ex vivo and in vivo genome editing strategy that could be broadly applied across all RDEB patient cohorts and that will enhance overall efficiency and correct the full spectrum of the disease.


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 = $329,375 )
2025	2025	REGENTS OF THE UNIVERSITY OF MINNESOTA	2221 UNIVERSITY AVE SE STE 100	MINNEAPOLIS	MN	55414	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	11	9/4/2025	COMPETING CONTINUATION	$329,375
														Subtotal = $329,375

Issue Date FY: 2024 ( Subtotal = $330,770 )
2024	2024	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	10	8/14/2024	NON-COMPETING CONTINUATION	$330,770
														Subtotal = $330,770

Issue Date FY: 2023 ( Subtotal = $341,000 )
2023	2023	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	9	8/18/2023	NON-COMPETING CONTINUATION	$341,000
														Subtotal = $341,000

Issue Date FY: 2022 ( Subtotal = $337,590 )
2022	2022	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE # 224	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	8	8/31/2022	NON-COMPETING CONTINUATION	$337,590
														Subtotal = $337,590

Issue Date FY: 2021 ( Subtotal = $330,770 )
2021	2021	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE # 224	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	001	7	9/17/2021	NON-COMPETING CONTINUATION	$330,770
2021	2020	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE # 224	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	6	8/26/2021	COMPETING CONTINUATION	$0
														Subtotal = $330,770

Issue Date FY: 2020 ( Subtotal = $339,167 )
2020	2020	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE # 224	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	6	8/28/2020	COMPETING CONTINUATION	$339,167
														Subtotal = $339,167

Issue Date FY: 2018 ( Subtotal = $0 )
2018	2017	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE # 224	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	5	7/26/2018	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2017 ( Subtotal = $435,018 )
2017	2017	REGENTS OF THE UNIVERSITY OF MINNESOTA	200 OAK ST SE # 224	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	5	6/1/2017	NON-COMPETING CONTINUATION	$435,018
														Subtotal = $435,018

Issue Date FY: 2016 ( Subtotal = $435,018 )
2016	2016	UNIVERSITY OF MINNESOTA, OFFICE OF STUDENT FINANCE AID	106 PLEASANT SE, 210 FRASER HL	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	4	3/16/2016	EXTENSION WITH OR WITHOUT FUNDS	$435,018
														Subtotal = $435,018

Issue Date FY: 2015 ( Subtotal = $435,018 )
2015	2015	UNIVERSITY OF MINNESOTA, OFFICE OF STUDENT FINANCE AID	106 PLEASANT SE, 210 FRASER HL	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	3	2/20/2015	NON-COMPETING CONTINUATION	$435,018
														Subtotal = $435,018

Issue Date FY: 2014 ( Subtotal = $437,074 )
2014	2014	UNIVERSITY OF MINNESOTA, OFFICE OF STUDENT FINANCE AID	106 PLEASANT SE, 210 FRASER HL	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	2	4/10/2014	NON-COMPETING CONTINUATION	$437,074
														Subtotal = $437,074

Issue Date FY: 2013 ( Subtotal = $458,962 )
2013	2013	UNIVERSITY OF MINNESOTA, OFFICE OF STUDENT FINANCE AID	106 PLEASANT SE, 210 FRASER HL	MINNEAPOLIS	MN	55455	HENNEPIN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	3/20/2013	NEW	$458,962
														Subtotal = $458,962

Grand Total All Awards = $4,209,762

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Skin-targeted Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa

Award Number: R01AR063070

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: 04/01/2013

PERIOD OF PERFORMANCE END DATE: 08/31/2030

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