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Pre-clinical development of SPY-DYS45-55, a CRISPR/Cas9 platform for Duchenne muscular dystrophy

Award Number: R44AR075469
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: 08/01/2019
PERIOD OF PERFORMANCE END DATE: 03/31/2023

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Pre-clinical development of SPY-DYS45-55, a CRISPR/Cas9 platform for Duchenne muscular dystrophy - MyoGene Bio is a startup dedicated to developing cutting edge genetic therapies for muscle diseases. In this proposal, the company will advance development of our potential therapeutic, SPY-DYS45-55, a CRISPR/Cas9 gene editing platform for Duchenne muscular dystrophy (DMD) in conjunction with research partners at UCLA. Duchenne is a devastating muscle wasting disorder with no cure that is caused by out-of-frame mutations in the DMD gene. SPY-DYS45-55 removes a mutational hotspot in DMD to restore the reading frame for half of all Duchenne patients by generating an in-frame exon 45-55 deletion. This deletion is associated with a very mild phenotype in human Becker muscular dystrophy patients. Systemic delivery of gene editing platforms to muscle represents a significant challenge. Certain serotypes of recombinant adenoassociated virus (AAV) have tropism to skeletal muscle and heart. However, the immune response to the virus prohibits repeated administration of AAV. Furthermore, pre-existing immunity in some individuals (anticipated to be present in up to 70% of adults), precludes their eligibility to take advantage of this treatment. The goal of this Fast Track proposal is to devise strategies to overcome these challenges. In Phase I, we will determine an optimal immunosuppression regimen that allows redosing of AAV in order to improve the efficacy and applicability of SPY-DYS45-55. In Phase II, we will assess the long-term functional benefit, toxicity, and off-target activity from single or multiple injections of AAV-SPY-DYS45-55 in our novel mouse model containing a mutated human DMD gene. Ultimately, these studies will generate the initial pre-clinical data needed to translate SPY-DYS45-55 to patients with Duchenne muscular dystrophy.


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: 2024 ( Subtotal = $0 )
2024	2022	MYOGENE BIO LLC	7098 MIRATECH DR STE 120	SAN DIEGO	CA	92121	SAN DIEGO	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	3	12/18/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2022 ( Subtotal = $499,207 )
2022	2022	MYOGENE BIO LLC	570 WESTWOOD PLZ STE 6350	LOS ANGELES	CA	90095	LOS ANGELES	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	3	11/23/2021	NON-COMPETING CONTINUATION	$499,207
														Subtotal = $499,207

Issue Date FY: 2021 ( Subtotal = $0 )
2021	2020	MYOGENE BIO LLC	570 WESTWOOD PLZ STE 6350	LOS ANGELES	CA	90095	LOS ANGELES	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	2	5/28/2021	EXTENSION WITH OR WITHOUT FUNDS	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $493,728 )
2020	2020	MYOGENE BIO LLC	570 WESTWOOD PLZ STE 6350	LOS ANGELES	CA	90095	LOS ANGELES	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	2	7/2/2020	EXTENSION WITH OR WITHOUT FUNDS	$493,728
2020	2019	MYOGENE BIO LLC	570 WESTWOOD PLZ STE 6350	LOS ANGELES	CA	90095	LOS ANGELES	USA	Arthritis, Musculoskeletal and Skin Diseases Research	001	1	7/9/2020	NEW	$0
														Subtotal = $493,728

Issue Date FY: 2019 ( Subtotal = $195,296 )
2019	2019	MYOGENE BIO LLC	1370 VETERAN AVE 208	LOS ANGELES	CA	90024	LOS ANGELES	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	7/30/2019	NEW	$195,296
														Subtotal = $195,296

Grand Total All Awards = $1,188,231

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Pre-clinical development of SPY-DYS45-55, a CRISPR/Cas9 platform for Duchenne muscular dystrophy

Award Number: R44AR075469

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: 08/01/2019

PERIOD OF PERFORMANCE END DATE: 03/31/2023

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