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Advancing manufacturing and release testing of a gene editing therapy for Duchenne muscular dystrophy

Award Number: SB1AR084953
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: 09/01/2025
PERIOD OF PERFORMANCE END DATE: 07/31/2028

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Advancing manufacturing and release testing of a gene editing therapy for Duchenne muscular dystrophy - Project Summary Optimization of the manufacturing process and analytical testing for adeno-associated virus (AAV) products is important to put in place as early as possible. Being able to use the same process and release testing throughout development will allow bridging of nonclinical and clinical study material and pooling of data from clinical trials. It is critical to begin work early on analytical development to have assays in place prior to manufacture of cGMP material and IND filing. Here, a dual AAV vector gene editing therapy is being developed for Duchenne muscular dystrophy. In order to include Cas9 inactivation and encompass the large size of Cas9, two vectors are used with one carrying Cas9 and one carrying the guide RNAs. In this proposal, a Design of Experiment (DoE) optimization will be performed on the upstream manufacturing process for both vectors with the aim of improving yield and quality of material. Then product-specific analytics will be optimized and other standard non-compendial assays tested on the two vectors. We will utilize technical assistance from an experienced AAV contract development and manufacturing organization and CMC and regulatory consultants to ensure the work is conducted to regulatory standards. This project will finalize standard operating procedures for manufacturing and release testing of the gene editing therapy for Duchenne. Thus, this work is critical to support the advancement of this dual vector gene editing therapy to the clinic and then commercialization. This therapy is impactful because it targets deletion of DMD exons 45-55, thereby restoring the reading frame for 50% of Duchenne muscular dystrophy patients who have no cure and only limited approved therapies.


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 = $499,300 )
2025	2025	MYOGENE BIO LLC	7098 MIRATECH DR STE 120	SAN DIEGO	CA	92121	SAN DIEGO	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	8/28/2025	NEW	$499,300
														Subtotal = $499,300

Grand Total All Awards = $499,300

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Advancing manufacturing and release testing of a gene editing therapy for Duchenne muscular dystrophy

Award Number: SB1AR084953

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: 09/01/2025

PERIOD OF PERFORMANCE END DATE: 07/31/2028

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