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Validated Immunoassays to Accelerate Therapeutic Genome Editing INDs

Award Number: U01AI191209
ORGANIZATION: NATIONAL INSTITUTE OF ALLERGY & INFECTIOUS DISEASES
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 05/22/2025
PERIOD OF PERFORMANCE END DATE: 04/30/2028

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Validated Immunoassays to Accelerate Therapeutic Genome Editing INDs - PROJECT SUMMARY / ABSTRACT This application is submitted in response to RFA-RM-24-007 with the overriding objective of providing validated assays to understand the immunology of gene editors in pre-clinical studies supporting investigational new drugs (INDs) and eventually in clinical trials. Immunomonitoring is critically important to ensure safety as gene editing therapies transition to the clinical setting for a wide range of applications and diseases. The goals of these studies will be accomplished through the following Specific Aims: (1) Validate a cytokine flow cytometry assay measuring T-cell immunity to commonly used gene editors, vectors, and related peptides from the human peptidome; and (2) Provide a validated multiplex assay for binding antibodies against the domains of relevant gene-editing proteins. We will validate these assays by demonstrating accuracy, precision, specificity, linearity, and range with Quality Assurance monitoring. The assays proposed are based on cytokine flow cytometry and the adaptation of a multiplex microbead immunoassay to accommodate multiple antigens of interest for genome editors being tested for future human use. We will also track the implications of T-cell responses to editors or self-proteins in vivo using total-body positron emission tomography (PET) to assist in fully characterizing the assays proposed. These studies will accelerate the progress of safe and effective somatic cell genome editing therapies to the clinic by providing rigorously validated assays for multiple editing strategies. The assays are designed to address many Cas9-only editors, prime editors, and base editors— while in addition providing a rigorous framework that can be adapted to future gene editors. FDA approved PET imaging methods will also provide a powerful tool for accurately understanding in vivo events and best practices for IND-enabling studies and clinical trials.


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 = $715,222 )
2025	2025	UNIVERSITY OF CALIFORNIA, DAVIS	1850 RESEARCH PARK DR STE 300	DAVIS	CA	95618	YOLO	USA	Trans-NIH Research Support	000	1	5/22/2025	NEW	$715,222
														Subtotal = $715,222

Grand Total All Awards = $715,222

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Validated Immunoassays to Accelerate Therapeutic Genome Editing INDs

Award Number: U01AI191209

ORGANIZATION: NATIONAL INSTITUTE OF ALLERGY & INFECTIOUS DISEASES

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

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

PERIOD OF PERFORMANCE START DATE: 05/22/2025

PERIOD OF PERFORMANCE END DATE: 04/30/2028

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