Wednesday, September 17, 2025 9/17/2025

Immune activation and subject-specific risk in ocular gene therapies

Award Number: K99EY036493
ORGANIZATION: NATIONAL EYE INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: TRAINING/TRAINEESHIP
PERIOD OF PERFORMANCE START DATE: 09/15/2025
PERIOD OF PERFORMANCE END DATE: 08/31/2026

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Immune activation and subject-specific risk in ocular gene therapies - Project summary Gene therapies for heritable eye disorders are being developed at an unprecedented rate, but the immune response, despite being a known reaction to gene therapies, is poorly characterized. Side effects to ocular gene therapies do not affect individuals to the same degree clinically, despite receiving similar dosing and treatments, underscoring the need for understanding subject-specific risk. In this proposal, I will 1) determine the effect of immunosuppression on toxicity risks after intraocular gene therapy, 2) discover genetic variants that modify susceptibility to gene therapy-related toxicity, and 3) test if subject-specific risks can be estimated before treatment using blood. Genotype-phenotype correlations will be performed to map genetic loci associated with toxicity phenotypes in mice with a diverse genetic background. To estimate risk specific to an individual animal, biomarkers in blood will be measured and analyzed by machine learning. The overall objective of the research is to use subject-specific characteristics to predict and mitigate toxicity risks. The long-term goal is to achieve precision medicine via individualized risk assessment for ocular and for all gene therapies. Though the proposed research uses mice as a model organism, the outcomes are highly clinically relevant and have immense potential for making a positive clinical impact. The proposed work will advance personalized design and optimization of ocular gene 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 = $86,858 )
2025	2025	THE UNIVERSITY OF IOWA	105 JESSUP HALL	IOWA CITY	IA	52242	JOHNSON	USA	Vision Research	000	1	9/8/2025	NEW	$86,858
														Subtotal = $86,858

Grand Total All Awards = $86,858

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Immune activation and subject-specific risk in ocular gene therapies

Award Number: K99EY036493

ORGANIZATION: NATIONAL EYE INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: TRAINING/TRAINEESHIP

PERIOD OF PERFORMANCE START DATE: 09/15/2025

PERIOD OF PERFORMANCE END DATE: 08/31/2026

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