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Clinical discovery and validation of delivery vectors to empower cures for CNS diseases

Award Number: RM1NS143853
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 02/15/2026
PERIOD OF PERFORMANCE END DATE: 01/31/2031

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Clinical discovery and validation of delivery vectors to empower cures for CNS diseases - Project Summary & Abstract Developing efficient gene therapies and biologics can unlock new cures for neurological diseases, but is hindered by inefficient delivery, especially to the central nervous system (CNS). Traditional delivery vehicle discovery and validation pipelines rely on cellular and animal models that often fail to predict CNS delivery in patients. What is needed is to assess delivery efficacy in humans at the beginning of the discovery process. We propose to revolutionize the field by discovering and validating vectors in physiologically maintained deceased (PMD) human cadavers. Delivery vector performance in a PMD cadaver will provide confidence in their efficacy in humans and greatly reduce the reliance on preclinical animal discovery research, ultimately increasing the success of clinical trials. Our ethics-informed PMD platform will provide a blueprint for the safe, ethical, and just use of human donors in discovery research. Our discovery platform will help future researchers and clinicians find the most effective vectors to enable gene and biologic therapies that could be used to treat millions of patients with CNS indications.


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: 2026 ( Subtotal = $2,656,684 )
2026	2026	REGENTS OF THE UNIVERSITY OF CALIFORNIA, SAN FRANCISCO, THE	1855 FOLSOM ST STE 425	SAN FRANCISCO	CA	94103	SAN FRANCISCO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	2/6/2026	NEW	$2,656,684
														Subtotal = $2,656,684

Grand Total All Awards = $2,656,684

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Clinical discovery and validation of delivery vectors to empower cures for CNS diseases

Award Number: RM1NS143853

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 02/15/2026

PERIOD OF PERFORMANCE END DATE: 01/31/2031

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