TASK AREA B: RSA NO. 231 FOR HUMORAL EVASION BY ADENO-ASSOCIATED VIRAL (AAV) VECTORS FOR DR. ASOKAN

NIH RePORTER · NIH · N01 · $218,836 · view on reporter.nih.gov ↗

Abstract

Pre-existing immunity to recombinant adeno-associated viral (AAV) vectors has been a major hindrance to vector dosing and re-administration, which may be required to ensure the durability and long-term efficacy of gene therapy for some diseases. Eliminating neutralizing antibodies against different AAV serotypes that arise from prior exposure could enable effective gene transfer and avoid the problems inherent with pre-existing antibodies. In this pilot study, the PI proposes to assess the ability of a rapid antibody clearing agent (ACA) (cysteine protease) to diminish pre-existing humoral immunity to AAV capsids and determine if this approach could enable AAV vector administration in young seropositive rhesus monkeys. If successful, the proposed approach could potentially expand patient recruitment in gene therapy clinical trials and also increase the availability of nonhuman primates for preclinical studies.

Key facts

NIH application ID
10124656
Project number
75N92019D00040-0-759202000001-1
Recipient
UNIVERSITY OF CALIFORNIA AT DAVIS
Principal Investigator
Alice F Tarantal
Activity code
N01
Funding institute
NIH
Fiscal year
2020
Award amount
$218,836
Award type
Project period
2020-02-03 → 2020-10-31