EFFICACY AND SAFETY EVALUATION OF A LEAD GENE THERAPY CANDIDATE IN PROPIONIC ACIDEMIA (PA) DISEASE MOUSE MODELS AND BIOANALYTICAL ASSAY METHODS DEVELOPMENT.

NIH RePORTER · NIH · N01 · $51,059 · view on reporter.nih.gov ↗

Abstract

The National Center for Advancing Translational Sciences (NCATS), through its Therapeutics for Rare and Neglected Diseases (TRND) program, is collaborating with the National Human Genome Research Institute (NHGRI) to advance an AAV9 Propionyl-CoA Carboxylase (PCCA) gene therapy candidate as a possible treatment for Propionic Acidemia (PA). The overall objective of this task order is for the contractor to evaluate the efficacy and safety of an Adeno-associated virus 9 (AAV9) Propionyl-CoA Carboxylase (PCCA) gene therapy candidate in propionic acidemia (PA) disease mouse models, previously developed at NHGRI, and develop bioanalytical assay methods that will be used to measure biodistribution and enzyme activity resulting from PCCA transgene expression.

Key facts

NIH application ID
11042082
Project number
75N95019D00012-P00003-759502000005-1
Recipient
LOVELACE BIOMEDICAL RESEARCH INSTITUTE
Principal Investigator
MIKE ROBERTSON
Activity code
N01
Funding institute
NIH
Fiscal year
2024
Award amount
$51,059
Award type
Project period
2020-09-25 → 2024-10-31