NCATS-DPI-22-0101 - CREATION OF AN OPEN-SOURCE PROCESS TO MANUFACTURE AAV VECTOR-BASED GENE THERAPY CANDIDATES FOR NON-COMMERCIAL CLINICAL STUDIES

NIH RePORTER · NIH · N01 · $9,308,321 · view on reporter.nih.gov ↗

Abstract

Project Objectives: • Develop an open- access process for the generation of at least two of the AAV serotypes broadly in current use. All AAV serotypes will use a GFP gene, and a gene of interest (GOI) identified by NCATS to validate the lab scale process. • Demonstrate the scalability of that process by manufacturing the NCATS GOI for all AAV serotypes (non-GMP) at 50 L pilot scale and subsequently at a 200 L bioreactor scale. • Develop all the required analytical methods for in-process testing and quality attributes of the material generated throughout the development stages to support regulatory filings. • Provide comprehensive project management support • Provide quality audit support during the process development and final process definition stage • Provide regulatory support for preparation of the Chemistry, Manufacturing and Controls (CMC) section of an Investigational New Drug (IND) application • Manufacture AAV-GOI material at cGMP for clinical use and validate relevant analytical methods for release and stability analysis. • Perform stability studies on the drug substance and drug product for up to 48 months • Provide inventory support for storage and shipping of cell banks, drug substance and drug product

Key facts

NIH application ID
10716633
Project number
75N91019D00024-0-759102200016-1
Recipient
LEIDOS BIOMEDICAL RESEARCH, INC.
Principal Investigator
VENKATA MANGALAMPALLI
Activity code
N01
Funding institute
NIH
Fiscal year
2022
Award amount
$9,308,321
Award type
Project period
2022-09-12 → 2027-09-11