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

> **NIH NIH N01** · LEIDOS BIOMEDICAL RESEARCH, INC. · 2022 · $9,308,321

## 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 organization:** LEIDOS BIOMEDICAL RESEARCH, INC.
- **Principal Investigator:** VENKATA MANGALAMPALLI
- **Activity code:** N01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $9,308,321
- **Award type:** —
- **Project period:** 2022-09-12 → 2027-09-11

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10716633

## Citation

> US National Institutes of Health, RePORTER application 10716633, NCATS-DPI-22-0101 - CREATION OF AN OPEN-SOURCE PROCESS TO MANUFACTURE AAV VECTOR-BASED GENE THERAPY CANDIDATES FOR NON-COMMERCIAL CLINICAL STUDIES (75N91019D00024-0-759102200016-1). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10716633. Licensed CC0.

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