# CMC SUPPORT FOR NCATS RARE DISEASE PLATFORM GENE THERAPY TRIAL PROJECTS

> **NIH NIH N01** · LEIDOS BIOMEDICAL RESEARCH, INC. · 2021 · $7,268,296

## Abstract

The objective of this SOW is for Leidos-BDP to conduct the chemistry, manufacturing, and controls (CMC) development and manufacturing tasks to enable regulatory filing and clinical studies for two gene therapy products to treat methylmalonic acidemia (MMA).
The objective has two sub-objectives:
Delivery of preclinical and clinical lots and associated data packages to timely enable clinical trials; Development of robust and scalable process for manufacture of AAV9 gene vectors, with IP freedom to operate.
The two sub-objectives may be addressed separately and in parallel, e. g. initial clinical lots may not necessarily be manufactured using IP-free or optimized technology, with technology development continuing while initial lots are manufactured using currently available methods.

## Key facts

- **NIH application ID:** 10414755
- **Project number:** 75N91019D00024-P00002-759101900138-1
- **Recipient organization:** LEIDOS BIOMEDICAL RESEARCH, INC.
- **Principal Investigator:** GEORGE MITRA
- **Activity code:** N01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $7,268,296
- **Award type:** —
- **Project period:** 2019-09-23 → 2024-09-22

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10414755

## Citation

> US National Institutes of Health, RePORTER application 10414755, CMC SUPPORT FOR NCATS RARE DISEASE PLATFORM GENE THERAPY TRIAL PROJECTS (75N91019D00024-P00002-759101900138-1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10414755. Licensed CC0.

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