CMC SUPPORT FOR NCATS RARE DISEASE PLATFORM GENE THERAPY TRIAL PROJECTS

NIH RePORTER · NIH · N01 · $5,077,161 · view on reporter.nih.gov ↗

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
11196049
Project number
75N91019D00024-P00008-759101900138-1
Recipient
LEIDOS BIOMEDICAL RESEARCH, INC.
Principal Investigator
GEORGE MITRA
Activity code
N01
Funding institute
NIH
Fiscal year
2024
Award amount
$5,077,161
Award type
Project period
2019-09-23 → 2027-09-18