NON-CLINICAL SAFETY ASSESSMENT OF GENE THERAPY (PCCA, MMAB, DOK7 AND COLQ) TO SUPPORT PLATFORM GENE THERAPY FOR CONGENITAL MYASTHENIC DISEASES (DOK7, COLQ) AND ACIDEMIA (PCCA, MMAB)

Lovelace Biomedical Research Institute, Albuquerque NM

Investigators

Abstract

This task order is for safety assessment studies of Pave-GT gene therapy to determine its toxicity and evaluate the implications. This memo requests a modification for the following changes: • Obligate $279,231 (FY25) on CLIN #3 for method development of MMAB gene vector. • Obligate $1,745,514 (FY25) on CLIN #4 to exercise Option 2 for 6-month toxicology study of MMAB gene vector. • Delete method development of MMAB gene vector from the descriptions of both CLINs 1 and 2 and de-obligate $279,231 (FY20) funding related to MMAB method development from CLIN 1. • Extend the period of performance end date by twelve (12) months to May 24, 2026. Method development of MMAB gene vector was originally priced to be performed in 2020; however, due to delayed availability of the test article to the Contractor this work is only now beginning in 2025. As a result of the significant delay, on April 4, 2025, the Contractor cannot ensure performance will be completed and the costs invoiced and reimbursed by September 30, 2025. The funds on CLIN #1 for these activities are an expiring line which will be cancelled on September 30, 2025. As a result, the Contractor submitted to the Contracting Officer a revised price to perform method development of MMAB gene vector in 2025 for the total amount $279,231. Because funds for this work were originally obligated at the time of base award, we request de-obligation of $279,231 (FY20) from CLIN #1 and obligation of $279,231 (FY25) on CLIN #3 for this work to begin in 2025. Rare Diseases