VIRALGEN VECTOR CORE SL:1516908 [25-000265]

Investigators

Abstract

NCATS requires stability data and related documentation that demonstrates stability and chain of custody of a cGMP MMA-101 gene therapy product. Viralgen Inc. manufactured MMA-101, a gene therapy product, to meet current good manufacturing practices (cGMP) for Selecta Biosciences Inc under contract to support a Selecta-sponsored clinical trial at the NIH Clinical Center. Selecta was the collaborator of NHGRI, who developed MMA-101. In 2023, Selecta withdrew the FDA-approved Investigatory New Drug (IND) appplication, after opening the MMA-101 study at the NIH Clinical Center, and then merged with Cartesian Inc. towards the end of 2023. At that point, Selecta/Cartesian donated the cGMP MMA-101 gene therapy product along with its rights and IND documentation to NIH/NHGRI, enabling the MMA-101 product to be tested in MMA patients. The objective of this purchase is to acquire the stability data and related documentation produced and owned by Viralgen in order to demonstrate the stability and chain-ofcustody for the MMA-101 cGMP material, to ensure it has not been compromised and can be tested in a clinical trial at NIH. Rare Diseases