REGULATORY SUPPORT FOR LEAD GENE THERAPY CANDIDATES AS A TREATMENT FOR ORGANIC ACIDEMIAS, PROPIONIC ACIDEMIA (PA) AND METHYLMALONIC ACIDEMIA TYPE B (MMAB)

Investigators

Abstract

The scope of this task order is for the regulatory strategy, planning, preparation, submission and maintenance of meeting requests and application packages (such as INTERACT meeting, pre-IND meeting, IND submission and maintenance) to the FDA to support the lead candidates for gene therapy treatments.