Dr. Traci Mondoro

The Cure Sickle Cell Initiative provides assistance with navigating the regulatory pathway for genetic therapies.

PACT

The Cure Sickle Cell Initiative collaborates with The Production Assistance for Cellular Therapies (PACT) program, an NIH-wide resource, to provide regulatory assistance to investigators who may require support with the following services: 

  • Guidance for pre-pre-IND INTERACT discussions with FDA
  • INTERACT meeting package preparation
  • Guidance for pre-IND discussions with FDA
  • Pre-IND meeting package preparation
  • Pre-clinical study design
  • Chemistry, Manufacturing and Controls (CMC) development
  • Initial Investigational New Drug (IND) preparation

Gap Analysis

Investigators may request support in the form of a Gap Analysis to shed light on what may be needed to support a successful IND application. The goal of this analysis is to improve understanding of how the data generated from the proof of concept, non-clinical studies, and the CMC section will map to the requirements of an IND application. The Gap Analysis may cover the following areas, as needed:

  • Nonclinical studies to support the proposed clinical development plan
  • Manufacturing process for both nonclinical and clinical trial materials
  • Process development and scale up
  • Process validation
  • SOP development and batch production records
  • Product cryopreservation / labeling / release / shipment
  • Technology transfer
  • Regulatory finalization and IND submission assistance
  • Phase I clinical development plan
  • FDA communications and commitments