Please note: while our cooperative can offer support in many ways, we do not offer direct financial project support or financial support for clinical trials.
IND-enabling pre-GMP process development guidance. The translation from the bench to the clinic requires performing a series of laboratory studies of increasing rigor from proof-of-principle, pre-clinical studies that may support pre-IND discussion with FDA, and formal IND enabling efficacy and toxicology studies. Investigators can be assisted in planning these studies and introduced to the appropriate resources; however, direct support for in vivo animal work is not available.
Pre-GMP vector production and transduction manufacturing process and product development. Technologies for vector and cell manufacture in support of gene therapy clinical trials require processes, equipment and facilities that are vastly different from those applicable to research or clinical laboratories. We will provide a collaborative platform to facilitate bench-to-bedside translation of clinical manufacturing of HSC-based gene and cell therapies for pediatric diseases.
Trial-supporting assay development/implementation. Translation of a gene therapy trial proposal to implementation is hampered by lack of support for the development and standardization of trial-supporting assays. We can support trial investigators with assay development.
Regulatory pathway planning and assistance. Experimental gene and cellular therapies are subject to highly specified regulatory oversights that can present a significant barrier to inexperienced sites. We will provide seasoned regulatory assistance via access to a regulatory affairs professional who is experienced in the nuances of gene therapy regulatory filings.
Development of reliance agreements to allow rapid implementation of new protocols via a simplified IRB review process. The creation of a designated pediatric gene therapy IRB and the use of a single IRB will assure the highest quality gene therapy human research and will improve efficiency of review and minimize regulatory burden.
Assistance in clinical trial protocol development, implementation and data management including packaging of data for potential licensing opportunities.
Assistance in clinical trials compliance, monitoring and reporting.