NATIONAL INSTITUTES OF HEALTH (NIH), INTRAMURAL RESEARCH PROGRAM (IRP), GENE THERAPY TASK FORCE (GTTF), CONTRACT DEVELOPMENT AND MANUFACTURING ORGANIZATIONS (CDMOS), SPECIALIZED SUPPORT SERVICESS4 AX

Axle Informatics, Llc, North Bethesda MD

Investigators

Abstract

The advancement of gene therapy research can offer effective and rapid treatments for multiple disorders and is a current priority for the National Institutes of Health (NIH). Concerted efforts across the NIH Intramural Research Program (IRP) are requiring increased support for advancement of gene therapies and new product development capabilities to test treatments. There are primary gene therapy challenges facing intramural investigators. First, as biopharmaceutical companies use fewer rare diseases as proofs of concept, the non-profit translational scientist is becoming more significant in advancing therapies for rare diseases. Second, this is a difficult process for intramural investigators to initiate and challenging to navigate, presenting a major barrier for those new to translation of gene therapies to the clinic. Intramural investigators without prior experience or commercial partners lack support and guidance to embark on clinical gene therapy projects. As a result of these challenges and the opportunities to advance gene therapy research, the NIH Intramural Research Program (IRP) formed a Gene Therapy Task Force (GTTF) of senior scientists to assess viable options to meet this demand. The Task Force focused on identifying potential contract development and manufacturing organizations (CDMOs) to provide commercial manufacturing of cell and gene therapies at a more comprehensive level. To assess the viability of this approach, the Task Force is seeking a capability test of a target CDMO as proof-of-concept for capability clearance. The sample CDMO shall have preclinical through commercial manufacturing capabilities of cell and gene therapies, component raw materials, process development, plasmid DNA, viral vectors, cell banking, cell processing, and support testing capabilities within one research facility for design application and use. The CDMO shall have the capability to assist investigators who lack the knowledge or infrastructure to bring innovative projects to the clinic.