Goal 3: Advance Translational Research

Expediting Gene and Cell Therapies to the Clinics

What methodologies will best enhance the translation of technologies for gene and cell therapies into potential products for clinical application and commercial development?


In considering a strategy for NHLBI investment in gene therapy, it is important to note that we are only at the beginning of a revolution that will eventually impact biomedical research across a broad range of specialties. NHLBI/NIH needs to create funding mechanisms that support key steps in these translational programs. For example, support of IND enabling nonclinical studies, or the manufacturing of clinical grade vector, are the types of biomedical research that are difficult to support through traditional investigator- initiated mechanisms.

Substantial gaps exist in key translational steps such as improved processes for vector manufacturing and better assays for characterizing vectors. NHLBI/NIH should support the development of these regulatory sciences and help create the infrastructure in areas of nonclinical safety testing and vector manufacturing that is accessible to academic investigators.

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66 net votes
97 up votes
31 down votes
Idea No. 103