Cellular Therapies (PACT) program has been working with researchers to manufacture cellular therapeutic products, including genetically modified cells, that can be used safely in clinical trials with patients.
NIH spends approximately $100 million on sickle cell disease research each year. Through this initiative, NHLBI seeks to support the development of cell and genetic therapies resources, clinical trials, comparator analyses of different management strategies, data repositories and resources, and patient and advocate engagement activities related to curative therapies for this condition.
Already in 2018, NHLBI committed an additional $7 million to jumpstart the initiative’s research and engagement infrastructure.
NHLBI has named hematologist Edward J. Benz Jr., M.D., President and CEO Emeritus of Dana-Farber Cancer Institute, as the Initiative’s executive director, and the Emmes Corporation, a contract research organization with specialized expertise in clinical trials, gene, and cell therapy development in preclinical studies, as its coordinating center.
The initiative and other research partners will establish a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost-effectiveness.
They will also establish national networks to