Gene and cell therapies, especially for rare diseases, require complex product-specific development of pre-clinical studies, costly GMP manufacturing, unique laboratory assays for monitoring, and extraordinary regulatory management to initiate, perform, and oversee clinical trials that provide initial assessments of safety and efficacy. Due to the rarity of many serious and life-threatening genetic diseases in the pediatric population, an increasing number of which may be curable using emerging gene and cell therapy approaches, a significant barrier to translation of these innovative therapies and future gene editing approaches is the complex regulatory environment involving biologicals and early phase human studies in children. With the recent successes in curative treatments of several monogenic diseases and emergence of cellular immune therapies in cancer, a key next step in the dissemination of these therapeutic applications is broadening the number of centers participating in foundational pediatric clinical trials utilizing these approaches.
The overall goal of the Growing Gene and Cell Therapy (GGACT) cooperative is to support investigators to rapidly translate complex gene and cell therapies to early phase, investigator-initiated clinical trials. The founding collaborative centers (Boston Children’s Hospital, Cincinnati Children’s Hospital Medical Center, and the University of California Los Angeles) have a history of working together to implement pioneering gene and cell therapy pediatric trials. Their collective experiences have required the development of both expertise and significant infrastructure at each institution that focuses on preclinical models of disease and testing, process development, GMP vector production and cellular manufacturing, protocol development and implementation, trial and data management, and regulatory expertise that is specifically focused on early phase biologics in a manner suitable for transferring to industry for later phase studies.
GGACT is supported by NIH/NCATS U01TR001814.
View support services available and complete application instructions here.
- Letter of Intent: April 2, 2018
- Final applications: June 25, 2018
- Notification of support: late August 2018