NIH Support for the development of therapeutic platform approaches to treat monogenic disease
- N1C Moderator
- Dec 1
- 1 min read
Updated: Dec 2
December 15, 2025
Philip J. (P.J.) Brooks is the Acting Director of NCATS’ Division of Rare Diseases Research Innovation. He also is the working group co-coordinator for the NIH Common Fund programs on Somatic Cell Genome Editing and the Newborn Screening by Whole-Geome Sequencing ( NBSxWGS | NIH Common Fund ). He is also one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project and the co-chair of the Bespoke Gene Therapy Consortium. He also represents NCATS in the International Rare Diseases Research Consortium (IRDiRC).
Dr. Brooks was selected as the recipient of the 2022 Sonia Skarlatos Public Service Award by the American Society of Gene & Cell Therapy for consistently fostering and enhancing the field of gene and cell therapy.
Prior to joining NCATS, Dr. Brooks was an investigator in the NIH intramural program, where he focused on rare neurologic diseases resulting from defective DNA repair, including xeroderma pigmentosum, Cockayne syndrome, ataxia telangiectasia, and Fanconi anemia.
Dr. Brooks will discuss relevant examples from the following NIH supported projects on therapeutic platforms:
and newborn screening
