Philip J. (P.J.) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project and the International Rare Diseases Research Consortium (IRDiRC)(link is external). He also is the working group co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing, one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project and the co-chair of the Bespoke Gene Therapy Consortium(link is external). 

In May 2022, Brooks was selected as the recipient of the 2022 Sonia Skarlatos Public Service Award(link is external) by the American Society of Gene & Cell Therapy for consistently fostering and enhancing the field of gene and cell therapy.

Brooks received his doctorate in neurobiology from The University of North Carolina at Chapel Hill. After completing a postdoctoral fellowship at The Rockefeller University, he became an investigator in the NIH intramural program, where he developed an internationally recognized research program focused on two distinct areas: the molecular basis of alcohol-related cancer, and rare neurologic diseases resulting from defective DNA repair, including xeroderma pigmentosum, Cockayne syndrome and Fanconi anemia.

Research Topics

Brooks’ research on rare genetic diseases gave him the opportunity to meet with patients and their families, which had a powerful impact on his view of the importance of translational science in rare diseases. He believes that focusing on commonalities across multiple rare diseases is an efficient approach to bring more patients into clinical trials more quickly. He has developed funding opportunities (e.g., RFA-TR-22-029: Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases) and initiated an IRDiRC working group(link is external) on shared molecular etiologies.

Brooks also focuses on gene-targeted therapies, such as gene therapy and gene editing, which have broad therapeutic potential for genetic disorders. However, the ability to deliver these complex molecules to the affected cell types remains the limiting factor in many diseases. Brooks developed funding opportunities to support innovative approaches to delivering genome editors to target cell types (e.g., PAR-20-098 and PAR-20-109). As part of phase 2 of the Somatic Cell Genome Editing program, he is the scientific lead for RFA-RM-22-016: Platform Clinical Trials of Genome Editors in Multiple Diseases, which aims to realize the platform potential of genome editors and increase the efficiency of the process of obtaining regulatory approval for genome editing clinical trials of more than one disease at a time.

Selected Publications

1. The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup
2. The NIH Somatic Cell Genome Editing Program
3. Expanding Rare Disease Drug Trials Based on Shared Molecular Etiology
4. Blinded by the UV Light: How the Focus on Transcription-Coupled NER Has Distracted from Understanding the Mechanisms of Cockayne Syndrome Neurologic Disease